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Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy (MoveDMD®)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02439216
Recruitment Status : Completed
First Posted : May 8, 2015
Results First Posted : September 23, 2022
Last Update Posted : September 23, 2022
Sponsor:
Information provided by (Responsible Party):
Catabasis Pharmaceuticals

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Muscular Dystrophy, Duchenne
Interventions Drug: Edasalonexent
Drug: Placebo
Enrollment 31
Recruitment Details This was a 3-part, Phase 1/2, multi-centre study conducted at 5 study centers in United States.
Pre-assignment Details A total of 32 patients were treated with edasalonexent in this study. 17 patients were enrolled in Part A and 16 completed 7 days of dosing, 1 patient screen failed due to inability to comply with study procedures and did not continue to Part B. A total of 31 patients (16 subjects from Part A and 15 New subjects who entered Part B) were enrolled in Part B, and all completed the 12 weeks of dosing. All 31 patients included in Part B enrolled in the open-label long-term extension phase, Part C.
Arm/Group Title Part A: Dose 1 Part A: Dose 2 Part A: Dose 3 Part B: Dose 1 Part B: Dose 2 Part B: Placebo 1 Part B: Placebo 2 Part C: Dose 1 Part C: Dose 2
Hide Arm/Group Description

Edasalonexent 33 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Placebo 67 mg/kg/day Capsules taken by mouth two times per day Placebo 100 mg/kg/day Capsules taken by mouth three times per day

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Period Title: PartA(7 Day Open-Label Treatment Period)
Started 5 6 6 0 0 0 0 0 0
Completed 4 6 6 0 0 0 0 0 0
Not Completed 1 0 0 0 0 0 0 0 0
Reason Not Completed
Screen failure             1             0             0             0             0             0             0             0             0
Period Title: Part B (12-week Double Blind)
Started [1] 0 0 0 10 [2] 10 [2] 5 [3] 6 [4] 0 0
Completed 0 0 0 10 10 5 6 0 0
Not Completed 0 0 0 0 0 0 0 0 0
[1]
It includes 16 Subjects who completed Part A and continued to Part B plus 15 New subjects who entered Part B
[2]
4 New subjects plus 6 subjects who completed Part A
[3]
3 New subjects plus 2 subjects who completed Part A
[4]
4 New subjects plus 2 subjects who completed Part A
Period Title: Part C (138-week, Open-label Treatment)
Started [1] 0 0 0 0 0 0 0 15 16
Completed 0 0 0 0 0 0 0 8 5
Not Completed 0 0 0 0 0 0 0 7 11
Reason Not Completed
Withdrawal by Subject             0             0             0             0             0             0             0             1             0
Lost to Follow-up             0             0             0             0             0             0             0             1             2
Progressive disease             0             0             0             0             0             0             0             0             3
Withdrawal by Parent or Guardian             0             0             0             0             0             0             0             5             6
[1]
It has Subjects completed Part B
Arm/Group Title Part A -CAT-1004 33 mg/kg/Day Part A - CAT-1004 67 mg/kg/Day Part A - CAT-1004 100 mg/kg/Day Part B - CAT-1004 67 mg/kg/Day Part B - CAT-1004 100 mg/kg/Day Part B: Overall Placebo Total
Hide Arm/Group Description

Edasalonexent 33 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Placebo1:Placebo 67 mg/kg/day Capsules taken by mouth two times per day Placebo2: Placebo 100 mg/kg/day Capsules taken by mouth three times per day Total of all reporting groups
Overall Number of Baseline Participants 5 6 6 10 10 11 48
Hide Baseline Analysis Population Description
Safety population: Part A:The Safety Population was all patients who received at least 1 dose of IP in Part A Part B:The Safety Population was all patients who received at least 1 dose of IP in Part B. Active B or C Safety Population: The Safety Population was all patients who received at least 1 dose of active treatment in Part B and all patients who received placebo in Part B and at least 1 dose of active treatment in Part C.
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Part A Number Analyzed 5 participants 6 participants 6 participants 0 participants 0 participants 0 participants 17 participants
5.2  (1.10) 5.5  (0.55) 5.7  (1.21) 5.5  (0.94)
Part B Number Analyzed 0 participants 0 participants 0 participants 10 participants 10 participants 11 participants 31 participants
5.97  (1.137) 6.00  (1.165) 6.34  (1.032) 6.11  (1.086)
[1]
Measure Analysis Population Description: Analysis population are reported separately based on Part A and Part B.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Part A Number Analyzed 5 participants 6 participants 6 participants 0 participants 0 participants 0 participants 17 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
5
 100.0%
6
 100.0%
6
 100.0%
17
 100.0%
Part B Number Analyzed 0 participants 0 participants 0 participants 10 participants 10 participants 11 participants 31 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
10
 100.0%
10
 100.0%
11
 100.0%
31
 100.0%
[1]
Measure Analysis Population Description: Analysis population are reported separately based on Part A and Part B.
Ethnicity (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Part A Number Analyzed 5 participants 6 participants 6 participants 0 participants 0 participants 0 participants 17 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
1
  16.7%
1
   5.9%
Not Hispanic or Latino
4
  80.0%
6
 100.0%
5
  83.3%
15
  88.2%
Unknown or Not Reported
1
  20.0%
0
   0.0%
0
   0.0%
1
   5.9%
Part B Number Analyzed 0 participants 0 participants 0 participants 10 participants 10 participants 11 participants 31 participants
Hispanic or Latino
2
  20.0%
1
  10.0%
0
   0.0%
3
   9.7%
Not Hispanic or Latino
8
  80.0%
9
  90.0%
11
 100.0%
28
  90.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Analysis Population Description: Analysis population are reported separately based on Part A and Part B.
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Part A Number Analyzed 5 participants 6 participants 6 participants 0 participants 0 participants 0 participants 17 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
5
 100.0%
6
 100.0%
6
 100.0%
17
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Part B Number Analyzed 0 participants 0 participants 0 participants 10 participants 10 participants 11 participants 31 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
  10.0%
0
   0.0%
0
   0.0%
1
   3.2%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
1
   9.1%
1
   3.2%
Black or African American
0
   0.0%
1
  10.0%
0
   0.0%
1
   3.2%
White
9
  90.0%
9
  90.0%
9
  81.8%
27
  87.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
1
   9.1%
1
   3.2%
[1]
Measure Analysis Population Description: Analysis population are reported separately based on Part A and Part B.
Region of Enrollment   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 5 participants 6 participants 6 participants 10 participants 10 participants 11 participants 48 participants
Part A
5
 100.0%
6
 100.0%
6
 100.0%
0
   0.0%
0
   0.0%
0
   0.0%
17
  35.4%
Part B
0
   0.0%
0
   0.0%
0
   0.0%
10
 100.0%
10
 100.0%
11
 100.0%
31
  64.6%
[1]
Measure Analysis Population Description: Analysis population are reported separately based on Part A and Part B
1.Primary Outcome
Title Change From Baseline to Week 12 in the Lower Leg Composite of the MRI T2 Relaxation Time (LLC5-T2) - Part B
Hide Description The LLC5-T2 calculated from the unweighted average of the T2 relaxation times of all 5 lower leg muscles for each patient at each evaluation (the medial gastrocnemius, peroneals, soleus, tibialis anterior, and tibialis posterior muscles). Increases in LLC5-T2 relaxation time indicate muscle damage, inflammation, edema, and fat infiltration and are highly correlated with muscle fat
Time Frame Baseline to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Part B Full Analysis Population: The Full Analysis Population was a modified intent-to-treat population and consisted of all patients who received at least 1 dose of investigational product (IP) in Part B and had a valid baseline and at least 1 post baseline timed function test (TFT) or MRI efficacy assessment.
Arm/Group Title Part B - CAT-1004 67 mg/kg/Day Part B - CAT-1004 100 mg/kg/Day Part B - Placebo (Overall)
Hide Arm/Group Description:

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Placebo 67 mg/kg/day Capsules taken by mouth two times per day

Placebo 100 mg/kg/day Capsules taken by mouth three times per day

Overall Number of Participants Analyzed 10 10 11
Mean (Standard Deviation)
Unit of Measure: msec
Baseline to Week 12 Endpoint - Soleus 2.04  (3.179) 1.26  (2.913) 0.34  (2.380)
Baseline to Week 12 Endpoint - Medial Gastrocnemius 0.35  (2.410) -0.16  (2.003) 1.16  (2.517)
Baseline to Week 12 Endpoint - Tibialis Anterior -0.25  (2.558) 0.01  (1.433) 0.00  (1.878)
Baseline to Week 12 Endpoint - Tibialis Posterior 0.62  (1.107) -1.05  (2.447) -0.42  (2.716)
Baseline to Week 12 Endpoint - Peroneal 0.28  (2.764) 0.63  (2.290) 1.27  (1.630)
2.Secondary Outcome
Title Change From Baseline in the Speed of Completing the 10-meter Walk/Run Test (10MWT) at Week 12 - Part B and Part C
Hide Description

The 10MWT determines the speed to walk a distance of 10 meters. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure.

For patients who are not able to complete the test, the speed of 0 will be imputed.

Time Frame Baseline to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
The Active B or C Full Analysis Population consisted of the pooled patient populations of Parts B and C that received treatment in either part and had valid baseline and efficacy measurements.
Arm/Group Title Part C - CAT-1004 67 mg/kg/Day Part C - CAT-1004 100 mg/kg/Day Part B - Overall Placebo
Hide Arm/Group Description:

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Placebo 67 mg/kg/day Capsules taken by mouth two times per day

Placebo 100 mg/kg/day Capsules taken by mouth three times per day

Overall Number of Participants Analyzed 15 16 11
Mean (Standard Deviation)
Unit of Measure: 10 meters/sec
-0.0008  (0.01742) -0.0041  (0.01584) -0.0100  (0.01627)
3.Secondary Outcome
Title Change From Baseline in the Speed of Completing the 4-Stairs Climb Task at Week 12 - Part B and Part C
Hide Description

The 4-stair climb test determines the speed to climb 4 standard steps. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure.

For patients who are not able to complete the test, the speed of 0 will be imputed.

Time Frame Baseline to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
The Active B or C Full Analysis Population consisted of the pooled patient populations of Parts B and C that received treatment in either part and had valid baseline and efficacy measurements.
Arm/Group Title Part C - CAT-1004 67 mg/kg/Day Part C - CAT-1004 100 mg/kg/Day Part B - Overall Placebo
Hide Arm/Group Description:

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Placebo 67 mg/kg/day Capsules taken by mouth two times per day

Placebo 100 mg/kg/day Capsules taken by mouth three times per day

Overall Number of Participants Analyzed 15 16 11
Mean (Standard Deviation)
Unit of Measure: 4 Stairs/Sec
-0.0032  (0.07487) -0.0046  (0.06223) -0.0121  (0.06647)
4.Secondary Outcome
Title Change From Baseline in the Speed of Completing the Stand From Supine Task at Week 12 - Part B and Part C
Hide Description

The stand from supine test determines the speed to stand from a supine position. The initial measurement was made in seconds and the speed of completing the test (i.e., calculated as the reciprocals of the time to complete; speed=1/time) is provided as the measure.

For patients who are not able to complete the test, the speed of 0 will be imputed.

Time Frame Baseline to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Active B or C Full Analysis Population
Arm/Group Title Part C - CAT-1004 67 mg/kg/Day Part C - CAT-1004 100 mg/kg/Day Part B - Overall Placebo
Hide Arm/Group Description:

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Placebo 67 mg/kg/day Capsules taken by mouth two times per day

Placebo 100 mg/kg/day Capsules taken by mouth three times per day

Overall Number of Participants Analyzed 15 16 11
Mean (Standard Deviation)
Unit of Measure: /Sec
-0.0293  (0.04930) -0.0042  (0.03890) -0.0007  (0.05449)
5.Secondary Outcome
Title Safety and Tolerability Measured by Number of Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
Hide Description A TEAE was any adverse event (AE) that started during or after the first dose of IP through the end of the safety follow-up period. Part B TEAEs were those that started on or after the first dose date in Part B through the date of Week 12 clinic dose. TEAEs for the Part C (Active B or C) analysis were those that started on or after the first dose date of active treatment in Part B or Part C. Drug related AEs included those marked as "Related" or "Possibly Related" to the study treatment.
Time Frame Screening to Week 152
Hide Outcome Measure Data
Hide Analysis Population Description

Part A Safety Population (referred to in tables as All Dosed Patients) The Safety Population was all patients who received at least 1 dose of IP in Part A.

Part B Safety Population: The Safety Population was all patients who received at least 1 dose of IP in Part B.

Active B or C Safety Population: The Safety Population was all patients who received at least 1 dose of active treatment in Part B and all patients who received placebo in Part B and at least 1 dose of active treatment in Part C.

Arm/Group Title Part A - CAT-1004 33 mg/kg/Day Part A - CAT-1004 67 mg/kg/Day Part A - CAT-1004 100 mg/kg/Day Part B - CAT-1004 67 mg/kg/Day Part B - CAT-1004 100 mg/kg/Day Part B - Overall Placebo Part C - CAT-1004 67 mg/kg/Day Part C - CAT-1004 100 mg/kg/Day
Hide Arm/Group Description:

Edasalonexent 33 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Placebo 67 mg/kg/day Capsules taken by mouth two times per day

Placebo 100 mg/kg/day Capsules taken by mouth three times per day

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Overall Number of Participants Analyzed 5 5 6 10 10 11 15 16
Measure Type: Count of Participants
Unit of Measure: Participants
Patients with any AEs after first dose
2
  40.0%
5
 100.0%
5
  83.3%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Patients with any AE related to study drug
2
  40.0%
2
  40.0%
5
  83.3%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Patients with any SAE
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   9.1%
0
   0.0%
1
   6.3%
Patients with any AE leading to discontinuation from study
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Patients with any TEAE
0
   0.0%
0
   0.0%
0
   0.0%
9
  90.0%
8
  80.0%
10
  90.9%
15
 100.0%
16
 100.0%
Patients with any drug-related TEAE
0
   0.0%
0
   0.0%
0
   0.0%
5
  50.0%
7
  70.0%
1
   9.1%
8
  53.3%
11
  68.8%
Patients with any TEAE leading to study drug discontinuation
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   6.3%
Patients with drug-related TEAEs leading to study drug discontinuation
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Time Frame Upto 152 weeks
Adverse Event Reporting Description Adverse events were collected from the time of signing the informed consent through study completion. A TEAE was an adverse event that started during or after the first dose of investigational product during Part B through the date of Week 12 clinic dose. At each level of summation (overall, system organ class, preferred term), patients reporting more than 1 AE were counted only once for the total incidence.
 
Arm/Group Title Part A - CAT-1004 33 mg/kg/Day Part A - CAT-1004 67 mg/kg/Day Part A - CAT-1004 100 mg/kg/Day Part B - CAT-1004 67 mg/kg/Day Part B - CAT-1004 100 mg/kg/Day Part B - Overall Placebo Part C - CAT-1004 67 mg/kg/Day Part C - CAT-1004 100 mg/kg/Day
Hide Arm/Group Description

Edasalonexent 33 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

Placebo 67 mg/kg/day Capsules taken by mouth two times per day

Placebo 100 mg/kg/day Capsules taken by mouth three times per day

Edasalonexent 67 mg/kg/day. Capsules taken by mouth two times per day

Edasalonexent

Edasalonexent 100 mg/kg/day. Capsules taken by mouth three times per day

Edasalonexent

All-Cause Mortality
Part A - CAT-1004 33 mg/kg/Day Part A - CAT-1004 67 mg/kg/Day Part A - CAT-1004 100 mg/kg/Day Part B - CAT-1004 67 mg/kg/Day Part B - CAT-1004 100 mg/kg/Day Part B - Overall Placebo Part C - CAT-1004 67 mg/kg/Day Part C - CAT-1004 100 mg/kg/Day
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/5 (0.00%)   0/6 (0.00%)   0/6 (0.00%)   0/10 (0.00%)   0/10 (0.00%)   0/11 (0.00%)   0/15 (0.00%)   0/16 (0.00%) 
Hide Serious Adverse Events
Part A - CAT-1004 33 mg/kg/Day Part A - CAT-1004 67 mg/kg/Day Part A - CAT-1004 100 mg/kg/Day Part B - CAT-1004 67 mg/kg/Day Part B - CAT-1004 100 mg/kg/Day Part B - Overall Placebo Part C - CAT-1004 67 mg/kg/Day Part C - CAT-1004 100 mg/kg/Day
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/5 (0.00%)   0/6 (0.00%)   0/6 (0.00%)   0/10 (0.00%)   1/10 (10.00%)   0/11 (0.00%)   0/15 (0.00%)   0/16 (0.00%) 
Infections and infestations                 
Cellulitis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Subcutaneous abscess   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Part A - CAT-1004 33 mg/kg/Day Part A - CAT-1004 67 mg/kg/Day Part A - CAT-1004 100 mg/kg/Day Part B - CAT-1004 67 mg/kg/Day Part B - CAT-1004 100 mg/kg/Day Part B - Overall Placebo Part C - CAT-1004 67 mg/kg/Day Part C - CAT-1004 100 mg/kg/Day
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/5 (40.00%)   5/6 (83.33%)   5/6 (83.33%)   9/10 (90.00%)   8/10 (80.00%)   9/11 (81.82%)   15/15 (100.00%)   16/16 (100.00%) 
Blood and lymphatic system disorders                 
Anaemia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Lymphocytosis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Cardiac disorders                 
Left ventricular hypertrophy   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Congenital, familial and genetic disorders                 
Pectus excavatum   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Ear and labyrinth disorders                 
Motion sickness   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Tinnitus   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Eye disorders                 
Dry eye   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Excessive eye blinking   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Eyelid rash   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Ocular hyperaemia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Gastrointestinal disorders                 
Diarrhoea   0/5 (0.00%)  0/6 (0.00%)  4/6 (66.67%)  4/10 (40.00%)  5/10 (50.00%)  1/11 (9.09%)  7/15 (46.67%)  10/16 (62.50%) 
Faeces soft   1/5 (20.00%)  1/6 (16.67%)  1/6 (16.67%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Abdominal pain upper   1/5 (20.00%)  0/6 (0.00%)  1/6 (16.67%)  2/10 (20.00%)  2/10 (20.00%)  0/11 (0.00%)  3/15 (20.00%)  4/16 (25.00%) 
Vomiting   0/5 (0.00%)  1/6 (16.67%)  1/6 (16.67%)  1/10 (10.00%)  3/10 (30.00%)  1/11 (9.09%)  7/15 (46.67%)  7/16 (43.75%) 
Constipation   0/5 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  1/15 (6.67%)  0/16 (0.00%) 
Faecal incontinence   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  2/10 (20.00%)  0/10 (0.00%)  0/11 (0.00%)  2/15 (13.33%)  0/16 (0.00%) 
Nausea   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  2/10 (20.00%)  0/11 (0.00%)  3/15 (20.00%)  3/16 (18.75%) 
Abdominal pain   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  3/15 (20.00%)  1/16 (6.25%) 
Abdominal discomfort   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  2/16 (12.50%) 
Faeces discoloured   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Gastric disorder   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Lip swelling   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  1/16 (6.25%) 
Abdominal pain lower   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Dysphagia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Flatulence   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Loose tooth   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Tooth impacted   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
General disorders                 
Pyrexia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  4/11 (36.36%)  5/15 (33.33%)  8/16 (50.00%) 
Pain   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  1/10 (10.00%)  0/11 (0.00%)  1/15 (6.67%)  2/16 (12.50%) 
Influenza like illness   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  3/15 (20.00%)  1/16 (6.25%) 
Fatigue   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Gait disturbance   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Thirst   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Abasia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Catheter site pain   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Device occlusion   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Peripheral swelling   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Infections and infestations                 
Nasopharyngitis   0/5 (0.00%)  1/6 (16.67%)  1/6 (16.67%)  2/10 (20.00%)  0/10 (0.00%)  1/11 (9.09%)  6/15 (40.00%)  4/16 (25.00%) 
Ear infection   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  2/10 (20.00%)  0/11 (0.00%)  4/15 (26.67%)  5/16 (31.25%) 
Viral rash   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  2/11 (18.18%)  0/15 (0.00%)  0/16 (0.00%) 
Gastroenteritis viral   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  1/15 (6.67%)  6/16 (37.50%) 
Pharyngitis streptococcal   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  1/15 (6.67%)  4/16 (25.00%) 
Influenza   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  3/16 (18.75%) 
Sinusitis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  2/15 (13.33%)  1/16 (6.25%) 
Conjunctivitis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Upper respiratory tract infection   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  2/15 (13.33%)  0/16 (0.00%) 
Atypical pneumonia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  1/16 (6.25%) 
Body tinea   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Cellulitis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  1/16 (6.25%) 
Fungal skin infection   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  0/16 (0.00%) 
Gastroenteritis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  1/16 (6.25%) 
Subcutaneous abscess   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  0/16 (0.00%) 
Viral infection   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Clostridium difficile infection   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Impetigo   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Lower respiratory tract infection   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Oral herpes   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Pneumonia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Pneumonia bacterial   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Rhinitis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Scarlet fever   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Viral upper respiratory tract infection   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Injury, poisoning and procedural complications                 
Fall   0/5 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  4/10 (40.00%)  2/10 (20.00%)  4/11 (36.36%)  12/15 (80.00%)  12/16 (75.00%) 
Skin abrasion   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  2/10 (20.00%)  1/10 (10.00%)  0/11 (0.00%)  5/15 (33.33%)  3/16 (18.75%) 
Laceration   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  1/11 (9.09%)  2/15 (13.33%)  2/16 (12.50%) 
Contusion   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  3/15 (20.00%)  4/16 (25.00%) 
Head injury   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  3/15 (20.00%)  2/16 (12.50%) 
Ligament sprain   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  3/15 (20.00%)  2/16 (12.50%) 
Arthropod bite   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  2/16 (12.50%) 
Arthropod sting   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Muscle strain   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Joint injury   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Foot fracture   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Humerus fracture   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Lip injury   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Mouth injury   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Post-traumatic pain   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Road traffic accident   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Scratch   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Thermal burn   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Tooth fracture   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Tooth injury   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Traumatic haematoma   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Investigations                 
Coagulation time prolonged   0/5 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Vitamin D decreased   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  2/16 (12.50%) 
Blood urine present   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Body temperature increased   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Cardiac murmur   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Influenza B virus test positive   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Protein urine present   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Serum ferritin decreased   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Metabolism and nutrition disorders                 
Increased appetite   0/5 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Decreased appetite   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  2/10 (20.00%)  0/11 (0.00%)  2/15 (13.33%)  4/16 (25.00%) 
Metabolic acidosis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Fluid retention   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Weight gain poor   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Musculoskeletal and connective tissue disorders                 
Musculoskeletal pain   0/5 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Muscle spasms   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  1/10 (10.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Pain in extremity   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  1/11 (9.09%)  3/15 (20.00%)  1/16 (6.25%) 
Arthralgia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Back pain   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Foot deformity   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Joint contracture   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Joint range of motion decreased   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Joint swelling   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Muscle contracture   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Muscle fatigue   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Tendinous contracture   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Weight bearing difficulty   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Nervous system disorders                 
Headache   0/5 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  3/16 (18.75%) 
Dizziness   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Psychiatric disorders                 
Anxiety   0/5 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  2/15 (13.33%)  0/16 (0.00%) 
Abnormal behaviour   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Aggression   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  1/15 (6.67%)  1/16 (6.25%) 
Attention deficit/hyperactivity disorder   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  1/16 (6.25%) 
Emotional distress   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Impulsive behaviour   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  0/16 (0.00%) 
Insomnia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  0/16 (0.00%) 
Nightmare   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Stubbornness   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Negativism   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Sleep terror   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Tic   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Renal and urinary disorders                 
Enuresis   0/5 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Pollakiuria   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  0/16 (0.00%) 
Nocturia   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Respiratory, thoracic and mediastinal disorders                 
Rhinorrhoea   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  2/10 (20.00%)  2/10 (20.00%)  1/11 (9.09%)  5/15 (33.33%)  5/16 (31.25%) 
Cough   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  1/10 (10.00%)  2/11 (18.18%)  4/15 (26.67%)  9/16 (56.25%) 
Asthma   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Productive cough   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Respiratory disorder   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  1/11 (9.09%)  0/15 (0.00%)  0/16 (0.00%) 
Epistaxis   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  2/15 (13.33%)  3/16 (18.75%) 
Oropharyngeal pain   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  2/16 (12.50%) 
Adenoidal hypertrophy   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Choking   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Laryngospasm   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Nasal congestion   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Sleep apnoea syndrome   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Rhinitis allergic   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Snoring   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Throat irritation   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Tonsillar hypertrophy   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Skin and subcutaneous tissue disorders                 
Dermatitis contact   1/5 (20.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Hyperhidrosis   0/5 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Swelling face   0/5 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  0/16 (0.00%) 
Rash   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  1/10 (10.00%)  1/11 (9.09%)  1/15 (6.67%)  2/16 (12.50%) 
Erythema   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  2/16 (12.50%) 
Skin reaction   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/10 (10.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Papule   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Pruritus   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  0/15 (0.00%)  1/16 (6.25%) 
Rash generalised   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Skin exfoliation   0/5 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  0/10 (0.00%)  0/10 (0.00%)  0/11 (0.00%)  1/15 (6.67%)  0/16 (0.00%) 
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Andrew Nichols, PhD - Chief Scientific Officer
Organization: Astria Therapeutics, Inc
Phone: 617-349-1971
EMail: anichols@astriatx.com
Layout table for additonal information
Responsible Party: Catabasis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02439216    
Other Study ID Numbers: CAT-1004-201
First Submitted: April 29, 2015
First Posted: May 8, 2015
Results First Submitted: January 10, 2022
Results First Posted: September 23, 2022
Last Update Posted: September 23, 2022