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Trial record 9 of 33 for:    CYSTEAMINE

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

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: NCT01733316
Recruitment Status : Completed
First Posted : November 27, 2012
Results First Posted : May 24, 2017
Last Update Posted : August 13, 2018
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma USA, Inc.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cystinosis
Interventions Drug: RP103
Drug: Cystagon®
Enrollment 41
Recruitment Details  
Pre-assignment Details  
Arm/Group Title All Participants
Hide Arm/Group Description

Cystagon® Phase: From Screening and during Months 1, 2, 3 participants received their usual dose of Cystagon® every 6 hours (Q6H).

RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants received RP103 every 12 hours (Q12H).

Long Term Phase: On or after Month 7, for the remainder of study participants received RP103 Q12H.

Period Title: Cystagon® Phase
Started 41
Completed 41
Not Completed 0
Period Title: RP103 Phase
Started 41
Completed 40
Not Completed 1
Reason Not Completed
Non-compliance             1
Period Title: Long Term Extension Phase
Started 38 [1]
Completed 33
Not Completed 5
Reason Not Completed
Adverse Event             1
Consent Withdrawn by Subject             2
Sponsor Decision             2
[1]
Two participants did not enter the long term follow up period.
Arm/Group Title All Participants
Hide Arm/Group Description

Cystagon® Phase: From Screening and during Months 1, 2, 3 participants received their usual dose of Cystagon® Q6H.

RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants received RP103 Q12H.

Long Term Phase: On or after Month 7, for the remainder of study participants received RP103 Q12H.

Overall Number of Baseline Participants 41
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 41 participants
24.5  (11.56)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants
Female
21
  51.2%
Male
20
  48.8%
1.Primary Outcome
Title Average Difference Between Morning and Non-Morning Log White Blood Cell (WBC) Cystine Values
Hide Description The primary analysis of WBC cystine was performed using the natural log transformed WBC cystine level; the log transformation is a normalizing transformation. For each participant, the difference between the morning and corresponding non-morning log WBC cystine value (non-morning minus morning) at each monthly visit during the Cystagon® phase (Months 1, 2, and 3) was computed and these differences were averaged. The average difference between morning and non-morning log WBC cystine value was similarly computed for each participant during the RP103 phase (Months 5, 6, and 7). The primary analysis compared within-subject pairs (Cystagon® phase paired with RP103 phase) of non-morning minus morning average differences of log WBC cystine level.
Time Frame While taking Cystagon® (Months 1, 2, 3): within 15 minutes pre-morning (AM) and pre-non AM dose. During 3 months of RP103 (Months 5, 6, 7): 30 minutes post-AM and post-evening (PM) dose.
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Pharmacodynamic (PD) Analysis Set: All participants who received at least one treatment of Cystagon® and RP103 and who had at least one WBC cystine level recorded after each of Cystagon® treatment and RP103 treatment. Only participants with an average difference during both the Cystagon® and RP103 phases were included.
Arm/Group Title Cystagon® Phase RP103 Phase
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From Screening and during Months 1, 2, 3: participants received their usual dose of Cystagon® Q6H.
During Months 3.5, 4, 5, 6, 7: participants received RP103 Q12H.
Overall Number of Participants Analyzed 40 40
Mean (Standard Deviation)
Unit of Measure: log [nmol ½ cystine/mg protein]
-0.229  (0.5027) 0.080  (0.3939)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Cystagon® Phase, RP103 Phase
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0048
Comments Paired t-test testing the null hypothesis that the population average difference during the Cystagon® phase is equal to the population average difference during the RP103 phase.
Method Paired t-test, two-sided
Comments [Not Specified]
2.Secondary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Discontinuations Due to AEs
Hide Description AE: any untoward medical occurrence that does not necessarily have a causal relationship with study drug. SAE: any untoward medical occurrence that at any dose: results in death; is life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; is a congenital anomaly or birth defect; or is medically significant, and though not included in the above list, is an important medical event, according to the Investigator. Treatment-emergent adverse events (TEAEs) occurred after first dose of study drug. Clinically significant abnormalities in laboratory values (hematology, blood chemistry, urinalysis), electrocardiograms (ECGs), vital signs, and physical examinations were to be reported as adverse events and so are included in this summary of TEAEs
Time Frame From first dose of study drug to 7 days after last dose. Median duration of exposure was 91 days (range 82-108) for Cystagon® phase, 119 days (range 98-137) for the RP103 phase, and 861 days (range 30 - 1350) during the long-term RP-103 phase.
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Hide Analysis Population Description
Safety Analysis Set: all participants who received at least 1 dose of study drug (RP103). The row "At least 1 TEAE" includes both serious and non-serious AEs.
Arm/Group Title Cystagon® Phase RP103 Phase Long-Term Phase
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From Screening and during Months 1, 2, 3: participants received their usual dose of Cystagon® Q6H.

Observations from Day 1 Cystagon® dosing up to the first dose of RP103 were attributed to the Cystagon® Phase.

During Months 3.5, 4, 5, 6, 7: participants received RP103 Q12H.

Observations on or after the first dose of RP103 up to Month 7 or study termination visit (whichever occurred first) were attributed to the RP103 Phase.

From Month 7 and for the remainder of study: participants received RP103 Q12H.

Observations on or after the Month 7 visit through study termination were attributed to the Long-Term RP103 Phase.

Overall Number of Participants Analyzed 41 41 38
Measure Type: Count of Participants
Unit of Measure: Participants
At least 1 TEAE
31
  75.6%
38
  92.7%
32
  84.2%
At least 1 TEAE-related to study drug
4
   9.8%
20
  48.8%
18
  47.4%
At least 1 grade ≥ 3 TEAE
5
  12.2%
4
   9.8%
13
  34.2%
At least 1 serious TEAE
5
  12.2%
6
  14.6%
13
  34.2%
At least 1 TEAE leading to discontinuation
0
   0.0%
0
   0.0%
1
   2.6%
3.Secondary Outcome
Title Halitosis Substudy: Maximum Plasma Concentration (Cmax) for Plasma Cysteamine
Hide Description Participants who reported halitosis (“bad breath”) as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of dimethylsulfide (DMS) in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state pharmacokinetic (PK) samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered.
Time Frame While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose
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Hide Analysis Population Description
PK Analysis Set: All participants who received at least one dose of RP103 and had available PK data at given timepoint.
Arm/Group Title Halitosis Substudy Participants
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Cystagon® Phase: From Screening and during Months 1, 2, 3 participants received their usual dose of Cystagon® Q6H.

RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants received RP103 Q12H.

Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: mg/L
Cystagon® dosing period Number Analyzed 19 participants
3.5  (1.87)
RP103 dosing period Number Analyzed 20 participants
2.9  (1.65)
4.Secondary Outcome
Title Halitosis Substudy: Time to Cmax (Tmax) for Plasma Cysteamine
Hide Description Participants who reported halitosis (“bad breath”) as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered.
Time Frame While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
PK Analysis Set: All participants who received at least one dose of RP103 and had available PK data at given timepoint.
Arm/Group Title Halitosis Substudy Participants
Hide Arm/Group Description:

Cystagon® Phase: From Screening and during Months 1, 2, 3 participants received their usual dose of Cystagon® Q6H.

RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants received RP103 Q12H.

Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: hour
Cystagon® dosing period Number Analyzed 19 participants
1.2  (0.87)
RP103 dosing period Number Analyzed 20 participants
3.2  (1.30)
5.Secondary Outcome
Title Area Under the Plasma Concentration Time Curve From Time Point 0 Through the Last Measurable Point (AUC0-t) for Plasma Cysteamine
Hide Description Participants who reported halitosis (“bad breath”) as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered.
Time Frame While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose, 30 minutes post-dose, 1, 2, 4 and 6 hours post-dose. While taking RP103 (Month 5, 6, or 7): 30 minutes after morning dose and 1, 2, 3, 4, 6, 8, 10, 12 hours post-dose
Hide Outcome Measure Data
Hide Analysis Population Description
PK Analysis Set: All participants who received at least one dose of RP103 and had available PK data at given timepoint.
Arm/Group Title Halitosis Substudy Participants
Hide Arm/Group Description:

Cystagon® Phase: From Screening and during Months 1, 2, 3 participants received their usual dose of Cystagon® Q6H.

RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants received RP103 Q12H.

Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: hr*mg/L
Cystagon® dosing period Number Analyzed 19 participants
7.8  (5.18)
RP103 dosing period Number Analyzed 20 participants
9.4  (4.86)
6.Secondary Outcome
Title Halitosis Substudy: Expired Air DMS Concentrations
Hide Description Participants who reported halitosis (“bad breath”) as a side effect while receiving Cystagon® were asked to participate in a substudy to investigate the concentration of DMS in expired air after the administration of study medication. To assess halitosis during study medication treatment, the steady state PK samples of cysteamine and DMS were collected over a 6 hour period when Cystagon® was administered and over a 12 hour period when RP103 was administered.
Time Frame While taking Cystagon® (Month 1, 2 or 3): Within 15 minutes prior to morning dose 30 min post-dose, 2, 3, 4 and 6 hours post-dose. While taking RP103 (Month 4, 5, or 7): Within 15 min. prior to morning dose. 1, 2, 3, 4, 5, 6, 8, 10, 12 hours post dose
Hide Outcome Measure Data
Hide Analysis Population Description
PK Analysis Set: All participants who received at least one dose of RP103 and had available PK data at given timepoint.
Arm/Group Title Halitosis Substudy Participants
Hide Arm/Group Description:

Cystagon® Phase: From Screening and during Months 1, 2, 3 participants received their usual dose of Cystagon® Q6H.

RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants received RP103 Q12H.

Overall Number of Participants Analyzed 20
Mean (Standard Deviation)
Unit of Measure: nmol/L
Cystagon®, within 15 minutes prior to dose Number Analyzed 18 participants
4.7  (5.54)
Cystagon®, 30 minutes post dose Number Analyzed 18 participants
4.7  (4.75)
Cystagon®, 1 hour post morning dose Number Analyzed 18 participants
6.9  (6.75)
Cystagon®, 2 hours post morning dose Number Analyzed 18 participants
11.9  (11.26)
Cystagon®, 3 hours post morning dose Number Analyzed 18 participants
11.2  (13.32)
Cystagon®, 4 hours post morning dose Number Analyzed 18 participants
8.8  (12.05)
Cystagon®, 6 hours post morning dose Number Analyzed 18 participants
4.4  (3.77)
RP103, within 15 minutes prior to dose Number Analyzed 20 participants
4.6  (6.05)
RP103, 1 hour post morning dose Number Analyzed 20 participants
5.4  (7.71)
RP103, 2 hours post morning dose Number Analyzed 20 participants
5.1  (6.28)
RP103, 3 hours post morning dose Number Analyzed 20 participants
7.7  (8.53)
RP103, 4 hours post morning dose Number Analyzed 20 participants
8.6  (11.22)
RP103, 5 hours post morning dose Number Analyzed 20 participants
11.2  (15.86)
RP103, 6 hours post morning dose Number Analyzed 20 participants
8.5  (13.87)
RP103, 8 hours post morning dose Number Analyzed 20 participants
5.7  (10.76)
RP103, 10 hours post morning dose Number Analyzed 20 participants
5.2  (8.13)
RP103, 12 hours post morning dose Number Analyzed 20 participants
4.3  (5.32)
Time Frame From first dose of study drug to 7 days after last dose. Median duration of exposure was 91 days (range 82-108) for Cystagon® phase, 119 days (range 98-137) for the RP103 phase, and 861 days (range 30-1350) during the long term RP-103 phase.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cystagon® Phase RP103 Phase Long-Term Phase
Hide Arm/Group Description From Screening and during Months 1, 2, 3: participants received their usual dose of Cystagon® Q6H. During Months 3.5, 4, 5, 6, 7: participants received RP103 Q12H. From Month 7 and for the remainder of study: participants received RP103 Q12H.
All-Cause Mortality
Cystagon® Phase RP103 Phase Long-Term Phase
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/41 (0.00%)   0/41 (0.00%)   1/38 (2.63%) 
Show Serious Adverse Events Hide Serious Adverse Events
Cystagon® Phase RP103 Phase Long-Term Phase
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   5/41 (12.20%)   6/41 (14.63%)   13/38 (34.21%) 
Blood and lymphatic system disorders       
Anaemia  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Gastrointestinal disorders       
Abdominal pain  1  1/41 (2.44%)  0/41 (0.00%)  0/38 (0.00%) 
Diarrhoea  1  0/41 (0.00%)  1/41 (2.44%)  2/38 (5.26%) 
Nausea  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Vomiting  1  0/41 (0.00%)  0/41 (0.00%)  2/38 (5.26%) 
General disorders       
Chest pain  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Death  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Malaise  1  0/41 (0.00%)  0/41 (0.00%)  2/38 (5.26%) 
Fatigue  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Pyrexia  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Infections and infestations       
Bacteraemia  1  0/41 (0.00%)  1/41 (2.44%)  0/38 (0.00%) 
Cellulitis  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Diverticulitis  1  1/41 (2.44%)  0/41 (0.00%)  0/38 (0.00%) 
Gastroenteritis  1  0/41 (0.00%)  1/41 (2.44%)  2/38 (5.26%) 
Lung infection  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Pneumonia  1  1/41 (2.44%)  0/41 (0.00%)  1/38 (2.63%) 
Sepsis  1  1/41 (2.44%)  0/41 (0.00%)  1/38 (2.63%) 
Septic shock  1  1/41 (2.44%)  0/41 (0.00%)  0/38 (0.00%) 
Urinary tract infection  1  1/41 (2.44%)  1/41 (2.44%)  0/38 (0.00%) 
Gastroenteritis norovirus  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Gastroenteritis viral  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Graft infection  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Onychomycosis  1  1/41 (2.44%)  0/41 (0.00%)  0/38 (0.00%) 
Tonsillitis  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Injury, poisoning and procedural complications       
Arteriovenous fistula thrombosis  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Procedural hypotension  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Arteriovenous fistula aneurysm  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Arteriovenous fistula site complication  1  0/41 (0.00%)  1/41 (2.44%)  2/38 (5.26%) 
Arteriovenous fistula site haemorrhage  1  0/41 (0.00%)  1/41 (2.44%)  1/38 (2.63%) 
Metabolism and nutrition disorders       
Dehydration  1  0/41 (0.00%)  0/41 (0.00%)  4/38 (10.53%) 
Hyperkalemia  1  0/41 (0.00%)  1/41 (2.44%)  0/38 (0.00%) 
Hypocalcaemia  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Musculoskeletal and connective tissue disorders       
Scoliosis  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Squamous cell carcinoma  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Nervous system disorders       
Migraine with aura  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Cerebral haemorrhage  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Dizziness  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Headache  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Loss of consciousness  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Syncope  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Psychiatric disorders       
Depression  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Psychotic disorder  1  0/41 (0.00%)  1/41 (2.44%)  0/38 (0.00%) 
Renal and urinary disorders       
Renal failure acute  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Reproductive system and breast disorders       
Testicular pain  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Respiratory, thoracic and mediastinal disorders       
Acute respiratory failure  1  1/41 (2.44%)  0/41 (0.00%)  0/38 (0.00%) 
Epistaxis  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Pneumonitis  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Surgical and medical procedures       
Arteriovenous fistula operation  1  1/41 (2.44%)  0/41 (0.00%)  0/38 (0.00%) 
Ossiculoplasty  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Renal transplant  1  0/41 (0.00%)  0/41 (0.00%)  2/38 (5.26%) 
Vascular disorders       
Hypertension  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
Peripheral artery thrombosis  1  0/41 (0.00%)  0/41 (0.00%)  1/38 (2.63%) 
1
Term from vocabulary, MedDRA 15.1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cystagon® Phase RP103 Phase Long-Term Phase
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   28/41 (68.29%)   33/41 (80.49%)   32/38 (84.21%) 
Blood and lymphatic system disorders       
Anaemia  1  0/41 (0.00%)  1/41 (2.44%)  4/38 (10.53%) 
Eye disorders       
Conjunctivitis  1  0/41 (0.00%)  1/41 (2.44%)  3/38 (7.89%) 
Gastrointestinal disorders       
Abdominal pain  1  1/41 (2.44%)  3/41 (7.32%)  5/38 (13.16%) 
Abdominal pain upper  1  2/41 (4.88%)  6/41 (14.63%)  3/38 (7.89%) 
Constipation  1  0/41 (0.00%)  2/41 (4.88%)  3/38 (7.89%) 
Diarrhoea  1  4/41 (9.76%)  9/41 (21.95%)  7/38 (18.42%) 
Nausea  1  4/41 (9.76%)  15/41 (36.59%)  5/38 (13.16%) 
Vomiting  1  2/41 (4.88%)  11/41 (26.83%)  7/38 (18.42%) 
General disorders       
Influenza like illness  1  0/41 (0.00%)  0/41 (0.00%)  4/38 (10.53%) 
Malaise  1  3/41 (7.32%)  1/41 (2.44%)  1/38 (2.63%) 
Pyrexia  1  2/41 (4.88%)  2/41 (4.88%)  2/38 (5.26%) 
Infections and infestations       
Bronchitis  1  0/41 (0.00%)  1/41 (2.44%)  2/38 (5.26%) 
Candidiasis  1  0/41 (0.00%)  1/41 (2.44%)  2/38 (5.26%) 
Ear infection  1  0/41 (0.00%)  0/41 (0.00%)  3/38 (7.89%) 
Gastroenteritis  1  1/41 (2.44%)  4/41 (9.76%)  4/38 (10.53%) 
Influenza  1  0/41 (0.00%)  0/41 (0.00%)  4/38 (10.53%) 
Nasopharyngitis  1  8/41 (19.51%)  5/41 (12.20%)  6/38 (15.79%) 
Respiratory tract infection  1  1/41 (2.44%)  2/41 (4.88%)  2/38 (5.26%) 
Upper respiratory tract infection  1  3/41 (7.32%)  0/41 (0.00%)  6/38 (15.79%) 
Urinary tract infection  1  2/41 (4.88%)  3/41 (7.32%)  4/38 (10.53%) 
Viral infection  1  1/41 (2.44%)  2/41 (4.88%)  2/38 (5.26%) 
Viral upper respiratory tract infection  1  0/41 (0.00%)  0/41 (0.00%)  2/38 (5.26%) 
Investigations       
Blood creatinine increased  1  0/41 (0.00%)  1/41 (2.44%)  2/38 (5.26%) 
Hepatic enzyme increased  1  0/41 (0.00%)  0/41 (0.00%)  2/38 (5.26%) 
Weight decreased  1  1/41 (2.44%)  1/41 (2.44%)  3/38 (7.89%) 
Metabolism and nutrition disorders       
Dehydration  1  1/41 (2.44%)  0/41 (0.00%)  2/38 (5.26%) 
Musculoskeletal and connective tissue disorders       
Arthralgia  1  0/41 (0.00%)  0/41 (0.00%)  2/38 (5.26%) 
Knee deformity  1  0/41 (0.00%)  0/41 (0.00%)  2/38 (5.26%) 
Nervous system disorders       
Headache  1  8/41 (19.51%)  6/41 (14.63%)  7/38 (18.42%) 
Renal and urinary disorders       
Renal impairment  1  0/41 (0.00%)  1/41 (2.44%)  2/38 (5.26%) 
Reproductive system and breast disorders       
Dysmenorrhoea  1  0/41 (0.00%)  1/41 (2.44%)  2/38 (5.26%) 
Respiratory, thoracic and mediastinal disorders       
Cough  1  2/41 (4.88%)  1/41 (2.44%)  3/38 (7.89%) 
Skin and subcutaneous tissue disorders       
Skin lesion  1  0/41 (0.00%)  0/41 (0.00%)  2/38 (5.26%) 
1
Term from vocabulary, MedDRA 15.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
Horizon requests that any investigator/institution that plans on presenting/publishing results provide written notification of their request 60 days prior to their presentation/publication. Horizon requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if Horizon needs to secure patent or proprietary protection.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Evelyn Olson, Director
Organization: Horizon Pharma USA, Inc.
Phone: 224-383-3000
EMail: clinicaltrials@horizonpharma.com
Layout table for additonal information
Responsible Party: Horizon Pharma USA, Inc.
ClinicalTrials.gov Identifier: NCT01733316     History of Changes
Other Study ID Numbers: RP103-07
2012-002773-64 ( EudraCT Number )
First Submitted: November 16, 2012
First Posted: November 27, 2012
Results First Submitted: April 10, 2017
Results First Posted: May 24, 2017
Last Update Posted: August 13, 2018