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Trial record 4 of 6 for:    Neurodegeneration with Brain Iron Accumulation (NBIA)

Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)

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: NCT01741532
Recruitment Status : Completed
First Posted : December 5, 2012
Results First Posted : May 23, 2019
Last Update Posted : July 3, 2019
Sponsor:
Collaborator:
Food and Drug Administration (FDA)
Information provided by (Responsible Party):
ApoPharma

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Pantothenate Kinase-Associated Neurodegeneration
Interventions Drug: Deferiprone oral solution
Drug: Placebo
Enrollment 89
Recruitment Details Patients were recruited at 4 study sites in Germany, Italy, the U.K., and the U.S.
Pre-assignment Details  
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description Patients randomized to this arm received deferiprone oral solution 80 mg/mL at a dosage of up to 15 mg/kg twice a day for up 18 months. Patients randomized to this arm received matching placebo solution twice a day, for up to 18 months, at a volume corresponding to that taken by patients who were receiving active product.
Period Title: Overall Study
Started 59 30
Exposed 58 30
Provided Post-baseline Efficacy Data 58 28
Completed 49 27
Not Completed 10 3
Reason Not Completed
Adverse Event             4             0
Withdrawal by Subject             1             1
Protocol Violation             0             1
Worsening of the disease             3             1
Sponsor decision             1             0
Medical event             1             0
Arm/Group Title Deferiprone Placebo Total
Hide Arm/Group Description Deferiprone 80 mg/mL oral solution, 15 mg/kg b.i.d. for 18 months Matching volume of placebo solution, twice daily for 18 months Total of all reporting groups
Overall Number of Baseline Participants 59 30 89
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 59 participants 30 participants 89 participants
20.8  (10.7) 19.2  (12.5) 20.2  (11.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 59 participants 30 participants 89 participants
Female
25
  42.4%
17
  56.7%
42
  47.2%
Male
34
  57.6%
13
  43.3%
47
  52.8%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 59 participants 30 participants 89 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
6
  10.2%
1
   3.3%
7
   7.9%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
   3.4%
0
   0.0%
2
   2.2%
White
51
  86.4%
28
  93.3%
79
  88.8%
More than one race
0
   0.0%
1
   3.3%
1
   1.1%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Change in Score on Barry-Albright Dystonia Scale
Hide Description The Barry-Albright Dystonia (BAD) scale rates severity of dystonia (sustained muscle contractions causing twisting and repetitive movements or abnormal postures) in 8 body regions. The individual scores are summed to provide a total score ranging from 0 to 32, with higher scores indicating greater severity. The co-primary endpoint in this study was the change from baseline to Month 18 in BAD total score.
Time Frame Baseline to 18 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description:
Patients randomized to this arm received deferiprone oral solution 80 mg/mL at a dosage of up to 15 mg/kg twice a day for up to 18 months.
Patients randomized to this arm received matching placebo solution twice a day, for up to 18 months, at a volume corresponding to what was given for the active product.
Overall Number of Participants Analyzed 58 28
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
2.48  (0.63) 3.99  (0.82)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0761
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
2.Primary Outcome
Title Score on Patient Global Impression of Improvement at End of Study
Hide Description The Patient Global Impression of Improvement (PGI-I) is a global index that assesses the response of a condition to a therapy by asking patients to rate their current state relative to their state at baseline. It consists of a 7-point rating scale, where 1=very much improved, 2= much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.
Time Frame Month 18
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description:
Patients randomized to this arm received deferiprone oral solution 80 mg/mL at a dosage of up to 15 mg/kg twice a day for up 18 months.
Patients randomized to this arm received matching placebo solution twice a day, for up to 18 months, at a volume corresponding to that taken by patients who were receiving active product.
Overall Number of Participants Analyzed 58 28
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
4.55  (0.30) 4.66  (0.38)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7279
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
3.Secondary Outcome
Title Change in Score on Unified Parkinson’s Disease Rating Scale
Hide Description The Unified Parkinson's Disease Rating Scale (UPDRS) is the major rating scale used to assess severity of symptoms of Parkinson's disease, some of which are similar to those of PKAN. The UPDRS subscales used in this study were Part I: Mentation, Behavior and Mood, scored from 0 (best) to 16 (worst); Part II: Activities of Daily Living, scored from 0 (best) to 52 (worst); Part III: Motor Examination, scored from 0 (best) to 108 (worst); and Part VI: Schwab and England Activities of Daily Living Scale, scored from 0% (worst) to 100% (best).
Time Frame Baseline to 18 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description:
Patients randomized to this arm received deferiprone oral solution 80 mg/mL at a dosage of up to 15 mg/kg twice a day for up 18 months.
Patients randomized to this arm received matching placebo solution twice a day, for up to 18 months, at a volume corresponding to that taken by patients who were receiving active product.
Overall Number of Participants Analyzed 58 28
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
UPDRS Part I -0.25  (0.43) -0.07  (0.55)
UPDRS Part II 1.09  (1.19) 2.36  (1.52)
UPDRS Part III 5.38  (2.20) 2.06  (2.79)
UPDRS Part VI -2.17  (2.94) -7.66  (3.85)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments Comparison of treatment groups on change in score on UPDRS Part I
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7228
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments Comparison of treatment groups on change in score on UPDRS Part II
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3677
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments Comparison of treatment groups on change in score on UPDRS Part III
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2182
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments Comparison of treatment groups on change in score on UPDRS Part VI
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1749
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
4.Secondary Outcome
Title Change in Score on Functional Independence Measure
Hide Description The Functional Independence Measure (FIM) scale is used to assess physical and cognitive disability in three areas of daily living: self-care, mobility, and cognition. Within each area, items are scored according to the level of assistance required to perform that activity of daily living. A score of 1-2 indicates that the patient is completely dependent on a helper to perform the task, a score of 3-5 indicates that the patient is moderately dependent, and a score of 6-7 indicates that no help is required. The individual scores are summed to provide a global score from 18 (worst) to 126 (best).
Time Frame Baseline to 18 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description:
Patients randomized to this arm received deferiprone oral solution 80 mg/mL at a dosage of up to 15 mg/kg twice a day for up 18 months.
Patients randomized to this arm received matching placebo solution twice a day, for up to 18 months, at a volume corresponding to that taken by patients who were receiving active product.
Overall Number of Participants Analyzed 58 28
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
5.40  (2.36) 0.69  (3.34)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1524
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
5.Secondary Outcome
Title Change in Score on WeeFIM
Hide Description The WeeFIM is the pediatric version of the Functional Independence Measure scale, and is used to assess physical and cognitive disability in three areas of daily living: self-care, mobility, and cognition. Within each area, items are scored according to the level of assistance required to perform that activity of daily living. A score of 1-2 indicates that the patient is completely dependent on a helper to perform the task, a score of 3-5 indicates that the patient is moderately dependent, and a score of 6-7 indicates that no help is required. The individual scores are summed to provide a global score from 18 (worst) to 126 (best).
Time Frame Baseline to 18 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description:
Patients randomized to this arm received deferiprone oral solution 80 mg/mL at a dosage of up to 15 mg/kg twice a day for up 18 months.
Patients randomized to this arm received matching placebo solution twice a day, for up to 18 months, at a volume corresponding to that taken by patients who were receiving active product.
Overall Number of Participants Analyzed 58 28
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
4.91  (5.30) -2.40  (5.42)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2026
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
6.Secondary Outcome
Title Change in Score on Pediatric Quality of Life
Hide Description The Pediatric Quality of Life (PedsQL) questionnaire is used to measure functional health and well-being from the patient's point of view. Separate versions of the questionnaire are available for children, young adults aged 18-25 years, and adults older than 25 years. Patients are asked to indicate how they have felt over the past month, and the scores of the 23 questions are used to generate an overall score that ranges from 0 (worst) to 100 (best).
Time Frame Baseline to 18 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description:
Patients randomized to this arm received deferiprone oral solution 80 mg/mL at a dosage of up to 15 mg/kg twice a day for up 18 months.
Patients randomized to this arm received matching placebo solution twice a day, for up to 18 months, at a volume corresponding to that taken by patients who were receiving active product.
Overall Number of Participants Analyzed 58 28
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
Patient self-report, total score 1.21  (3.68) 1.34  (4.49)
Parent proxy-report, total score -4.90  (3.99) -2.37  (4.90)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments Patient self-report, total score
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9759
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments Parent proxy-report, total score
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5781
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
7.Secondary Outcome
Title Change in Score on Pittsburgh Sleep Quality Index
Hide Description The Pittsburgh Sleep Quality Index (PSQI) is a self-rated questionnaire that assesses sleep quality and disturbances over a 1-month time interval. A total of 19 individual items are used to generate 7 "component" scores: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction, and a score is generated that ranges from 0 (best) to 21 (worst).
Time Frame Baseline to 18 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description:
Patients randomized to this arm received deferiprone oral solution 80 mg/mL at a dosage of up to 15 mg/kg twice a day for up 18 months.
Patients randomized to this arm received matching placebo solution twice a day, for up to 18 months, at a volume corresponding to that taken by patients who were receiving active product.
Overall Number of Participants Analyzed 58 28
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
0.48  (0.61) 0.14  (0.80)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6323
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
8.Secondary Outcome
Title Change in Level of Brain Iron
Hide Description Neurodegeneration in patients with PKAN is associated with localized brain iron accumulation, with the highest amount of accumulation seen in the globus pallidus, one of the main areas for motor control. MRI R2* scans of this region were performed at baseline and Month 18 in a subset of patients who did not have a deep brain stimulation (DBS) device implanted, and for whom the use of anesthesia, if required, was deemed acceptable by the investigator.
Time Frame Baseline to 18 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description:
The subset of deferiprone recipients who underwent MRI R2* imaging
The subset of placebo recipients who underwent MRI R2* imaging
Overall Number of Participants Analyzed 24 16
Least Squares Mean (Standard Error)
Unit of Measure: Hz
-36.1  (3.11) -0.50  (3.97)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Deferiprone, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0000
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Time Frame Adverse events were collected from baseline until 30 days after the last dose of study medication.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Deferiprone Placebo
Hide Arm/Group Description Study participants who received at least one dose of deferiprone in the trial Study participants who received at least one dose of placebo in the trial
All-Cause Mortality
Deferiprone Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/58 (0.00%)      0/30 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Deferiprone Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   18/58 (31.03%)      10/30 (33.33%)    
Blood and lymphatic system disorders     
Neutropenia  1  5/58 (8.62%)  7 2/30 (6.67%)  2
Gastrointestinal disorders     
Intestinal dilatation  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Intestinal obstruction  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Nausea  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Volvulus  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Vomiting  1  1/58 (1.72%)  1 1/30 (3.33%)  1
General disorders     
Condition aggravated  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Obstruction  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Pyrexia  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Infections and infestations     
Bacterial disease carrier  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Bronchitis  1  2/58 (3.45%)  2 0/30 (0.00%)  0
Cytomegalovirus infection  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Device related infection  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Pneumonia  1  2/58 (3.45%)  2 0/30 (0.00%)  0
Pneumonia bacterial  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Urinary tract infection  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Wound infection  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Injury, poisoning and procedural complications     
Chemical eye injury  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Postoperative ileus  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Toxicity to various agents  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Unintentional medical device removal  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Investigations     
Medical observation  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Transaminases increased  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Metabolism and nutrition disorders     
Dehydration  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Nervous system disorders     
Dystonia  1  3/58 (5.17%)  3 2/30 (6.67%)  3
Hypotonia  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Lethargy  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Oromandibular dystonia  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Syncope  1  1/58 (1.72%)  1 1/30 (3.33%)  1
Product Issues     
Device malfunction  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Psychiatric disorders     
Staring  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Renal and urinary disorders     
Urinary bladder rupture  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Choking  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Cough  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Pneumonia aspiration  1  1/58 (1.72%)  1 1/30 (3.33%)  1
Respiratory disorder  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Respiratory failure  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Surgical and medical procedures     
Colectomy  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Gastrointestinal tube insertion  1  1/58 (1.72%)  1 1/30 (3.33%)  1
Intestinal anastomosis  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Intrathecal pump insertion  1  1/58 (1.72%)  2 0/30 (0.00%)  0
Laparotomy  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Medical device battery replacement  1  2/58 (3.45%)  2 1/30 (3.33%)  1
Nasal septal operation  1  0/58 (0.00%)  0 1/30 (3.33%)  1
Tracheostomy  1  1/58 (1.72%)  3 0/30 (0.00%)  0
Tracheostomy tube removal  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Wound treatment  1  1/58 (1.72%)  1 0/30 (0.00%)  0
Vascular disorders     
Thrombosis  1  1/58 (1.72%)  1 0/30 (0.00%)  0
1
Term from vocabulary, MedDRA (19.1)
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%
Deferiprone Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   57/58 (98.28%)      30/30 (100.00%)    
Blood and lymphatic system disorders     
Anaemia  1  12/58 (20.69%)  23 0/30 (0.00%)  0
Leukocytosis  1  2/58 (3.45%)  3 2/30 (6.67%)  2
Ear and labyrinth disorders     
Ear pain  1  1/58 (1.72%)  1 3/30 (10.00%)  3
Gastrointestinal disorders     
Abdominal pain upper  1  4/58 (6.90%)  6 5/30 (16.67%)  7
Constipation  1  2/58 (3.45%)  3 4/30 (13.33%)  4
Diarrhoea  1  4/58 (6.90%)  7 3/30 (10.00%)  8
Influenza  1  3/58 (5.17%)  4 0/30 (0.00%)  0
Toothache  1  2/58 (3.45%)  2 2/30 (6.67%)  3
Vomiting  1  9/58 (15.52%)  17 8/30 (26.67%)  19
General disorders     
Abasia  1  2/58 (3.45%)  2 2/30 (6.67%)  2
Condition aggravated  1  10/58 (17.24%)  12 9/30 (30.00%)  10
Pain  1  4/58 (6.90%)  4 0/30 (0.00%)  0
Pyrexia  1  16/58 (27.59%)  34 13/30 (43.33%)  28
Infections and infestations     
Bronchitis  1  7/58 (12.07%)  15 2/30 (6.67%)  2
Ear infection  1  0/58 (0.00%)  0 2/30 (6.67%)  2
Gastrointestinal infection  1  3/58 (5.17%)  3 3/30 (10.00%)  4
Localised infection  1  0/58 (0.00%)  0 2/30 (6.67%)  2
Nasopharyngitis  1  11/58 (18.97%)  20 6/30 (20.00%)  10
Rhinitis  1  4/58 (6.90%)  6 1/30 (3.33%)  1
Upper respiratory tract infection  1  8/58 (13.79%)  9 4/30 (13.33%)  5
Viral infection  1  4/58 (6.90%)  5 1/30 (3.33%)  1
Injury, poisoning and procedural complications     
Arthropod bite  1  1/58 (1.72%)  1 2/30 (6.67%)  4
Contusion  1  2/58 (3.45%)  2 2/30 (6.67%)  2
Fall  1  2/58 (3.45%)  3 2/30 (6.67%)  2
Laceration  1  6/58 (10.34%)  15 3/30 (10.00%)  3
Skin abrasion  1  0/58 (0.00%)  0 2/30 (6.67%)  4
Investigations     
Blood iron decreased  1  3/58 (5.17%)  3 2/30 (6.67%)  2
Body temperature increased  1  3/58 (5.17%)  3 0/30 (0.00%)  0
Neutrophil count decreased  1  10/58 (17.24%)  18 3/30 (10.00%)  20
Serum ferritin decreased  1  19/58 (32.76%)  23 5/30 (16.67%)  5
White blood cell count increased  1  0/58 (0.00%)  0 2/30 (6.67%)  2
Metabolism and nutrition disorders     
Iron deficiency  1  9/58 (15.52%)  9 3/30 (10.00%)  3
Musculoskeletal and connective tissue disorders     
Arthralgia  1  8/58 (13.79%)  8 1/30 (3.33%)  1
Back pain  1  3/58 (5.17%)  3 2/30 (6.67%)  8
Muscle spasms  1  3/58 (5.17%)  4 2/30 (6.67%)  2
Pain in extremity  1  10/58 (17.24%)  13 4/30 (13.33%)  7
Nervous system disorders     
Aphasia  1  3/58 (5.17%)  3 2/30 (6.67%)  2
Drooling  1  2/58 (3.45%)  2 2/30 (6.67%)  2
Dystonia  1  25/58 (43.10%)  49 14/30 (46.67%)  20
Freezing phenomenon  1  0/58 (0.00%)  0 3/30 (10.00%)  3
Headache  1  13/58 (22.41%)  43 9/30 (30.00%)  30
Migraine  1  3/58 (5.17%)  21 0/30 (0.00%)  0
Renal and urinary disorders     
Urinary incontinence  1  3/58 (5.17%)  4 2/30 (6.67%)  2
Reproductive system and breast disorders     
Dysmenorrhoea  1  0/58 (0.00%)  0 2/30 (6.67%)  8
Respiratory, thoracic and mediastinal disorders     
Cough  1  10/58 (17.24%)  12 5/30 (16.67%)  9
Nasal congestion  1  1/58 (1.72%)  1 2/30 (6.67%)  3
Oropharyngeal pain  1  9/58 (15.52%)  10 3/30 (10.00%)  3
Rhinorrhoea  1  4/58 (6.90%)  6 2/30 (6.67%)  2
Skin and subcutaneous tissue disorders     
Hyperhidrosis  1  3/58 (5.17%)  3 1/30 (3.33%)  1
Rash  1  4/58 (6.90%)  4 1/30 (3.33%)  1
1
Term from vocabulary, MedDRA (19.1)
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.
Sponsor retained title to and the right to publish all documentation, records, raw data, specimens or other work product generated in connection with the trial. Such publications shall not be made without the prior written consent of Sponsor. Neither Party will use the other Party's name in connection with any publication or promotion without the other Party's prior written consent. However, Sponsor has the right to publish appropriate information in order to satisfy regulatory requirements.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Caroline Fradette
Organization: ApoPharma Inc.
Phone: 416-401-7543
EMail: cfradett@apopharma.com
Layout table for additonal information
Responsible Party: ApoPharma
ClinicalTrials.gov Identifier: NCT01741532     History of Changes
Other Study ID Numbers: TIRCON2012V1
1R01FD004103-01 ( U.S. FDA Grant/Contract )
First Submitted: December 3, 2012
First Posted: December 5, 2012
Results First Submitted: May 1, 2019
Results First Posted: May 23, 2019
Last Update Posted: July 3, 2019