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| ID | Type | Description | Link |
|---|---|---|---|
| 1R01FD004103-01 | U.S. FDA Grant/Contract | View source |
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| Name | Class |
|---|---|
| Food and Drug Administration (FDA) | FED |
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A multi-center, placebo controlled, double-blind trial comparing the efficacy and safety of 18 months of treatment with deferiprone versus placebo in patients with PKAN.
This investigator-initiated trial was funded by the European Commission's Seventh Framework Programme (FP7/2007-2013, HEALTH-F2-2011, grant agreement No. 277984) to the TIRCON consortium (Treat Iron-Related Childhood-Onset Neurodegeneration) and by the FDA Office of Orphan Products Development (OOPD) (Dr. Elliott Vichinsky).
This is a multi-center, double-blind, randomized, placebo-controlled, 18-month study in patients with PKAN aged 4 years and older. Participants are randomized in a 2:1 ratio to receive either deferiprone oral solution or placebo, twice a day for 18 months. Efficacy assessments, an MRI scan to measure iron levels in the globus pallidus, pharmacokinetic evaluations, and safety assessments are conducted at specified time points. Following completion of the trial, eligible patients are invited to enroll in an 18-month extension study, TIRCON2012V1-EXT, in which all participants receive deferiprone.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Deferiprone | Experimental | Deferiprone 80 mg/mL oral solution |
|
| Placebo | Placebo Comparator | Matching placebo solution |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deferiprone oral solution | Drug | Deferiprone 80 mg/mL oral solution will be administered twice daily (b.i.d.) for 18 months. An initial dose of 5 mg/kg b.i.d. will be administered for 6 weeks. The dose will then be escalated to 10 mg/kg b.i.d. and finally to 15 mg/kg b.i.d. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Score on Barry-Albright Dystonia Scale | 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. | Baseline to 18 Months |
| Score on Patient Global Impression of Improvement at End of Study | 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. | Month 18 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Score on Unified Parkinson's Disease Rating Scale | 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). |
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Main Inclusion Criteria:
Main Exclusion Criteria:
Other protocol inclusion or exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Fernando Tricta, MD | ApoPharma Inc. | Study Chair |
| Thomas Klopstock, MD | Friedrich-Baur-Institute, Department of Neurology, University of Munich Ziemssenstr | Principal Investigator |
| Elliott Vichinsky, MD | Children's Hospital & Research Center at Oakland Hematology/ Oncology, Pediatric Rehabilitation | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital & Research Center at Oakland | Oakland | California | 94609 | United States | ||
| Klinikum der Universität München |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31202468 | Derived | Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, Buchner B, Steele HE, Horvath R, Chinnery PF, Basu A, Kupper C, Neuhofer C, Kalman B, Dusek P, Yapici Z, Wilson I, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick SJ, Spino M, Blamire AM, Hogarth P, Vichinsky E. Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study. Lancet Neurol. 2019 Jul;18(7):631-642. doi: 10.1016/S1474-4422(19)30142-5. |
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Patients were recruited at 4 study sites in Germany, Italy, the U.K., and the U.S.
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| ID | Title | Description |
|---|---|---|
| FG000 | Deferiprone | 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. |
| FG001 | Placebo | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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Placebo solution matched deferiprone oral solution in appearance, taste, and packaging
|
| Placebo | Drug | A deferiprone matching placebo oral solution will be given twice daily for 18 months. |
|
| Baseline to 18 Months |
| Change in Score on Functional Independence Measure | 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). | Baseline to 18 Months |
| Change in Score on WeeFIM | 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). | Baseline to 18 Months |
| Change in Score on Pediatric Quality of Life | 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). | Baseline to 18 Months |
| Change in Score on Pittsburgh Sleep Quality Index | 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). | Baseline to 18 Months |
| Change in Level of Brain Iron | 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. | Baseline to 18 Months |
| Munich |
| 80336 |
| Germany |
| Foundation Neurological Institute C. Besta | Milan | 20133 | Italy |
| Newcastle University Institute of Human Genetics | Newcastle upon Tyne | NE1 3BZ | United Kingdom |
| Exposed |
|
| Provided Post-baseline Efficacy Data |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Deferiprone | Deferiprone 80 mg/mL oral solution, 15 mg/kg b.i.d. for 18 months |
| BG001 | Placebo | Matching volume of placebo solution, twice daily for 18 months |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Score on Barry-Albright Dystonia Scale | 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. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline to 18 Months |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Primary | Score on Patient Global Impression of Improvement at End of Study | 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. | Posted | Least Squares Mean | Standard Error | score on a scale | Month 18 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Score on Unified Parkinson's Disease Rating Scale | 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). | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline to 18 Months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Score on Functional Independence Measure | 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). | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline to 18 Months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Score on WeeFIM | 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). | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline to 18 Months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Score on Pediatric Quality of Life | 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). | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline to 18 Months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Score on Pittsburgh Sleep Quality Index | 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). | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline to 18 Months |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Level of Brain Iron | 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. | Posted | Least Squares Mean | Standard Error | Hz | Baseline to 18 Months |
|
|
Adverse events were collected from baseline until 30 days after the last dose of study medication.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Deferiprone | Study participants who received at least one dose of deferiprone in the trial | 0 | 58 | 18 | 58 | 57 | 58 |
| EG001 | Placebo | Study participants who received at least one dose of placebo in the trial | 0 | 30 | 10 | 30 | 30 | 30 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Intestinal dilatation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Volvulus | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Obstruction | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bacterial disease carrier | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Device related infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Chemical eye injury | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Postoperative ileus | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Toxicity to various agents | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Unintentional medical device removal | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Medical observation | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dystonia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypotonia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oromandibular dystonia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Device malfunction | Product Issues | MedDRA (19.1) | Systematic Assessment |
| |
| Staring | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Urinary bladder rupture | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Colectomy | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Gastrointestinal tube insertion | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Intestinal anastomosis | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Intrathecal pump insertion | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Laparotomy | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Medical device battery replacement | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Nasal septal operation | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Tracheostomy | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Tracheostomy tube removal | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Wound treatment | Surgical and medical procedures | MedDRA (19.1) | Systematic Assessment |
| |
| Thrombosis | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Leukocytosis | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abasia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Condition aggravated | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Blood iron decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Body temperature increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Serum ferritin decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Aphasia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Drooling | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dystonia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Freezing phenomenon | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Caroline Fradette | ApoPharma Inc. | 416-401-7543 | cfradett@apopharma.com |
| ID | Term |
|---|---|
| D006211 | Pantothenate Kinase-Associated Neurodegeneration |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D019150 | Neuroaxonal Dystrophies |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| D000077543 | Deferiprone |
| D007531 | Isoflurophate |
| D020084 | Long Interspersed Nucleotide Elements |
| ID | Term |
|---|---|
| D011728 | Pyridones |
| D011725 | Pyridines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D063066 | Organofluorophosphonates |
| D063065 | Organophosphonates |
| D009943 | Organophosphorus Compounds |
| D009930 | Organic Chemicals |
| D018626 | Retroelements |
| D020071 | Interspersed Repetitive Sequences |
| D012091 | Repetitive Sequences, Nucleic Acid |
| D001483 | Base Sequence |
| D015394 | Molecular Structure |
| D001669 | Biochemical Phenomena |
| D055598 | Chemical Phenomena |
| D040342 | Genetic Structures |
| D055614 | Genetic Phenomena |
| D040481 | Genome Components |
| D016678 | Genome |
Not provided
Not provided
| Male |
|
| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
|
| Unknown or Not Reported |
|
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|
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|
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