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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01741532 |
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Date of registration:
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03/12/2012 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
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Scientific title:
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A Randomized, Double-blind, Placebo-controlled Trial of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN) |
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Date of first enrolment:
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December 13, 2012 |
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Target sample size:
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89 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01741532 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 3
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Countries of recruitment
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Germany
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Italy
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Poland
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United Kingdom
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United States
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Contacts
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Name:
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Elliott Vichinsky, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's Hospital & Research Center at Oakland Hematology/ Oncology, Pediatric Rehabilitation |
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Name:
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Thomas Klopstock, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Friedrich-Baur-Institute, Department of Neurology, University of Munich Ziemssenstr |
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Name:
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Fernando Tricta, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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ApoPharma Inc. |
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Key inclusion & exclusion criteria
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Main Inclusion Criteria:
- Males or females 4 years of age and older at screening visit;
- Have PKAN, confirmed by genetic testing (supporting evidence required);
- Barry-Albright Dystonia (BAD) total score = 3 at the screening visit;
- Patients who have Deep Brain Stimulation (DBS) systems or baclofen pumps in place will
be eligible for the study, but they must have had a stable setting for at least two
months prior to the screening visit and stimulation parameters / pump settings must
remain stable for the duration of the trial:
Main Exclusion Criteria:
- Evidence of iron deficiency defined by Fe:TIBC ratio <15%, or serum ferritin <12
ng/mL;
- Treatment with deferiprone in the past 12 months;
- Previous failure of treatment with deferiprone, or previous discontinuation of
treatment with deferiprone due to adverse events;
- Conditions known to contraindicate the use of deferiprone (history of agranulocytosis
or recurrent episodes of neutropenia);
- A serious, unstable chronic illness not related to PKAN condition during the past 3
months before screening visit including but not limited to: hepatic, renal,
gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or
immunologic disease;
- Evidence of abnormal liver or renal function (serum liver enzyme level(s) > 3 times
upper limit of normal at screening) or abnormal creatinine levels at screening visit;
- Disorders associated with neutropenia (ANC < 1.5 x 10^9/L) or thrombocytopenia
(platelet count < 50 x 10^9/L) in the 12 months preceding the initiation of the study
medication. Exception: for patients whose neutropenia was attributed by the treating
physician to episodes of infection or to drugs associated with a decline in the
neutrophil count and in whom the ANC has fully recovered at the screening visit;
- History of malignancy;
Other protocol inclusion or exclusion criteria may apply.
Age minimum:
4 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pantothenate Kinase-Associated Neurodegeneration
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Intervention(s)
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Drug: Deferiprone oral solution
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Drug: Placebo
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Primary Outcome(s)
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Score on Patient Global Impression of Improvement at End of Study
[Time Frame: Month 18]
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Change in Score on Barry-Albright Dystonia Scale
[Time Frame: Baseline to 18 Months]
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Secondary Outcome(s)
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Change in Score on Pittsburgh Sleep Quality Index
[Time Frame: Baseline to 18 Months]
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Change in Score on WeeFIM
[Time Frame: Baseline to 18 Months]
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Change in Score on Functional Independence Measure
[Time Frame: Baseline to 18 Months]
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Change in Level of Brain Iron
[Time Frame: Baseline to 18 Months]
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Change in Score on Pediatric Quality of Life
[Time Frame: Baseline to 18 Months]
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Change in Score on Unified Parkinson's Disease Rating Scale
[Time Frame: Baseline to 18 Months]
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Secondary ID(s)
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1R01FD004103-01
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TIRCON2012V1
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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