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. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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12 December 2020 |
Main ID: |
NCT03041116 |
Date of registration:
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02/12/2016 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Patients
PKAN |
Scientific title:
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Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), A Phosphopantothenate Replacement Therapy, in Pantothenate Kinase-Associated Neurodegeneration (PKAN) Patients: A Randomized, Double Blind, Placebo Controlled Study With an Open Label Extension |
Date of first enrolment:
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July 17, 2017 |
Target sample size:
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82 |
Recruitment status: |
Active, not recruiting |
URL:
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https://clinicaltrials.gov/show/NCT03041116 |
Study type:
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Interventional |
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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Canada
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Czechia
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France
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Germany
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Italy
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Norway
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Poland
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Spain
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United States
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Contacts
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Name:
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Thomas Klopstock, MD |
Address:
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Telephone:
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Email:
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Affiliation:
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Klinikum der Universität München |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. The patient has a diagnosis of PKAN as indicated by confirmed mutations in the
pantothenate kinase 2 (PANK2) gene.
2. The patient is male or female aged 6 to 65 years, inclusive.
3. The patient has a score of = 6 on the PKAN-specific activities of daily living measure
(PKAN-ADL)
Exclusion Criteria:
1. The patient has required regular or intermittent invasive ventilatory support to
maintain vital signs within 24 weeks prior to randomization.
2. The patient has had a deep brain stimulation (DBS) device implanted within 6 months
prior to screening.
3. The patient has taken deferiprone within 30 days prior to screening.
4. The patient is unable to maintain stable doses of allowed concomitant medications for
the first 24 weeks of the study.
Age minimum:
6 Years
Age maximum:
65 Years
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: fosmetpantotenate (RE-024)
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Drug: Placebo
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Primary Outcome(s)
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Evaluate safety and tolerability by occurrence of adverse events classified by MedDRA classification dictionary and safety assessments including vital signs, physical examinations, clinical laboratory tests, C-SSRS assessments, and electrocardiograms.
[Time Frame: 24 weeks]
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Evaluate change in the score of a PKAN specific activities of daily living measure (PKAN-ADL)
[Time Frame: 24 weeks]
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Secondary Outcome(s)
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Evaluate change in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III score
[Time Frame: 24 weeks]
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Secondary ID(s)
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024PKAN15004
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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