Brief Title
MD1003-AMN MD1003 in Adrenomyeloneuropathy
Official Title
MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
Brief Summary
The primary objective of the trial is to demonstrate the superiority of biotin at 300 mg/day over placebo in the clinical improvement (walking tests) of patients with adrenomyeloneuropathy
Detailed Description
AMN and progressive multiple sclerosis share some similarities including progressive spastic paraparesis and secondary energy failure leading to progressive axonal degeneration. Therefore, it was hypothesized that high doses of biotin might be efficient in patients with AMN.
Study Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
Secondary Outcome
Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%
Condition
Adrenomyeloneuropathy
Intervention
MD1003 100 mg capsule
Study Arms / Comparison Groups
MD1003
Description: MD1003 100mg capsules, 1 capsule tid for 24 months
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
67
Start Date
October 2014
Completion Date
June 2017
Primary Completion Date
June 2016
Eligibility Criteria
Inclusion Criteria: - ABCD1 gene mutation identified - Elevated plasma VLCFA - Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk - EDSS score ≥ 3.5 and ≤ 6.5 - Normal brain MRI or brain MRI showing : - abnormalities that can be observed in AMN patients without cerebral demyelination with a maximum Loes score of 4 - and/or stable (≥6 months) cerebral demyelination without gadolinium enhancement with a Loes score ≤12. - Appropriate steroid replacement if adrenal insufficiency is present - Likely to be able to participate in all scheduled evaluation visits and complete all required study procedures - Signed and dated written informed consent to participate in the study in accordance with local regulations - Affiliated to a Health Insurance Exclusion Criteria: - Brain MRI abnormalities with a Loes score > 12 or with gadolinium enhancement - Any progressive neurological disease other than AMN - Impossibility to perform the walk tests and the TUG test - Patients with uncontrolled hepatic disorder, renal or cardiovascular disease, or any progressive malignancy - Any new medication for AMN including Fampridine initiated less than 1 month prior to inclusion - Contra-indications for MRI procedure such as subjects with paramagnetic materials in the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants. - Inclusion in another therapeutic clinical trial for ALD - Not easily contactable by the investigator in case of emergency or not capable to call the investigator
Gender
Male
Ages
18 Years - 60 Years
Accepts Healthy Volunteers
No
Contacts
Patrick Aubourg, MD, ,
Location Countries
France
Location Countries
France
Administrative Informations
NCT ID
NCT02961803
Organization ID
MD1003CT2014-01AMN
Responsible Party
Sponsor
Study Sponsor
MedDay Pharmaceuticals SA
Study Sponsor
Patrick Aubourg, MD, Principal Investigator, Hopital Le Kremlin-Bicêtre
Verification Date
November 2016