MD1003-AMN MD1003 in Adrenomyeloneuropathy

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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

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

Study Phase

Phase 2/Phase 3

Study Type


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%




MD1003 100 mg capsule

Study Arms / Comparison Groups

Description:  MD1003 100mg capsules, 1 capsule tid for 24 months


* 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


Estimated Enrollment


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

          -  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




18 Years - 60 Years

Accepts Healthy Volunteers



Patrick Aubourg, MD, , 

Location Countries


Location Countries


Administrative Informations



Organization ID


Responsible Party


Study Sponsor

MedDay Pharmaceuticals SA

Study Sponsor

Patrick Aubourg, MD, Principal Investigator, Hopital Le Kremlin-Bicêtre

Verification Date

November 2016