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
      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