MD1003-AMN MD1003 in Adrenomyeloneuropathy

Overview

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

Full Title of Study: “MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: June 2016

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.

Interventions

  • Drug: MD1003 100 mg capsule
  • Drug: Placebo

Arms, Groups and Cohorts

  • Experimental: MD1003
    • MD1003 100mg capsules, 1 capsule tid for 24 months
  • Placebo Comparator: Placebo
    • Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months

Clinical Trial Outcome Measures

Primary Measures

  • Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
    • Time Frame: Baseline and 12 Months

Secondary Measures

  • Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%
    • Time Frame: Baseline, 9 months, 12 months
    • at Months 9 and Months 12 compared to the best value among screening and baseline.
  • Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20%
    • Time Frame: Baseline, 9 months, 12 months
    • at Months 9 and Months 12 compared to the best value among screening and baseline
  • Mean Change in TW25 (time to walk 25 feet)
    • Time Frame: Baseline and 12 months
  • Timed up and Go test (TUG)
    • Time Frame: 12 Months
  • Euroqol EQ-5D questionnaire
    • Time Frame: 12 months
    • Quality of Life questionnaire
  • Qualiveen Questionnaire
    • Time Frame: 12 Months
    • Qualiveen to evaluate urinary function

Participating in This Clinical Trial

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 Eligibility: Male

Minimum Age: 18 Years

Maximum Age: 60 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • MedDay Pharmaceuticals SA
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Patrick Aubourg, MD, Principal Investigator, Hopital Le Kremlin-Bicêtre
    • Frederic Sedel, MD, Study Director, Medday Pharmeuticals

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.