Launch of the MD1003 trial in adrenomyeloneuropathy in France and Spain

Written on Monday 24 November 2014

We are happy to announce the launch in France and Spain of the clinical trial testing the MD1003 drug developed by MedDay Pharmaceuticals in patients with adrenomyeloneuropathy (AMN). Authorizations for the investigational center in Germany are awaited. The trial, funded by ELA for 800.000 euros, will gather 60 AMN men with no cerebral involvement. Below is a detailed presentation of the trial.




  • Authorized in France and Spain
  • On-going patient recruitment in France and Spain
  • Awaiting authorization in Germany

Drug: MD1003 (biotin)

  • To show clinical efficacy of the MD1003 treatment in AMN compared to placebo
  • To evaluate the safety of the treatment

Duration of the trial: 2 years
Start date: November 2014
Estimated end of the trial: April 2017

Principal investigator: Pr Patrick Aubourg, Bicetre hospital, Le Kremlin-Bicetre, France

  • Pr Patrick Aubourg, Bicetre hospital, Le Kremlin-Bicetre, France
  • Dr Yann Nadjar, Pitié-Salpêtrière hospital, Paris, France
  • Dr Aurora Pujol, IDIBELL, Duran i Reinals hospital, L’Hospitalet de Llobregat, Spain
  • Dr Carlos Casasnoves, IDIBELL, Duran i Reinals hospital, L’Hospitalet de Llobregat, Spain
  • Pr Wolfgang Köhler, Hubertusburg hosital, Wemsdorf, Germany


  • Bicetre hospital, Le Kremlin-Bicetre, France
  • Pitié-Salpêtrière hospital, Paris, France
  • Duran i Reinals hospital, L’Hospitalet de Llobregat, Spain
  • Hubertusburg hospital, Wermsdorf, Germany

Sponsor: MedDay Pharmaceuticals, France –
Funding: ELA – 800.000 euros
Reference : EudraCT 2014-000698-38
60 AMN men divided into 2 groups the first year of the treatment as follows:

  • 20 patients treated with a placebo
  • 40 patients treated with the MD1003 drug

During the second year of the trial, all the patients will receive the MD1003 drug.

Experimental treatment
Oral daily intake of MD1003 capsules at a dose of 300 mg or of placebo

Inclusion criteria

  • 18 to 60 years old men
  • Confirmed ABCD1 gene mutation
  • Increased very long chain fatty acids in plasma
  • Clinical signs of AMN with walking impairment
  • Normal cerebral MRI or MRI showing abnormalities of AMN patients without cerebral involvement
  • Corticoid treatment if adrenal insufficiency

Exclusion criteria

  • IMRI showing abnormalities of AMN patients with cerebral involvement
  • Other progressive neurological disease than AMN
  • Unable to perform walking and balance tests
  • Patients with liver impairment, renal or cardiovascular disease or evolutive cancer
  • Intake of drugs for AMN including fampridine if initiated less than a month before inclusion
  • MRI contraindications
  • Participation to another clinical trial for ALD

Outcome measures
Principal outcome measure: Mean change observed during the 2 Minutes walking test prior and after the 12 months treatment.

Secondary outcome measures

  • TW25FW walking test measuring the time to walk 25 feet
  • Time to stand up and go
  • Euroqol ED-5D quality of life questionnaire
  • Qualiveen questionnaire on urinary function
  • Safety assessment of elevated dose of biotin

Exploratories studies will also be conducted in some centers like MRIs, nerve velocity conduction and muscular strength.

Additional information

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Fax: +34 932 607 414
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