MD1003-AMN MD1003 in Adrenomyeloneuropathy

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02961803
• Date of registration: 09/11/2016
• Prospective Registration: No
• Primary sponsor: MedDay Pharmaceuticals SA
• Public title: MD1003-AMN MD1003 in Adrenomyeloneuropathy
• Scientific title: MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
• Date of first enrolment: October 2014
• Target sample size: 67
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02961803
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

France, Germany, Spain

Contacts

• Name: Patrick Aubourg, MD
• Affiliation: Hopital Le Kremlin-Bicêtre

• Name: Frederic Sedel, MD
• Affiliation: Medday Pharmeuticals

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 60 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Adrenomyeloneuropathy

AMN

Adrenoleukodystrophy

Intervention(s)

Drug: MD1003 100 mg capsule

Drug: Placebo

Primary Outcome(s)

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

Secondary Outcome(s)

Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20% [Time Frame: Baseline, 9 months, 12 months]

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]

Qualiveen Questionnaire [Time Frame: 12 Months]

Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20% [Time Frame: Baseline, 9 months, 12 months]

Secondary ID(s)

MD1003CT2014-01AMN

Source(s) of Monetary Support

Please refer to primary and secondary sponsors