MD1003 IN ADRENOMYELONEUROPATHY

Phase 1

• Conditions: Adrenomyeloneuropathy; MedDRA version: 17.0Level: LLTClassification code 10069075Term: Adrenomyeloneuropathy without cerebral involvementSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2014-000698-38-ES
• Lead Sponsor: MEDDAY SAS

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-06-13
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 60

Inclusion Criteria

- Men of 18 to 60 years old, inclusive
- 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 60
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Brain MRI abnormalities with a Loes score > 12 or with gadolinium enhacement
- 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 evolutive malignancy
- Any new medication for AMN including Fampridine initiated less than 1 month prior to inclusion
- Contraindications 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
- Patient not easily reachable by the investigator in case of emergency or not capable to call the investigator

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified