Overview
MD1003-AMN MD1003 in Adrenomyeloneuropathy
Status:
Completed
Completed
Trial end date:
2017-06-01
2017-06-01
Target enrollment:
0
0
Participant gender:
Male
Male
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 adrenomyeloneuropathyPhase:
Phase 2/Phase 3Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
MedDay Pharmaceuticals SATreatments:
Biotin
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