NCT02961803CompletedPhase 2
MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
MedDay Pharmaceuticals SA4 sites in 3 countries67 target enrollmentStarted: October 2014Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Enrollment
- 67
- Locations
- 4
- Primary Endpoint
- Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
Study Overview
Brief 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 adrenomyeloneuropathy
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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 18 Years to 60 Years (Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
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 ≤
- •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
Arms & Interventions
MD1003
Experimental
MD1003 100mg capsules, 1 capsule tid for 24 months
Intervention: MD1003 100 mg capsule (Drug)
Placebo
Placebo Comparator
Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months
Intervention: MD1003 100 mg capsule (Drug)
Placebo
Placebo Comparator
Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
Time Frame: Baseline and 12 Months
Secondary Outcomes
- Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20%(Baseline, 9 months, 12 months)
- Timed up and Go test (TUG)(12 Months)
- Euroqol EQ-5D questionnaire(12 months)
- Mean Change in TW25 (time to walk 25 feet)(Baseline and 12 months)
- Qualiveen Questionnaire(12 Months)
- Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%(Baseline, 9 months, 12 months)
Investigators
Study Sites (4)
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