MD1003-AMN MD1003 in Adrenomyeloneuropathy

Adrenoleukodystrophy Clinical Trial

Official title:

MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02961803
• Other study ID #: MD1003CT2014-01AMN
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: November 9, 2016
• Last updated: October 6, 2017
• Start date: October 2014
• Est. completion date: June 2017

Study information

• Verified date: November 2016
• Source: MedDay Pharmaceuticals SA
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 67
• Est. completion date: June 2017
• Est. primary completion date: June 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 60 Years

Eligibility

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

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Adrenomyeloneuropathy
• AMN

Intervention

Drug:
• MD1003 100 mg capsule

• Placebo

Locations

Country	Name	City	State
France	Hôpital Bicêtre	Le Kremlin Bicêtre
France	Groupe Hospitalier Pitié-Salpêtrière	Paris
Germany	MS-Ambulanz Fachkrankenhaus Hubertusburg	Wermsdorf
Spain	Hospital Duran i Reynals / Bellvitge	Barcelona

Sponsors (1)

Lead Sponsor	Collaborator
MedDay Pharmaceuticals SA

Countries where clinical trial is conducted

France,  Germany,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline		Baseline and 12 Months
Secondary	Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%	at Months 9 and Months 12 compared to the best value among screening and baseline.	Baseline, 9 months, 12 months
Secondary	Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20%	at Months 9 and Months 12 compared to the best value among screening and baseline	Baseline, 9 months, 12 months
Secondary	Mean Change in TW25 (time to walk 25 feet)		Baseline and 12 months
Secondary	Timed up and Go test (TUG)		12 Months
Secondary	Euroqol EQ-5D questionnaire	Quality of Life questionnaire	12 months
Secondary	Qualiveen Questionnaire	Qualiveen to evaluate urinary function	12 Months

External Links

•   Sponsor