Effect of MD1003 in Amyotrophic Lateral Sclerosis

NCT ID: NCT03114215

Last Updated: 2019-06-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-06-29
• Study Completion Date: 2017-12-31

Brief Summary

This is a 6-month double blind randomized 2:1 placebo-controlled study with two arms (placebo, biotin 300 mg/day). The study will be followed by a 6-month extension phase during which all patients will receive biotin 300 mg/day.

Conditions

ALS; Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: OTHER
• Blinding Strategy: QUADRUPLE

Study Groups

MD1003

The investigational drug will consist in capsules of 100 mg biotin and excipients (lactose, magnesium stearate, croscarmellose sodium, Silica) tid during 12 months

Group Type: ACTIVE_COMPARATOR

MD1003

Intervention Type: DRUG

capsules 100mg 3 times per day

PLACEBO

This formulation consists in lactose powder and other excipients (magnesium stearate, croscarmellose sodium, Silica) as placebo, tid during 6 months and then switch to MD1003 tid during 6 additional months.

Group Type: PLACEBO_COMPARATOR

MD1003

Intervention Type: DRUG

capsules 100mg 3 times per day

Placebo oral capsule

Intervention Type: DRUG

capsules 100mg lactose 3 times per day

Interventions

MD1003

capsules 100mg 3 times per day

Intervention Type: DRUG

Placebo oral capsule

capsules 100mg lactose 3 times per day

Intervention Type: DRUG

Other Intervention Names

BIOTIN

Eligibility Criteria

Inclusion Criteria

• Age: 25 to 80 years, inclusive
• Male or female subjects with probable or confirmed ALS (revised international El Escorial criteria, Forbes et al., 2001).
• Patients presenting first motor deficits due to ALS for a maximum of three years at the first consultation in an ALS centre.
• Patients monitored for at least 6 months in an ALS centre or for whom the previous monitoring parameters are available (excepted for MIP and SNIP).
• Patients who have lost at least 5 points on the ALSFRS-R (ALS functional rating scale) during the last 12 months or at least 2 points during the preceding 6 months
• Patients who have been treated with riluzole for at least 3 months at a stable dose. In case of intolerance to this product or refusal for this treatment, patients who have not been treated with riluzole for at least 1 month before inclusion
• For patients with spinal form (onset of the disease affecting limbs) or respiratory form, slow vital capacity > 60% of predicted value.
• For patients with a bulbar form, slow vital capacity > 60% of theoretical value or, if spirometry not assessable (severe bulbar disability), patient should not have significant abnormality in both nocturnal capnography and nocturnal oximetry (median pCO2 (carbon dioxide partial pressure ) < 52 mmHg, SaO2 (arterial oxygen saturation ) < 90% less than 5% of the time during night) less than 3 months prior inclusion.
• Patients who are willing to give written consent (or oral consent in the presence of a trusted person if the patient is no longer able to write)
• Patients likely to be able to participate in all scheduled evaluation and complete all required study procedures (except for spirometry in bulbar patients with severe disability).

Exclusion Criteria

• Patients on non-invasive ventilation for respiratory insufficiency due to ALS for more than 10 hours a day
• Patients with an ALSFRS-R score at inclusion of < 20 (maximum score without disability = 48)
• Patients who have lost less than 5 points on the ALSFRS-R during the last year or less than 2 points during the preceding 6 months
• Patients with a gastrostomy
• Patients who have lost more than 15% of their reference weight (defined as weight before disease onset)
• Patient with dyspnoea at rest or with the least effort (score < 3 on the dyspnoea item of the ALSFRS-R)
• Patients with dementia
• Patient with severe or rapidly progressive form of ALS for whom the investigator estimates the life expectancy less than 3 months
• Patients with another progressive disease that has not been stabilized at the time of inclusion
• Patients with cancer, except basal cell carcinoma, for less than 5 years, or who require continuous treatment for cancer even if it is older
• Pregnant women.
• Subject who are not covered by a social security scheme.
• Subject under temporary or permanent Judicial Protection.
• Contraception: Both male subjects, and female subjects who are not either surgically sterile (tubal ligation/obstruction or removal of ovaries or uterus) or post-menopausal (no spontaneous menstrual periods for at least one year confirmed by a negative hormone panel), must commit to using two highly effective method of birth control for the duration of the study and for two months after the treatment termination.

• Minimum Eligible Age: 25 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

MedDay Pharmaceuticals SA

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hopital Gui De Chauliac

Montpellier, , France

Countries

France

References

Juntas-Morales R, Pageot N, Bendarraz A, Alphandery S, Sedel F, Seigle S, Camu W. High-dose pharmaceutical grade biotin (MD1003) in amyotrophic lateral sclerosis: A pilot study. EClinicalMedicine. 2020 Jan 27;19:100254. doi: 10.1016/j.eclinm.2019.100254. eCollection 2020 Feb.

Reference Type: DERIVED

PMID: 32140672 (View on PubMed)

Other Identifiers

MD1003CT2015-02-ALS

Identifier Type: -

Identifier Source: org_study_id