Effect of MD1003 in Progressive Multiple Sclerosis (SPI2)
Purpose
The purpose of this study is to demonstrate the superiority of MD1003 over placebo in the disability of patients suffering from progressive multiple sclerosis and especially those with gait impairment.
Condition
- Multiple Sclerosis
Eligibility
- Eligible Ages
- Between 18 Years and 65 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Patient aged 18-65 years old - Signed and dated written informed consent form in accordance with local regulations: having freely given their written informed consent to participate in the study - Diagnosis of primary or secondary progressive MS fulfilling revised McDonald criteria (2010) and Lublin criteria (2014) - Documented evidence of clinical disability progression within the 2 years prior to inclusion, i.e. a) progression of EDSS during the past two years of at least 1 point sustained for at least 6 months if inclusion EDSS is from 3.5 to 5.5 or at least 0.5 point increase sustained for at least 6 months if inclusion EDSS is from 6 to 6.5 or b) increase of TW25 by at least 20% in the last two years sustained for at least 6 months or c) other well-documented objective worsening validated by the Adjudication Committee - EDSS at inclusion from 3.5 to 6.5 - TW25 < 40 seconds at inclusion visit - Kurtzke pyramidal functional subscore ≥2 defined as "minimal disability: patient complains of motor-fatigability or reduced performance in strenuous motor tasks (motor performance grade 1) and/or BMRC grade 4 in one or two muscle groups"
Exclusion Criteria
- Clinical evidence of a relapse in 24 months prior to inclusion - Treatment with any product containing biotin as single ingredient within six months prior to inclusion (multivitamin supplementation authorized if biotin < 1mg per day) - Concomitant treatment with fampridine at inclusion or in the 30 days prior to inclusion - New immunosuppressive/immunomodulatory drug initiated less than 90 days prior to inclusion - Treatment with botulinum toxin (except for cosmetic purpose) initiated within 6 months prior to inclusion - In-patient rehabilitation program within the 3 months prior to inclusion - Pregnancy, breastfeeding or women with childbearing potential without acceptable form of contraception - Men unwilling to use an acceptable form of contraception - Any general chronic handicapping/incapacitating disease other than MS - Any serious disease necessitating biological follow-up with biological tests using biotinylated antibodies or substrates - Past history of rhabdomyolysis/metabolic myopathy - Known fatty acids beta oxidation defect - Known hypersensitivity or intolerance to biotin, analogues or excipients, patients with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption - Patients with hypersensitivity or any contra-indication to Gadolinium - Patients with uncontrolled hepatic disorder, renal or cardiovascular disease, or cancer - Laboratory tests out of normal ranges considered by the investigator as clinically significant with regards to the study continuation - Patients with history or presence of alcohol abuse or drug addiction - Untreated or uncontrolled psychiatric disorders, especially suicidal risk assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) - Participation in another research study involving an investigational product (IP) in the 90 days prior to inclusion, or planned use during the study duration - Patients likely to be non-compliant to the study procedures or for whom a long-term follow-up seems to be difficult to achieve - Relapse that occurs between inclusion and randomization visit
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Intervention Model Description
- PART 1: Total duration of Part 1 is 27 months. The randomized double-blind placebo-controlled period ranges from 15 to 27 months depending upon the randomization date of an individual patient. Once the last month 15 evaluation of the study has been completed, patients will switch to the active drug at the next planned visit. Participants and study personnel will remain blinded as to the original treatment assignment. Maximum duration of double-blind period per patient will be no longer than 27 months. PART 2: At the last evaluation of Part 1 (Visit 11/Month 27) all participants will be offered active treatment in an open label extension for 39 additional months (From V11/M27 to V18/M66). The purpose of the active drug extension is to further define the safety of MD1003.
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Placebo Comparator GROUP 1 |
Placebo capsule, 1 capsule tid (morning,noon and evening) for 15 months and up to 27 months. |
|
|
Experimental GROUP 2 |
MD1003 capsule, 1 capsule tid (morning,noon and evening) for 15 months and up to 27 months. |
|
More Details
- Status
- Terminated
- Sponsor
- MedDay Pharmaceuticals SA