Effect of MD1003 in Progressive Multiple Sclerosis (SPI2)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02936037
• Date of registration: 14/10/2016
• Prospective Registration: Yes
• Primary sponsor: MedDay Pharmaceuticals SA
• Public title: Effect of MD1003 in Progressive Multiple Sclerosis (SPI2) SPI2
• Scientific title: Effect of MD1003 in Progressive Multiple Sclerosis: a Randomized Double Blind Placebo Controlled Study
• Date of first enrolment: December 2016
• Target sample size: 642
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02936037
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, Czech Republic, Czechia, Germany, Hungary, Italy, Netherlands, Poland, Spain, Sweden, Turkey, United Kingdom, United States

Contacts

• Name: Frederic Sedel, MD, PHD
• Affiliation: Medday Pharmaceuticals

• Name: Bruce Cree, MD, PHD
• Affiliation: University of California, San Francisco

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Multiple Sclerosis

Intervention(s)

Drug: PLACEBO

Drug: MD1003 100mg capsule

Primary Outcome(s)

Proportion of Patients Improved on Either Expanded Disability Status Scale (EDSS) or Time to Walk 25 Feet (TW25) [Time Frame: 15 months]

Secondary Outcome(s)

CGI-I Score (Clinical Global Impression of Change - Improvement), Evaluated Both by the Patient (SGI) and by the Evaluating Physician (CGI) [Time Frame: 15 months]

Mean Change in TW25 Between M0 and M15 [Time Frame: 15 months]

Time to 12-Weeks Confirmed EDSS Progression [Time Frame: 3 to 27 months]

Secondary ID(s)

MD1003CT2016-01MS-SPI2

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 23/11/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02936037