Efficacy and Safety of Masitinib in the Treatment of Progressive Multiple Sclerosis

• ClinicalTrials.gov Identifier: NCT01433497
• Recruitment Status: Completed
• First Posted: September 14, 2011
• Last Update Posted: April 8, 2020
• Sponsor: AB Science
• Information provided by (Responsible Party): AB Science

Tracking Information

• First Submitted Date: September 12, 2011
• First Posted Date: September 14, 2011
• Last Update Posted Date: April 8, 2020
• Actual Study Start Date: August 2011
• Actual Primary Completion Date: September 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: December 5, 2018)

EDSS [ Time Frame: 96 weeks ]

Expanded Disability Status Scale (EDSS) after 96 weeks of treatment

• Original Primary Outcome Measures (submitted: September 13, 2011): Multiple Sclerosis Functional Composite (MSFC) [ Time Frame: week 96 ]

Current Secondary Outcome Measures (submitted: December 5, 2018)

• MSQOL-54 [ Time Frame: 96 weeks ]
  Multiple Sclerosis Quality of Life 54 items (MSQOL-54)
• MSFC [ Time Frame: 96 weeks ]
  Multiple Sclerosis Functional Composite (MSFC)

Original Secondary Outcome Measures (submitted: September 13, 2011)

• EDSS [ Time Frame: week 96 ]
• Multiple Sclerosis Quality of Life 54 items (MSQOL-54) [ Time Frame: week 96 ]
• Timed 25-foot walk [ Time Frame: week 96 ]
• Nine-hole peg test [ Time Frame: week 96 ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Efficacy and Safety of Masitinib in the Treatment of Progressive Multiple Sclerosis
• Official Title: A 96 Week, Prospective, Multicentre, Randomized, Double-blind, Placebo-controlled, 2 Parallel-groups, Phase 3 Study to Compare Efficacy and Safety of Masitinib 4.5 mg/kg/Day Versus Placebo in the Treatment of Patients With Primary Progressive or Relapse-free Secondary Progressive Multiple Sclerosis
• Brief Summary: The purpose of this study is to compare the safety and efficacy of masitinib 6 mg/kg/day versus placebo in the treatment of patients with primary progressive multiple sclerosis or relapse-free secondary progressive multiple sclerosis.
• Detailed Description: Masitinib is a selective tyrosine kinase inhibitor that is thought to exert a neuroprotective effect through its activity on mast cells and other non-neuronal cells of the central nervous system, with subsequent modulation of inflammatory and neurodegenerative processes. The objective of this study is to compare the efficacy and safety of masitinib at 4.5 mg/kg/day versus matched placebo, or masitinib at 4.5 mg/kg/day with a dose escalation to 6 mg/kg/day after 3 months of treatment versus matched placebo, in the treatment of patients with primary progressive multiple sclerosis or relapse-free secondary progressive multiple sclerosis. Approximately 600 patients will be randomized into four treatment groups with a 2:2:1:1 design. The primary outcome measure is the Expanded Disability Status Scale (EDSS) after 96 weeks of treatment in the overall study population with subgroup analysis performed in stratum (primary progressive multiple sclerosis / secondary progressive multiple sclerosis).
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Multiple Sclerosis, Secondary Progressive
• Multiple Sclerosis, Primary Progressive
• Multiple Sclerosis, Relapse Free

Intervention

• Drug: Masitinib
  Other Name: AB1010
• Drug: Placebo
  Other Name: Placebo Oral Tablet

Study Arms

• Experimental: Experimental Arm A
  Participants receive masitinib (4.5 mg/kg/day), given orally twice daily.
  Intervention: Drug: Masitinib
• Experimental: Experimental Arm B
  Participants receive masitinib (4.5 mg/kg/day), given orally twice daily, with a dose escalation to 6 mg/kg/day after 3 months of treatment.
  Intervention: Drug: Masitinib
• Placebo Comparator: Placebo Comparator A
  Participants receive placebo given orally twice daily.
  Intervention: Drug: Placebo
• Placebo Comparator: Placebo Comparator B
  Participants receive placebo, given orally twice daily, with a matched dose escalation after 3 months of treatment.
  Intervention: Drug: Placebo

• Publications *: Vermersch P, Brieva-Ruiz L, Fox RJ, Paul F, Ramio-Torrenta L, Schwab M, Moussy A, Mansfield C, Hermine O, Maciejowski M; AB07002 Study Group. Efficacy and Safety of Masitinib in Progressive Forms of Multiple Sclerosis: A Randomized, Phase 3, Clinical Trial. Neurol Neuroimmunol Neuroinflamm. 2022 Feb 21;9(3):e1148. doi: 10.1212/NXI.0000000000001148. Print 2022 May.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: December 5, 2018): 656
• Original Estimated Enrollment (submitted: September 13, 2011): 450
• Actual Study Completion Date: February 2020
• Actual Primary Completion Date: September 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

Main inclusion criteria:

- Patient suffering from either primary progressive or secondary progressive multiple sclerosis without relapse within 2 years before inclusion according to the revised McDonald's criteria.

Main exclusion criteria:

- Patient suffering from a disease other than MS that would better explain the patient's neurological clinical signs and symptoms and/or MRI lesions

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 75 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Bulgaria, France, Germany, Greece, Poland, Romania, Spain
• Removed Location Countries: United States

Administrative Information

• NCT Number: NCT01433497
• Other Study ID Numbers: AB07002
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: AB Science
• Original Responsible Party: Same as current
• Current Study Sponsor: AB Science
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Patrick Vermersch, MD, PhD; Hôpital Salengro, Lille, France

• PRS Account: AB Science
• Verification Date: April 2020