Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)

Conditions

• Relapsing Multiple Sclerosis
• Relapsing Remitting Multiple Sclerosis
• Secondary Progressive Multiple Sclerosis

Eligibility Criteria

Sex

ALL

Age

18 Years - 55 Years

Healthy Volunteers

Not accepted

Interventions

• Autologous Hematopoietic Stem Cell Transplantation — PROCEDURE
  1. PBSC mobilization \& collection regimen per protocol/ institutional standards includes: intravenous cyclophosphamide (Cytoxan®), 4 grams/m\^2); intravenous mesna (Mesnex®),a total delivery of 4 grams/m\^2); oral dexamethasone, 10 mg dose, four times daily); subcutaneous filgrastim,10 mcg/kg/day until leukapheresis goal is completed; and CD34+ peripheral blood stem cells collection by leukapheresis. 2. Conditioning per protocol\& institutional standards: * 6-day BEAM (e.g. Carmustine (BCNU), Etoposide (VP-16), Cytarbine (Ara-C), and Melphalan) chemotherapy protocol and, * rabbit anti-thymocyte globulin (rATG) 2.5 mg/kg/day x2 3. Autologous cryopreserved graft infusion: The target Cluster of Differentiation (CD)34+ cell dose for infusion is 5 x 10\^6 CD34+ cells/kg (minimum 4 x 10\^6 CD34+ cells/kg; maximum 7.5 x 10\^6 CD34+ cells/kg). For 1\&2 above: Ideal body weight (IBW) versus Actual Body Weight (ABW) are applicable.
• Best Available Therapy (BAT) — BIOLOGICAL
  Disease-modifying therapy (DMT) selected by the Site Investigator from the below: * cladribine * natalizumab * alemtuzumab * ocrelizumab, * rituximab, * ofatumumab, or * ublituximab

Study Details

This is a multi-center prospective rater-masked (blinded) randomized controlled trial of 156 participants, comparing the treatment strategy of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) to the treatment strategy of Best Available Therapy (BAT) for treatment-resistant relapsing multiple sclerosis (MS). Participants will be randomized at a 1 to 1 (1:1) ratio. All participants will be followed for 72 months after randomization (Day 0, Visit 0).

Key Dates

Start date

Dec 19, 2019

Status verified

Jan 2026

Primary completion

Oct 31, 2026

Completion

Oct 31, 2029

Study Design

Enrollment

156 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: AHSCT
  AHSCT: Myeloablative and Immunoablative therapy followed by Autologous Hematopoietic Stem Cell Transplantation Participants will undergo: 1. Mobilization and graft collection: mobilization of peripheral blood stem cells (PBSC) with cyclophosphamide, filgrastim, and dexamethasone. The autologous graft will be collected by leukapheresis and cryopreserved. 2. Conditioning: high dose myeloablative and immunoablative conditioning with a six-day BEAM chemotherapy and rabbit anti-thymocyte globulin regimen will be initiated ≥30 days after cyclophosphamide mobilization. 3. Autologous cryopreserved graft infusion: the cryopreserved peripheral blood stem cells (PBSC) graft will be thawed and infused the day following completion of the conditioning regimen. Each bag will be thawed and infused according to institutional standards consistent with the Foundation for the Accreditation of Cellular Therapy (FACT) guidelines. Participants will receive prednisone following graft infusion.
• Active Comparator: Best Available Therapy (BAT)
  Participants randomized to BAT: Best available therapy will be selected by the Site Investigator from: Cladribine (Mavenclad®), natalizumab (Tysabri®), alemtuzumab (Campath®, Lemtrada®), ocrelizumab (Ocrevus®), ublituximab (BRIUMVI™), rituximab (Rituxan®), or ofatumumab (Arzerra®) (after approval by the FDA for relapsing MS).

Primary Outcome Measure

Multiple Sclerosis (MS) Relapse-Free Survival [ Time Frame: From Day 0 (Randomization to Treatment) Up to 36 Months (3 Years) ]

Locations (22)

Find similar trials in Palo Alto, CA

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