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

Summary

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).

Conditions

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

Interventions

PROCEDURE

Autologous Hematopoietic Stem Cell Transplantation

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.

BIOLOGICAL

Best Available Therapy (BAT)

Disease-modifying therapy (DMT) selected by the Site Investigator from the below: * cladribine * natalizumab * alemtuzumab * ocrelizumab, * rituximab, * ofatumumab, or * ublituximab

Sponsors & Collaborators

• Immune Tolerance Network (ITN)

  collaborator NETWORK

• Blood and Marrow Transplant Clinical Trials Network

  collaborator NETWORK

• PPD Development, LP

  collaborator INDUSTRY

• Rho Federal Systems Division, Inc.

  collaborator INDUSTRY

• National Institute of Allergy and Infectious Diseases (NIAID)

  lead NIH

Principal Investigators

• Jeffrey A. Cohen, MD · Mellen Center for MS Treatment and Research, Cleveland Clinic

• George E. Georges, MD · Northwestern University

• Paolo A. Muraro, MD, PhD · Department of Medicine, Imperial College London

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

55 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2019-12-19

Primary Completion

2026-10-31

Completion

2029-10-31

FDA Drug

Yes

Countries

• United States

Study Locations