Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)

Description

The goal of this two-part clinical trial is: 1) to evaluate the safety and efficacy of Debamestrocel - MSC-NTF (NurOwn) compared to placebo in participants with early symptomatic ALS and moderate disease presentation in ALS; followed by 2) further evaluation by providing NurOwn to all participants in an open label extension period. Researchers will compare NurOwn to a placebo (a look-alike substance that contains no drug) to evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS. Participants will: Receive NurOwn or a placebo every 8 weeks for 24 weeks. After that, every participant will receive NurOwn every 8 weeks for an additional 24 weeks. They will visit the clinic approximately every 8 weeks for checkups and tests.

Conditions

Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis

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Study Overview

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Study Details

Study overview

The goal of this two-part clinical trial is: 1) to evaluate the safety and efficacy of Debamestrocel - MSC-NTF (NurOwn) compared to placebo in participants with early symptomatic ALS and moderate disease presentation in ALS; followed by 2) further evaluation by providing NurOwn to all participants in an open label extension period. Researchers will compare NurOwn to a placebo (a look-alike substance that contains no drug) to evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS. Participants will: Receive NurOwn or a placebo every 8 weeks for 24 weeks. After that, every participant will receive NurOwn every 8 weeks for an additional 24 weeks. They will visit the clinic approximately every 8 weeks for checkups and tests.

A Two-Part Phase 3b Randomized, Double-Blind, Placebo-Controlled, Followed by Open-Label Extension, Multicenter Study of the Efficacy and Safety of MSC-NTF (NurOwn) in Participants With With Early Symptomatic and Moderate Disease Presentation in Amyotrophic Lateral Sclerosis (ALS)

Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)

Condition
Amyotrophic Lateral Sclerosis (ALS)
Intervention / Treatment

-

Contacts and Locations

Phoenix

Barrow Neurological Institute, Phoenix, Arizona, United States, 85013

La Jolla

University of California San Diego Medical Center, La Jolla, California, United States, 92093

Los Angeles

University of Southern California, Los Angeles, California, United States, 90033

San Francisco

California Pacific Medical Center, San Francisco, California, United States, 94115

San Francisco

University of California, San Francisco, San Francisco, California, United States, 94143

Aurora

University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States, 80045

Davie

Nova Southeastern University, Davie, Florida, United States, 33328

Jacksonville

Mayo Clinic, Jacksonville, Florida, United States, 32224

Tampa

University of South Florida, Tampa, Florida, United States, 33612

Chicago

Northwestern Medicine, Chicago, Illinois, United States, 60611

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Male and female participants 18 to 75 years old (inclusive) at Screening Visit 1.
  • 2. ALS diagnosed as laboratory-supported probable, clinically probable, or definite as defined by the revised El Escorial criteria.
  • 3. Having onset of ALS symptoms, including muscle weakness, within 24 months from Screening Visit 1.
  • 4. ≥2 points on each item of the ALSFRS-R at the Screening Visit 1.
  • 5. ≤45 points on ALSFRS-R total score at Screening Visit 1.
  • 6. Upright slow vital capacity (SVC) measure ≥65% of predicted for gender, height, and age at Screening Visit 1.
  • 7. Participants must adhere to highly effective methods of contraception as specified in the study protocol.
  • 1. Prior stem cell therapy of any kind.
  • 2. Active participation in any other ALS interventional study.
  • 3. Inability to lie flat for the duration of IT cell treatment and/or bone marrow biopsy, or inability to tolerate study procedures for any other reason.
  • 4. Any unstable clinically significant medical condition other than ALS
  • 5. Any history of malignancy, within the previous 5 years, with the exception of localized skin cancers, cervical cancer in-situ or prostate cancer in-situ (with no evidence of metastasis, significant invasion, or reoccurrence within 3 years of baseline).
  • 6. Primary brain cancer or cancer with CNS involvement is exclusionary.
  • 7. Other types of motor neuron disease such as primary lateral sclerosis, progressive muscular atrophy, and progressive bulbar palsy.
  • 8. Usage of a feeding tube at Screening Visit 1 or Screening Visit 2.
  • 9. Pregnant women or women currently breastfeeding.

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Brainstorm-Cell Therapeutics,

Study Record Dates

2029-05-01