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ClinicalTrials.gov ID

NCT06973629

Lead Sponsor

Brainstorm-Cell TherapeuticsINDUSTRY

Overall Status

Not yet recruiting

Study Type

Interventional

Phase

Phase 3

Enrollment

200Estimated

Last Update Posted

May 22, 2025Actual

Start Date

Jun 30, 2025Estimated

Primary Completion Date

Nov 1, 2028Estimated

Completion Date

May 1, 2029Estimated

Official Title

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)

Brief Summary

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.

Detailed Description

This is a multicenter, Phase 3b study to assess the efficacy and safety of NurOwn in participants with early symptomatic ALS and moderate disease presentation in ALS. The study comprises two parts: a 24-week randomized, double blind placebo controlled period (Part A) followed by a 24-week open label expansion period (Part B).

Up to approximately 200 participants are planned to be enrolled and randomized 1:1 to the NurOwn and placebo groups in Part A. All eligible participants who complete Part A will have the option of entering Part B.

The trial includes a 9-week screening period. After the first screening visit (Screening Visit 1), there will be a Screening Visit 2, during which randomization 1:1 to the NurOwn and placebo groups will occur after confirming that all entry criteria are met. Following randomization, bone marrow aspiration will be scheduled. Stem cells from the bone marrow of all participants will be isolated, and then cryopreserved. Prior to each intrathecal (IT) dose administration, cells will be thawed, propagated, and induced into MSC-NTF cells (NurOwn).

In Part A, participants will receive NurOwn or placebo via IT injection every eight weeks at Weeks 0, 8, and 16.

In Part B, participants will receive NurOwn via IT injection every eight weeks at Weeks 24, 32, and 40.

All participants will be offered the option to participate in the collection and storage of blood/serum, CSF, and buccal samples for future analysis of biomarkers and genetic testing.

• Amyotrophic Lateral Sclerosis (ALS)
• Amyotrophic Lateral Sclerosis

• Debamestrocel - MSC-NTF (NurOwn)
• Placebo

Ages Eligible for Study

Adult (18-64)Older Adult (65+)

Sexes Eligible for Study

All

Accepts Healthy Volunteers

No

Eligibility Criteria

Inclusion 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.

Exclusion Criteria:

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.

Primary Purpose

Treatment

Allocation

Randomized

Interventional Model

Parallel Assignment

Masking

Quadruple

Lead Sponsor

Brainstorm-Cell TherapeuticsINDUSTRY

contact@mayo.edu904-953-0853

contact@northwestern.edu312-926-2000

contact@mayo.edu