MSC-NTF (NurOwn) for ALS

Recruiting at 14 trial locations

Overseen By Chief Medical Officer, Brainstorm Cell Therapeutics, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Brainstorm-Cell Therapeutics

Disqualifiers: Prior stem cell therapy, Cancer, Other motor neuron diseases, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 8

200 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for people with early symptomatic ALS and moderate disease presentation. Participants will be involved in the study for a total of 48 months, receiving treatments every 8 weeks and visiting the clinic regularly for checkups.

Inclusion Criteria

≤45 points on ALSFRS-R total score at Screening Visit 1

≥2 points on each item of the ALSFRS-R at the Screening Visit 1

Participants must adhere to highly effective methods of contraception as specified in the study protocol

Exclusion Criteria

Active participation in any other ALS interventional study

I cannot lie flat for treatments or tolerate certain medical procedures.

Pregnant women or women currently breastfeeding

Timeline

Screening

Participants are screened for eligibility to participate in the trial

9 weeks

2 visits (in-person)

Treatment (Part A)

Participants receive NurOwn or placebo via IT injection every eight weeks

24 weeks

3 visits (in-person)

Open-label Extension (Part B)

All participants receive NurOwn via IT injection every eight weeks

24 weeks

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Debamestrocel - MSC-NTF (NurOwn)
• Placebo

Trial Overview The trial is testing Debamestrocel - MSC-NTF (NurOwn) against a placebo to see if it's safe and effective in treating ALS. After an initial comparison phase of 24 months, all participants will receive NurOwn for another 24 months.

Participant Groups

Group I: Debamestrocel - MSC-NTF (NurOwn) Experimental Treatment 1 Intervention

NTF-secreting mesenchymal stem cells (MSC-NTF cells) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients

Group II: Placebo Placebo Group 1 Intervention

Placebo is comprised of Dulbecco Modified Eagle Medium (DMEM)

Who Is Running the Clinical Trial?

Brainstorm-Cell Therapeutics

Lead Sponsor

Trials

6

Recruited

290+