Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

Quick Info:

Status:
Currently Recruiting
Estimated Enrollment:
200
Phase:
3
Treatment Type:
Biological: Autologous MSC-NTF cells - NurOwn
Trial Type:
Randomized, placebo controlled, multidose
Sponsor:
Brainstorm-Cell Therapeutics
Primary Investigator:
Contact Information:

Enrollment Criteria:

Forced Ventilation

Breathing Ability

Percent lung function (FVC) or (SVC)
≥65% svc
Months/Onset

Months Since Onset

Number of months since first
symptoms of ALS
<24 months
BiPap Allowed

Non-Invasive Ventilation (NIV)

Can PALS use a BiPAP in the trial?
no
DPS Allowed

Diaphragm Pacer (DPS)

Can PALS use a DPS in the trial?
no
Edaravone Usage

Edaravone Usage

Can a PALS use edaravone (Radicut/Radicava)
while enrolled in the trial?
No

Update Notes:

11/29/2018No significant updates
5/25/2018No significant updates
3/14/2018All enrollment sites accepting patients
3/8/2018Location updated
12/12/2017Location updated
9/19/2017Recruitment status at location updated
9/13/2017Recruitment Status updated
7/31/2017Trial added

Locations:

Massachusetts General Hospital, Boston, 02114
University of California, Irvine, Irvine, 92697
Mayo Clinic, Rochester, 55902
University of Massachusetts Medical School, Worcester, 01655
California Pacific Medical Center, San Francisco, 94115
Cedars-Sinai Medical Center, Angeles, 90048

Other Information:

Purpose: This study will evaluate the safety and efficacy of repeated administration of NurOwn® (MSC-NTF cells) therapy, which is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patient's own bone marrow, propagated ex vivo and induced to secrete Neurotrophic factors (NTFs). The autologous NurOwn® (MSC-NTF cells) are back-transplanted into the patient intrathecally by standard lumbar puncture where neurons and glial cells are expected to take up the neurotrophic factors secreted by the transplanted cells
Eligibility: 18-60 years, all genders, not accepting healthy volunteers. Participants taking a stable dose of Riluzole permitted. ALS FRS ≥25
Details: Neurotrophic factors (NTFs) are potent survival factors for embryonic, neonatal, and adult neurons and are considered potential therapeutic candidates for ALS. Delivery of multiple NTFs to the immediate environment of afflicted neurons in ALS patients is expected to improve their survival and thus slow down disease progression and alleviate symptoms. NTF-secreting mesenchymal stromal cells (MSC-NTF cells) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients. Participants meeting the inclusion and exclusion criteria will be randomized and will undergo bone-marrow aspiration. MSC of the participants randomized to the treatment group will be induced into MSC-NTF cells. Participants will undergo a total of three intrathecal (IT) transplantations with NurOwn® (MSC-NTF cells) or matching placebo at three bi-monthly intervals
Collaborator(s):
  • California Institute for Regenerative Medicine
News Articles and Summaries:
ALS Forum:
First Published on Clinicaltrials.gov: 8/29/2017
ClinicalTrials.gov ID: NCT03280056
Trial Protocol as Published on Clinicaltrials.gov:
ClinicalTrials.gov