Quick Info
Status
Currently Recruiting
Estimated Enrollment
200
Phase
3
Treatment Type
Biological: Autologous MSC-NTF cells - NurOwn
Trial Type
Randomized, placebo controlled, multidose
Primary Investigator
Merit E Cudkowicz, Robert H Brown, Anthony J. Windebank, MDNamit A Goyal, MD, Robert G Miller, MD
Contact Information
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
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
≥65% svc
Months Since Onset
Number of months since first symptoms of ALS.
<24 months
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
no
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
no
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
No
Update Notes
No significant updates.
7/8/2019
No significant updates
3/29/2019
No significant updates
11/29/2018
No significant updates
5/25/2018
All enrollment sites accepting patients
3/14/2018
Location updated
3/8/2018
Location updated
12/12/2017
Recruitment status at location updated
9/19/2017
Recruitment Status updated
9/13/2017
Trial added
7/31/2017

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
ALS Forum
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