Amyotrophic Lateral SclerosisNCT04681118
Expanded Access Protocol: Repeated Administration of Nurown® (Autologous MSC-NTF Cells) for the Treatment of ALS
A study testing NurOwn (MSC-NTF cells) in people with Amyotrophic Lateral Sclerosis, run by Brainstorm-Cell Therapeutics at 5 sites in United States.
Trial summary
Expanded Access for treatment with investigational product MSC-NTF cells(NurOwn®) for participants who completed all scheduled treatments and follow-up assessments in the BCT-002-US study Read more about this study
- Who is it for?
- Ages 18 to 63 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- NurOwn (MSC-NTF cells)
- How many people
- Not given
- Where
- 5 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 63 years
- All sexes
- Adult
Who may be able to join
- 1. Participation in the BCT-002-US study, successful completion of all scheduled treatments and follow-up assessments.
- 2 Able to provide to the Investigator written informed consent regarding the investigational drug, or, as applicable, on whose behalf a legally authorized representative of the participant has provided such consent.
- 3. Able to safely undergo all study procedures.
Who cannot join
- Inability to lie flat for the duration of intrathecal cell transplantation and/or bone marrow biopsy, or inability to tolerate treatment procedures for any other reason (lying flat with BiPAP or NIV are not exclusionary).
- History of clinically significant autoimmune disease (excluding thyroid disease) myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.
- Any unstable clinically significant medical condition other than ALS (e.g., within six months of baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure), treatment with anticoagulants that, in the opinion of the Investigator, would compromise the safety of the participant.
- Any history of malignancy within the previous 5 years, with the exception of non- melanoma localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).
- Current use of immunosuppressant medication or use of such medication within 4 weeks of the pre-treatment visit.
- Any history of acquired or inherited immune deficiency syndrome.
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- Tracheostomy and/or mechanical ventilation. Feeding tube use, BiPAP or NIV are not exclusionary.
- Pregnant women or women currently breastfeeding or unwilling to use effective birth control methods during the treatment (if of childbearing age).
- Positive test result for Hepatitis B virus (HBV; surface antigen (HBsAg) and IgM antibodies to core antigen (IgM anti-HBc)), Hepatitis C virus (HCV), Human Immune deficiency virus (HIV) 1 and 2.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Massachusetts General Hospital
Boston, Massachusetts, 02115
University of California Irvine Alpha Stem Cell Clinic
Irvine, California, 92697
Mayo Clinic
Rochester, Minnesota, 55905
California Pacific Medical Center
San Francisco, California, 94115
UMass Medical School
Worcester, Massachusetts, 01655
About this study
Official title: Intermediate-size Patient Population Expanded Access Protocol: Repeated Administration of Nurown® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
Expanded Access for treatment with investigational product MSC-NTF cells(NurOwn®) for participants who completed all scheduled treatments and follow-up assessments in the BCT-002-US study
Read the detailed description
This is an Intermediate-size patient population Expanded Access Protocol (EAP) to provide NurOwn® (MSC-NTF cells) for eligible ALS participants who completed the BCT-002-US phase 3 ALS clinical trial and have received all three IT treatments.
The participants will undergo up to three intrathecal (IT) treatments with NurOwn® (MSC-NTF cells).
In the first treatment period, the participant received up to three intrathecal (IT) treatments with NurOwn® (MSC-NTF cells) at T1, T2 and T3 and was followed for 12 weeks after the third treatment.
In the second treatment period, the participant will receive up to three intrathecal (IT) treatments with NurOwn® (MSC-NTF cells) every 8 weeks at T4, T5 and T6.
Following the last EAP treatment, the participant will be followed for three additional monthly visits (in-person, if feasible, or by telephone call or telemedicine in consideration of the ongoing COVID-19 pandemic) through the final protocol visit, during which the ALSFRS-R and safety assessments will be collected
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- NurOwn (MSC-NTF cells)
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NurOwn (MSC-NTF cells)Biological
Intrathecal administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Brainstorm-Cell Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04681118
- Sponsor's protocol ID
- BCT-003-US
- Status last verified
- Apr 2024
- First submitted
- 18 Dec 2020
- Last updated
- 23 Apr 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intermediate-size Patient Population Expanded Access Protocol: Repeated Administration of Nurown® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT04681118
- Acronym / sponsor ID
- BCT-003-US
- Conditions
- Amyotrophic Lateral Sclerosis
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Brainstorm-Cell TherapeuticsIndustry (pharma, biotech, devices)
- First posted
- 23 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Expanded Access for treatment with investigational product MSC-NTF cells(NurOwn®) for participants who completed all scheduled treatments and follow-up assessments in the BCT-002-US study
Detailed description
This is an Intermediate-size patient population Expanded Access Protocol (EAP) to provide NurOwn® (MSC-NTF cells) for eligible ALS participants who completed the BCT-002-US phase 3 ALS clinical trial and have received all three IT treatments.
The participants will undergo up to three intrathecal (IT) treatments with NurOwn® (MSC-NTF cells).
In the first treatment period, the participant received up to three intrathecal (IT) treatments with NurOwn® (MSC-NTF cells) at T1, T2 and T3 and was followed for 12 weeks after the third treatment.
In the second treatment period, the participant will receive up to three intrathecal (IT) treatments with NurOwn® (MSC-NTF cells) every 8 weeks at T4, T5 and T6.
Following the last EAP treatment, the participant will be followed for three additional monthly visits (in-person, if feasible, or by telephone call or telemedicine in consideration of the ongoing COVID-19 pandemic) through the final protocol visit, during which the ALSFRS-R and safety assessments will be collected
Study design
Who may qualify
- Ages
- 18 to 63 years
- Sex
- All
- Age groups
- Adult
Key inclusion criteria (3 listed)
- 1. Participation in the BCT-002-US study, successful completion of all scheduled treatments and follow-up assessments.
- 2 Able to provide to the Investigator written informed consent regarding the investigational drug, or, as applicable, on whose behalf a legally authorized representative of the participant has provided such consent.
- 3. Able to safely undergo all study procedures.
Key exclusion criteria (9 listed)
- Inability to lie flat for the duration of intrathecal cell transplantation and/or bone marrow biopsy, or inability to tolerate treatment procedures for any other reason (lying flat with BiPAP or NIV are not exclusionary).
- History of clinically significant autoimmune disease (excluding thyroid disease) myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.
- Any unstable clinically significant medical condition other than ALS (e.g., within six months of baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure), treatment with anticoagulants that, in the opinion of the Investigator, would compromise the safety of the participant.
- Any history of malignancy within the previous 5 years, with the exception of non- melanoma localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).
- Current use of immunosuppressant medication or use of such medication within 4 weeks of the pre-treatment visit.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NurOwn (MSC-NTF cells)Intrathecal administration | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing NurOwn (MSC-NTF cells) in people with Amyotrophic Lateral Sclerosis, run by Brainstorm-Cell Therapeutics at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Apr 2024.