ALS - Amyotrophic Lateral SclerosisPhase 1NCT07838389
Evaluation of Donor-Derived B Cell Infusions in Patients With Amyotrophic Lateral Sclerosis (ALS)
A Phase 1 study testing B cell infusion in people with ALS - Amyotrophic Lateral Sclerosis, run by Mark Poznansky at 1 site in United States.
Trial summary
This study is testing whether giving people with ALS specially matched immune cells called B cells from a related donor is safe. Up to 10 participants will each receive two B cell infusions at least 60 days apart. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- ALS - Amyotrophic Lateral Sclerosis
- What is being tested?
- B cell infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2027to Jan 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of ALS that meets the revised El Escorial criteria for probable lab-supported, probable, or definite ALS.
- Age 18-70 years.
- Vital capacity of >65%.
- ALSFRS-R score of >30.
- Absence of clinically important abnormalities on screening labs, including but not limited to ALT/AST < 3x ULN, Bilirubin <1.5x ULN (unless there is documented history of Gilbert's disease), serum Cr < 1.5x ULN, WBC > 3.5, HCT >33, Plts > 120.
- Negative tests for HIV, HepB sAg, HCV.
- Available potential donor who must be a haploidentical match to the recipient and agree to be the B cell donor for this study.
- Note: Participants are eligible if they are taking any, all, or none of the following: riluzole, edaravone, and/or high-dose methylcobalamine; and the start date of these medications are at least 30 days prior to the baseline visit.
Who cannot join
- Unable or unwilling to comply with protocol requirements.
- Known current or history of recurrent bacterial, viral, fungal, mycobacterial, or other opportunistic infection.
- Use of tofersen (Qalsody).
- Use of Rituximab (Rituxan), Ocrelizumab (Ocrevus), Ofatumumab (Kesimpta, Arzerra), Epratuzumab (LymphoCide), Belimumab (Benlysta), MEDI-551, or other anti-CD20, anti-CD19, or anti-CD22 antibodies.
- Use of Atacicpet or other BlyS or APRIL inhibitors.
- Use of Chimeric antigen-receptor T cells targeting CD19 or CD20, including tisagenlecleucel (Kymriah), Aaxicabtagene ciloleucel (Yescarta), brexucabtagene autoleucel (Tecartus), and lisocabtagene maraleucel (Breyanzi).
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- Additional active neurological disease that could confound the participant's ALS symptoms.
- Major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks of screening.
- Immunization with live-attenuated vaccines within 4 weeks of screening. Study participant must agree to forgo live-attenuated vaccines throughout the study.
- Chronic immunosuppression (any chronic immunosuppressive immunotherapy including daily oral steroid use > 6 months).
- Other active autoimmune disease except psoriasis or hypothyroidism.
- Active malignancy (excluding skin cancer other than melanoma) within 5 years.
- History of any of the following: cardiac insufficiency (defined as New York Heart Association III/IV), unstable ischemic heart disease, uncontrolled cardiac arrhythmias, or uncontrolled hypertension (defined as systolic blood pressure >170 mmHg or diastolic blood pressure >110 mmHg).
- Unstable cardiac, renal, hepatic, endocrine, pulmonary or hematologic disease, at the discretion of the PI.
- Anything that, in the opinion of the PI, would place the participant at increased risk or preclude the participant's full compliance with or the completion of the study.
- Participant unwilling to practice contraception for the duration of the study. Men and women of childbearing potential (WOCBP) should use an approved method of birth control and agree to continue to use this method for the duration of the study.
- Acceptable methods of contraception include abstinence, female participants/partner's use of hormonal contraceptive (oral, implanted, or injected) in conjunction with a barrier method (WOCBP only) (e.g., diaphragm, cervical cap, male condom, and female condom and spermicidal foam, sponges, and film), female participant's/partner's use of an intrauterine device (IUD), or if the female participant/partner is surgically sterile (e.g., bilateral tubal ligation, hysterectomy) or two years postmenopausal at time of screening. All male participants/partners (excluding men who have been sterilized) must agree to consistently and correctly use a condom for duration of the study. In addition, participants may not donate sperm for the duration of the study.
- Participation in another interventional trial or use of an experimental ALS product with an ongoing clinical development program in an expanded access program, compassionate use program or through practitioner prescription. Over the counter medications are acceptable, as are standard of care ALS medications.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital
Boston, Massachusetts, 02114
Dario Gelevski, 617-726-0563, DGELEVSKI@mgh.harvard.edu
James D Berry, MD, MPH
About this study
Official title: Evaluation of Haploidentical, Donor-Derived B Lymphocyte Infusions in Patients With Amyotrophic Lateral Sclerosis
This study is testing whether giving people with ALS specially matched immune cells called B cells from a related donor is safe. Up to 10 participants will each receive two B cell infusions at least 60 days apart. The main goals are to watch closely for side effects, see whether the participant's immune system rejects the donor cells, and determine whether this treatment should be studied further in people with ALS. The first three participants will receive the infusions in the hospital with extra safety spacing between participants; if those infusions appear safe, the remaining participants may receive them in an outpatient setting.
Read the detailed description
This is a phase 1, non-randomized, open-label study to assess the safety and pharmacokinetics of up to 2 doses of donor B cells. Up to 10 recipients will each receive 2 infusions of donor B cells at doses of ≥ 2.0 x 10^8 and ≤ 1.0 x 10^9 donor B cells delivered at least 60 days apart. The first three participants will receive infusions as inpatients in a staggered fashion 30 days apart. After completion of a 30-day observation of the third participant after the second infusion, study investigators will determine whether the infusions are safe and well-tolerated and that there are no indications of significant immune rejection of the donor B cells. In such a case the remaining participants will receive 2 infusions in an outpatient setting with no mandatory staggering interval. Each participant will be followed for six months after the second infusion.
Donors. Each subject will be consented along with a relative meeting the HLA matching requirements to serve as a donor. The patient and potential donor will be HLA (A, B and DR- B1) tested and the potential donor will be a haploidentical match to the recipient, will have cleared all medical and infectious diseases screening tests, and will have agreed to be the B cell donor for this study. Each donor will undergo one or more leukapheresis procedures each yielding up to 1.5 mL of a concentrated white cell fraction. Purified B cells will be derived from these cells for a target final yield of between 2.0 x 108 and 1.0 x 109 B cells. Purified donor B cells will be stored in liquid nitrogen and thawed at bedside for infusion into participant.
Recipients. Prior to infusion, each enrolled subject will have a pre-infusion visit (study Day -50 to -40) where medical history will be taken, vitals and ALS-related measurements will be taken and a blood sample drawn to establish baseline blood counts, chemistry, and immunologic profile.
Treatment. All participants will receive up to 2 infusions of ≥ 2.0 x 10^8 and ≤ 1.0 x 10^9 donor B cells with the second infusion no less than 60 days after the first infusion. Participants will receive infusions with no preparatory treatment. Donor B cells in a concentration of 1.0 x 10^7 donor B cells/mL in a volume of up to 100 mL will be delivered over a 2-hour period through a peripheral line pre-incubated with a 4% solution of human serum albumin.
Evaluation. The first three participants will receive infusions as inpatients to carefully monitor responses to infusions for up to 24 hours. These participants will be staggered 30 days apart. If infusions of inpatient participants appear to be well tolerated and safe, the remaining participants will receive infusions as outpatients with no staggering.
Safety monitoring will include periodic measurement of vital signs and specific blood tests, monitoring of changes in medical history, reports of AEs, evaluation of ALS status through ALSFRS-R and grip strength, and assessment of Columbia Suicide Severity Rating Scale.
Blood tests will be taken periodically to monitor for changes in CBC, including ESR and CRP, and a metabolic panel that includes an expanded range of kidney function tests as well as ferritin. Study investigators will evaluate blood markers of immune rejection at time points up to the first four weeks after each infusion.
During the study, blood tests will be taken periodically to monitor other immunologic markers including flow cytometry using an established antibody panel developed for the previously-conducted expanded access studies.
- Conditions
- ALS - Amyotrophic Lateral Sclerosis
- Being tested
- B cell infusion
- Keywords
- cell therapy, B lymphocytes, immunotherapy, allogeneic 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.
B cell infusionBiological
infusion of haploidentical donor CD19+ B cells
| Study group | Role | What they receive |
|---|---|---|
| ALS patients receiving infusions of haploidentical donor B cells | Experimental | ALS patients receiving infusions of haploidentical donor B cells between 200 million and 1 billion cells per infusion with the second infusion 60 days after the first. Receives: Biological: B cell infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Infusion associated adverse events of special interest
Safety and tolerability of up to 2 infusions of donor B cells at individual doses of between 2.0 x 10^8 and 1.0 x 10^9 donor B cells defined by:
- Incidence of all AEs, as defined for the 30-day time point, at 60 days.
- Incidence of Treatment Emergent Serious Adverse Events (TE-SAEs) at 30 days defined as the composite of graft-versus-host disease, other persistent immunologic abnormalities, bleeding disorders, stroke, myocardial infarction, or death.
Measured: Up to 6 months after 2nd infusion
Evidence of host rejection of infused cells
Donor cell rejection: Indications of immune rejection of B cell infusions defined by changes in specific blood markers and ex vivo cell assays after either the first or second infusion of donor B cells compared to baseline levels of each.
Measured: Within 4 weeks of 2nd infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2026
- Study startSep 2027 (estimated)
- Primary completionJan 2030 (estimated)
- Study completionJan 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALS - 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mark Poznansky
- Sponsor type
- University, hospital or other
- Collaborators
- Alira Health, Dana-Farber Cancer Institute, Massachusetts Eye and Ear Infirmary
- Responsible party
- Sponsor-investigator, Mark Poznansky, Massachusetts General Hospital
- Investigators
- James D Berry, MD, MPH, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07838389
- Sponsor's protocol ID
- VICBcellALSTrial
- Status last verified
- Sep 2026
- First submitted
- 11 Sep 2026
- Last updated
- 24 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Haploidentical, Donor-Derived B Lymphocyte Infusions in Patients With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT07838389
- Acronym / sponsor ID
- VICBcellALSTrial
- Conditions
- ALS - Amyotrophic Lateral Sclerosis
- Keywords
- cell therapy, B lymphocytes, immunotherapy, allogeneic cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Mark PoznanskyUniversity, hospital or other
- Collaborators
- Alira Health, Dana-Farber Cancer Institute, Massachusetts Eye and Ear Infirmary
- Study start
- Sep 2027 (estimated)
- Primary completion
- Jan 2030 (estimated)
- Study completion
- Jan 2030 (estimated)
- First posted
- 24 Sep 2026 (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.
This study is testing whether giving people with ALS specially matched immune cells called B cells from a related donor is safe. Up to 10 participants will each receive two B cell infusions at least 60 days apart. The main goals are to watch closely for side effects, see whether the participant's immune system rejects the donor cells, and determine whether this treatment should be studied further in people with ALS. The first three participants will receive the infusions in the hospital with extra safety spacing between participants; if those infusions appear safe, the remaining participants may receive them in an outpatient setting.
Detailed description
This is a phase 1, non-randomized, open-label study to assess the safety and pharmacokinetics of up to 2 doses of donor B cells. Up to 10 recipients will each receive 2 infusions of donor B cells at doses of ≥ 2.0 x 10^8 and ≤ 1.0 x 10^9 donor B cells delivered at least 60 days apart. The first three participants will receive infusions as inpatients in a staggered fashion 30 days apart. After completion of a 30-day observation of the third participant after the second infusion, study investigators will determine whether the infusions are safe and well-tolerated and that there are no indications of significant immune rejection of the donor B cells. In such a case the remaining participants will receive 2 infusions in an outpatient setting with no mandatory staggering interval. Each participant will be followed for six months after the second infusion.
Donors. Each subject will be consented along with a relative meeting the HLA matching requirements to serve as a donor. The patient and potential donor will be HLA (A, B and DR- B1) tested and the potential donor will be a haploidentical match to the recipient, will have cleared all medical and infectious diseases screening tests, and will have agreed to be the B cell donor for this study. Each donor will undergo one or more leukapheresis procedures each yielding up to 1.5 mL of a concentrated white cell fraction. Purified B cells will be derived from these cells for a target final yield of between 2.0 x 108 and 1.0 x 109 B cells. Purified donor B cells will be stored in liquid nitrogen and thawed at bedside for infusion into participant.
Recipients. Prior to infusion, each enrolled subject will have a pre-infusion visit (study Day -50 to -40) where medical history will be taken, vitals and ALS-related measurements will be taken and a blood sample drawn to establish baseline blood counts, chemistry, and immunologic profile.
Treatment. All participants will receive up to 2 infusions of ≥ 2.0 x 10^8 and ≤ 1.0 x 10^9 donor B cells with the second infusion no less than 60 days after the first infusion. Participants will receive infusions with no preparatory treatment. Donor B cells in a concentration of 1.0 x 10^7 donor B cells/mL in a volume of up to 100 mL will be delivered over a 2-hour period through a peripheral line pre-incubated with a 4% solution of human serum albumin.
Evaluation. The first three participants will receive infusions as inpatients to carefully monitor responses to infusions for up to 24 hours. These participants will be staggered 30 days apart. If infusions of inpatient participants appear to be well tolerated and safe, the remaining participants will receive infusions as outpatients with no staggering.
Safety monitoring will include periodic measurement of vital signs and specific blood tests, monitoring of changes in medical history, reports of AEs, evaluation of ALS status through ALSFRS-R and grip strength, and assessment of Columbia Suicide Severity Rating Scale.
Blood tests will be taken periodically to monitor for changes in CBC, including ESR and CRP, and a metabolic panel that includes an expanded range of kidney function tests as well as ferritin. Study investigators will evaluate blood markers of immune rejection at time points up to the first four weeks after each infusion.
During the study, blood tests will be taken periodically to monitor other immunologic markers including flow cytometry using an established antibody panel developed for the previously-conducted expanded access studies.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALS patients receiving infusions of haploidentical donor B cells | Experimental | Biological: B cell infusionALS patients receiving infusions of haploidentical donor B cells between 200 million and 1 billion cells per infusion with the second infusion 60 days after the first. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Confirmed diagnosis of ALS that meets the revised El Escorial criteria for probable lab-supported, probable, or definite ALS.
- Age 18-70 years.
- Vital capacity of >65%.
- ALSFRS-R score of >30.
- Absence of clinically important abnormalities on screening labs, including but not limited to ALT/AST < 3x ULN, Bilirubin <1.5x ULN (unless there is documented history of Gilbert's disease), serum Cr < 1.5x ULN, WBC > 3.5, HCT >33, Plts > 120.
Key exclusion criteria (18 listed)
- Unable or unwilling to comply with protocol requirements.
- Known current or history of recurrent bacterial, viral, fungal, mycobacterial, or other opportunistic infection.
- Use of tofersen (Qalsody).
- Use of Rituximab (Rituxan), Ocrelizumab (Ocrevus), Ofatumumab (Kesimpta, Arzerra), Epratuzumab (LymphoCide), Belimumab (Benlysta), MEDI-551, or other anti-CD20, anti-CD19, or anti-CD22 antibodies.
- Use of Atacicpet or other BlyS or APRIL inhibitors.
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 |
|---|---|---|
| B cell infusioninfusion of haploidentical donor CD19+ B cells | Biological | ALS patients receiving infusions of haploidentical donor B cells |
Primary outcome
Infusion associated adverse events of special interest
Time frame: Up to 6 months after 2nd infusion
Safety and tolerability of up to 2 infusions of donor B cells at individual doses of between 2.0 x 10^8 and 1.0 x 10^9 donor B cells defined by: * Incidence of all AEs, as defined for the 30-day time point, at 60 days. * Incidence of Treatment Emergent Serious Adverse Events (TE-SAEs) at 30 days defined as the composite of graft-versus-host disease, other persistent immunologic abnormalities, bleeding disorders, stroke, myocardial infarction, or death.
Evidence of host rejection of infused cells
Time frame: Within 4 weeks of 2nd infusion
Donor cell rejection: Indications of immune rejection of B cell infusions defined by changes in specific blood markers and ex vivo cell assays after either the first or second infusion of donor B cells compared to baseline levels of each.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2026.
- Individual participant dataSharing planned: Undecided
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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.
James D Berry, MD, MPH principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing B cell infusion in people with ALS - Amyotrophic Lateral Sclerosis, run by Mark Poznansky at 1 site 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 8 inclusion and 18 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: opening soon, as of the sponsor's last update on 24 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mark C Poznansky, MD, PhD (617-724-6375, mpoznansky@mgb.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Infusion associated adverse events of special interest (time frame: Up to 6 months after 2nd infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
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 24 Sep 2026.