Amyotrophic Lateral SclerosisNCT06169176
RAPA-501 Therapy of ALS Expanded Access Protocol
A study testing RAPA-501 Autologous T stem cells in people with Amyotrophic Lateral Sclerosis, run by Rapa Therapeutics LLC at 10 sites in United States.
Trial summary
RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem cells in patients living with amyotrophic lateral sclerosis (pwALS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- RAPA-501 Autologous T stem cells
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 10 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients ≥ 18 years of age.
- Patients with sporadic or familial ALS diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria.
- Pulmonary slow vital capacity (SVC) < 50% of predicted normal (as measured within three months prior to screening or at the time of screening; inability to measure an SVC value at the time of screening that is due to severe reduction in respiratory function will also fulfill this eligibility criterion #3). However, SVC values ≥50% are acceptable if an EAP recipient of RAPA-501 is re-enrolled to the study.
- Must have a source of autologous T cells potentially sufficient to manufacture RAPA-501 cells, as defined by a peripheral CD3+ T cell count ≥ 500 cells per μl.
- Patients who are taking riluzole (Rilutek®), edaravone (Radicava®), and/or sodium phenylbutyrate/taurursodial (Relyvrio™) are eligible if taking the drug for at least 30 days prior to the screening visit.
- Patients must be ≥ two (2) weeks removed from major surgery, or investigational therapy.
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- Patients must have no ongoing, unstable serious illness other than ALS, as determined by the Site Investigator.
- Serum creatinine less than or equal to 2.0 mg/dL.
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x upper limit of normal (ULN).
- Bilirubin ≤ 1.5 (except if due to Gilbert's disease).
- No history of abnormal bleeding tendency.
- Voluntary written consent must be given before performance of any study related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to future care.
- Not enrolled in another interventional clinical trial or expanded access protocol and must have stopped taking other experimental drug(s) at least 2 weeks prior to screening.
Who cannot join
- Active uncontrolled infection.
- Hypertension not adequately controlled by ≤ 3 medications.
- History of documented pulmonary embolus within 6 months of enrollment.
- Clinically significant cardiac pathology, as defined by: myocardial infarction within 6 months prior to enrollment, Class III or IV heart failure according to NYHA, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
- HIV, hepatitis B, or hepatitis C seropositive.
- Pregnant or breastfeeding subjects.
- Subjects of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception.
- Subjects may be excluded at the Principal Investigator discretion or if it is deemed that allowing participation would represent an unacceptable medical or psychiatric risk.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Emory University
Atlanta, Georgia, 30322
Massachusetts General Hospital
Boston, Massachusetts, 02114
Hackensack University Medical Center
Hackensack, New Jersey, 07601
University of Iowa Health Care
Iowa City, Iowa, 52242
University of California Irvine Health
Irvine, California, 92868
University of Minnesota
Minneapolis, Minnesota, 55455
Barrow Neurological Institute
Phoenix, Arizona, 85013
Providence Portland Medical Center
Portland, Oregon, 97213
University of California, San Francisco
San Francisco, California, 94143
Mayo Clinic Hospital Phoenix
Scottsdale, Arizona, 85259
About this study
Official title: Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG /Th2 T Stem Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis
RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem cells in patients living with amyotrophic lateral sclerosis (pwALS).
Read the detailed description
ALS is a rare disease that is considered an orphan disease according to the Orphan Drug Act.
This is an open-label, non-randomized, multi-center intermediate size expanded access clinical trial of single-agent RAPA-501 T stem cells in patients with high-risk ALS who are not eligible for other ALS clinical trials.
After a subject consents to the study, an apheresis procedure will be performed to collect cells to manufacture the investigational product, RAPA-501 T stem cells. RAPA-501 cells are manufactured ex vivo using epigenetic reprogramming to yield a T stem cell population that is enriched for a dual anti-inflammatory phenotype based on hybrid TREG and Th2 differentiation. RAPA-501 T stem cells express both the TREG and Th2 transcription factors FOXP3 and GATA3, are enriched for expression of the ATP ectonucleotidase molecules CD39 and CD73, are enriched for the T cell homing molecule CD103, and suppress both effector T cell inflammatory molecules and CNS microglial cell inflammatory molecules.
This study is evaluating RAPA-501 T stem cell therapy at the dose of 80 x 10EE6 cells per infusion, with up to 4 infusions separated by six weeks between doses (infusion at time 0, and then after week 6, 12, and 18). Study subjects are then followed for several months to capture major clinical events and to assess survival. The total duration of RAPA-501 T stem cell therapy and follow-up interval on this protocol is approximately 8-months (250 days). The primary objective in the expanded access cohort is to determine the feasibility and safety of the highest-dose established safe dose of RAPA-501 (80 x 10EE6 cells per infusion). Secondary study objectives relate to assessment of overall survival compared to historical controls and determining the ALS disease activity pre-therapy, during study interventions, and throughout the post-therapy observation interval of RAPA-501 therapy through the monitoring of ALSFRS-R scores, SVC values, hand grip strength, and ROADS Scale. In addition, secondary study objectives relate to characterizing immune system parameters pre- and post- therapy and the potential effect of RAPA-501 therapy on serum markers of neurodegeneration.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- RAPA-501 Autologous T stem cells
- Keywords
- Amyotrophic Lateral Sclerosis, RAPA-501, ALS, Autologous TREG/Th2 T stem cell therapy, Rare Disease, Orphan Disease
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RAPA-501 Autologous T stem cellsOther
Experimental: Intermediate size expanded access cohort. Single-agent RAPA-501 T stem cells, (80 x 10EE6 cells per infusion; acceptable dose range of RAPA-501 T stem cells is 20-to-80 x 10EE6 cells per infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2023
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
- Rapa Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Massachusetts General Hospital, National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Sponsor
- Investigators
- Daniel H Fowler, M.D., Study director, Rapa Therapeutics LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06169176
- Sponsor's protocol ID
- RAPA-501-ALS-EAP
- Other IDs
- 1U01NS136020-01
- Status last verified
- Feb 2026
- First submitted
- 4 Dec 2023
- Last updated
- 12 Feb 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG /Th2 T Stem Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT06169176
- Acronym / sponsor ID
- RAPA-501-ALS-EAP
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Amyotrophic Lateral Sclerosis, RAPA-501, ALS, Autologous TREG/Th2 T stem cell therapy, Rare Disease, Orphan Disease
- Study type
- Expanded access
- Recruitment status
- Temporarily Not Available
- Lead sponsor
- Rapa Therapeutics LLCIndustry (pharma, biotech, devices)
- Collaborators
- Massachusetts General Hospital, National Institute of Neurological Disorders and Stroke (NINDS)
- First posted
- 13 Dec 2023 (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.
RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem cells in patients living with amyotrophic lateral sclerosis (pwALS).
Detailed description
ALS is a rare disease that is considered an orphan disease according to the Orphan Drug Act.
This is an open-label, non-randomized, multi-center intermediate size expanded access clinical trial of single-agent RAPA-501 T stem cells in patients with high-risk ALS who are not eligible for other ALS clinical trials.
After a subject consents to the study, an apheresis procedure will be performed to collect cells to manufacture the investigational product, RAPA-501 T stem cells. RAPA-501 cells are manufactured ex vivo using epigenetic reprogramming to yield a T stem cell population that is enriched for a dual anti-inflammatory phenotype based on hybrid TREG and Th2 differentiation. RAPA-501 T stem cells express both the TREG and Th2 transcription factors FOXP3 and GATA3, are enriched for expression of the ATP ectonucleotidase molecules CD39 and CD73, are enriched for the T cell homing molecule CD103, and suppress both effector T cell inflammatory molecules and CNS microglial cell inflammatory molecules.
This study is evaluating RAPA-501 T stem cell therapy at the dose of 80 x 10EE6 cells per infusion, with up to 4 infusions separated by six weeks between doses (infusion at time 0, and then after week 6, 12, and 18). Study subjects are then followed for several months to capture major clinical events and to assess survival. The total duration of RAPA-501 T stem cell therapy and follow-up interval on this protocol is approximately 8-months (250 days). The primary objective in the expanded access cohort is to determine the feasibility and safety of the highest-dose established safe dose of RAPA-501 (80 x 10EE6 cells per infusion). Secondary study objectives relate to assessment of overall survival compared to historical controls and determining the ALS disease activity pre-therapy, during study interventions, and throughout the post-therapy observation interval of RAPA-501 therapy through the monitoring of ALSFRS-R scores, SVC values, hand grip strength, and ROADS Scale. In addition, secondary study objectives relate to characterizing immune system parameters pre- and post- therapy and the potential effect of RAPA-501 therapy on serum markers of neurodegeneration.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Male or female patients ≥ 18 years of age.
- Patients with sporadic or familial ALS diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria.
- Pulmonary slow vital capacity (SVC) < 50% of predicted normal (as measured within three months prior to screening or at the time of screening; inability to measure an SVC value at the time of screening that is due to severe reduction in respiratory function will also fulfill this eligibility criterion #3). However, SVC values ≥50% are acceptable if an EAP recipient of RAPA-501 is re-enrolled to the study.
- Must have a source of autologous T cells potentially sufficient to manufacture RAPA-501 cells, as defined by a peripheral CD3+ T cell count ≥ 500 cells per μl.
- Patients who are taking riluzole (Rilutek®), edaravone (Radicava®), and/or sodium phenylbutyrate/taurursodial (Relyvrio™) are eligible if taking the drug for at least 30 days prior to the screening visit.
Key exclusion criteria (8 listed)
- Active uncontrolled infection.
- Hypertension not adequately controlled by ≤ 3 medications.
- History of documented pulmonary embolus within 6 months of enrollment.
- Clinically significant cardiac pathology, as defined by: myocardial infarction within 6 months prior to enrollment, Class III or IV heart failure according to NYHA, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
- HIV, hepatitis B, or hepatitis C seropositive.
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 |
|---|---|---|
| RAPA-501 Autologous T stem cellsExperimental: Intermediate size expanded access cohort. Single-agent RAPA-501 T stem cells, (80 x 10EE6 cells per infusion; acceptable dose range of RAPA-501 T stem cells is 20-to-80 x 10EE6 cells per infusion. | Other | – |
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 12 Feb 2026.
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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.
Daniel H Fowler, M.D. study director
Rapa Therapeutics LLC
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing RAPA-501 Autologous T stem cells in people with Amyotrophic Lateral Sclerosis, run by Rapa Therapeutics LLC at 10 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 13 inclusion and 8 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 12 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 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 12 Feb 2026.