Adenosine Deaminase Severe Combined Immune DeficiencyPhase 1/2NCT05432310

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

A Phase 1/2 study testing A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme in people with Adenosine Deaminase Severe Combined Immune Deficiency, run by University of California, Los Angeles at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial summary

The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. Read more about this study

Who is it for?: Ages 1 month and older
Conditions the study is for: Adenosine Deaminase Severe Combined Immune Deficiency
What is being tested?: A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 20 participants
Where: 1 siteUnited States
Study dates: 4 Jan 2023to 31 Dec 2027
Phase: Phase 1/2
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 1 month and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

All subjects must fulfill the following criteria to be included in the study:
Provision of written informed consent prior to any study related procedures. In this study consent must be provided by the parents/legal guardians and, where applicable according to local laws, a signed assent from the child,
Subjects ≥30 days of age,
With a diagnosis of ADA-SCID based on:
Evidence of ADA deficiency, defined as:
Decreased ADA enzymatic activity in erythrocytes, leukocytes, skin fibroblasts, or in cultured fetal cells to levels consistent with ADA-SCID as determined by the reference laboratory, or ii. Identified mutations in ADA alleles consistent with a severe reduction in ADA activity,

Show all 14Show fewer

Evidence of ADA-SCID based on either:
Family history of a first order relative with ADA deficiency and clinical and laboratory evidence of severe immunologic deficiency, or ii. Evidence of severe immunologic deficiency in subjects prior to the institution of immune restorative therapy, based on
Lymphopenia (absolute lymphocyte count (ALC) <400 cells/mL) OR absence or low number of T cells (absolute CD3+ count < 300 cells/mL), or
Severely decreased T lymphocyte blastogenic responses to phytohemagglutinin (either <10% of lower limit of normal controls for the diagnostic laboratory, or <10% of the response of the normal control of the day, or stimulation index <10), or
Identification of SCID by neonatal screening revealing low T Cell Receptor Excision Circles (TREC) levels.
Ineligible for matched family allogeneic bone marrow (BM) transplantation, defined as the absence of a medically eligible HLA-identical sibling or family donor, with normal immune function, who could serve as an allogeneic bone marrow donor.
Females of child-bearing age will be required to provide a negative pregnancy test 30 days prior to Visit 2.
Subjects and their parents/legal guardians must be willing and able to comply with study restrictions and to remain at the clinic for the required duration during the study period and willing to return to the clinic for the follow up evaluation as specified in the protocol.

Who cannot join

Subjects will not be eligible for the study if any of the following criteria is fulfilled:
Ineligible for autologous HSCT as per clinical site criteria
Other conditions which in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the mobilization of peripheral blood or the leukapheresis process, the administration of busulfan and the infusion of transduced cells, or which indicate an inability of the subject or subject's parent/legal guardian to comply with the protocol
Hematologic abnormality, defined as:
Anemia (Hb <8.0 g/dl).
Neutropenia (ANC <500/mm3). Note: ANC <500 with absence of myelodysplastic syndrome on bone marrow aspirate and biopsy and normal marrow cytogenetics are acceptable for eligibility.

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Thrombocytopenia (platelet count <50,000/mm3, at any age).
Prothrombin time or international normalized ratio (INR) and partial thromboplastin time (PTT) >2 x upper limit of normal (ULN) (subjects with a correctable deficiency controlled on medication will not be excluded).
Cytogenetic abnormalities on peripheral blood or bone marrow or amniotic fluid (if available).
Prior allogeneic HSCT with cytoreductive conditioning.
Pulmonary abnormality, defined as:
Resting O2 saturation by pulse oximetry <90% on room air.
Chest X-ray indicating active or progressive pulmonary disease. Note: Chest X ray indicating residual signs of treated pneumonitis is acceptable for eligibility.
Cardiac abnormality, defined as:
Abnormal ECG indicating cardiac pathology.
Uncorrected congenital cardiac malformation with clinical symptoms.
Active cardiac disease, including clinical evidence of congestive heart failure, cyanosis, hypotension.
Poor cardiac function as evidenced by left ventricular ejection fraction <40% on echocardiogram.
Neurologic abnormality, defined as:
Significant neurologic abnormality revealed by examination.
Uncontrolled seizure disorder.
Renal abnormality, defined as:
Renal insufficiency: serum creatinine ≥1.2 mg/dl (106 µmol/L), or ≥3+ proteinuria.
Abnormal serum sodium, potassium, calcium, magnesium or phosphate levels at >2 x ULN.
Hepatic/gastrointestinal abnormality, defined as:
Serum transaminases >5 x ULN.
Serum bilirubin >2 x ULN.
Serum glucose >1.5 x ULN.
Oncologic disease, defined as:
Evidence of active malignant disease other than dermatofibrosarcoma protuberans (DFSP).
Evidence of DFSP expected to require anti-neoplastic therapy within the 5 years following the infusion of genetically corrected cells (if anti-neoplastic therapy has been completed, a subject with a history of DFSP can be included).
Evidence of DFSP expected to be life limiting within the 5 years following the infusion of genetically corrected cells.
Known sensitivity to Busulfan.
Confirmation of an infectious disease by deoxyribonucleic acid (DNA) polymerase chain reaction (PCR) positive at time of assessment for the following:
HIV-1,
Hepatitis B,
Parvovirus B19.
The subject is pregnant or has a major congenital anomaly.
Is likely to require treatment during the study with drugs that are not permitted by the study protocol.
The subject has previously received another form of gene therapy.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of California, Los Angeles (UCLA)
Los Angeles, California, 90095

Other trials in United States

About this study

Official title: Efficacy and Safety of Cryopreserved Autologous Mobilized Peripheral Blood CD34+ HSPCs Transduced Ex Vivo With the EFS-ADA Lentiviral Vector in Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency

Read the detailed description

The study is open to twenty (20) infants and children diagnosed with ADA-deficient SCID who did not have a medically eligible, human leukocyte antigen (HLA)-identical sibling donor for bone marrow transplantation. The EFS-ADA lentiviral vector with the human ADA complementary DNA (cDNA) will be used to transduce autologous CD34+ cells from Granulocyte Colony Stimulating Factor (G-CSF)/Plerixafor mobilized Peripheral Blood (mPB) of these subjects. The subjects will receive pharmacokinetically-adjusted busulfan reduced intensity conditioning prior to re-infusion of their gene-modified cells. Overall survival at two years is the primary endpoint. During the follow-up phase, the investigators aim to determine whether the cells could engraft and produce mature cells that contain and express the corrected ADA gene in the absence of pegylated adenosine deaminase (PEG-ADA) enzyme replacement therapy (ERT), which will be withheld starting on Day +30 following transplant. Efficacy studies to evaluate the level of immune reconstitution, will be performed in the two years of the study. Patients will be asked to enroll into a long-term follow-up study to reach a total of 15 years follow-up after gene therapy.

Conditions: Adenosine Deaminase Severe Combined Immune Deficiency
Being tested: A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
Keywords: Gene Therapy, Hematopoietic Stem Cell, Lentiviral Vector, Reduced Intensity Conditioning with Busulfan
Medical subject headings: Severe combined immunodeficiency due to adenosine deaminase deficiency

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzymeCombination product
Autologous transplantation of EFS-ADA lentiviral vector transduced, mPB CD34+ cells by central venous infusion, following reduced intensity conditioning with busulfan

Study groups in NCT05432310
Study group	Role	What they receive
Autologous mobilized peripheral blood (mPB) transduced with EFS ADA lentiviral vector	Experimental	Evaluate safety and efficacy of this autologous gene therapy Receives: Combination Product: A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme

What is being measured

The primary outcome is the main question the study is designed to answer.

Survival
The primary study outcome will be to determine survival for all subjects 2 years after gene therapy
Measured: 24 months

Secondary outcomes (9)

Evaluate Safety from clinical adverse events.
Evaluate safety of the treatment by recording clinical adverse events (AE).
Measured: 24 months
Evaluate Safety from replication competent lentivirus by quantitative polymerase chain reaction (qPCR) assay.
Evaluate safety by recording incidents of replication competent lentivirus by qPCR assay..
Measured: 24 months
Evaluate Safety from vector-related clonal expansion by non-restrictive Linear Amplification Polymerase Chain Reaction (nrLAM-PCR)
Evaluate safety by recording incidence of vector-related clonal expansion by nrLAM-PCR
Measured: 24 months
Record event free survival at 24 months
Record Event Free Survival as a definition of "failure" of the therapy. Event-free survival is defined as the proportion of subjects alive with no "event", an "event" being the resumption of PEG-ADA ERT or the need for a rescue allogeneic hematopoietic stem cell transplant (HSCT), or death.
Measured: 24 months
Determine incidence of Infection over two years after gene therapy
Determine the incidence and severity of infections post-gene therapy (subsequent to hematopoietic reconstitution). Over 2 years, record the incidence of hospitalizations or outpatient-based treatments for systemic bacterial, fungal, or viral infections (including, but not limited to Cytomegalovirus (CMV) infections).
Measured: 24 months
Neuro-developmental Outcomes by neurodevelopmental testing (subjects 5-7 yeas of age)
Measure neuro-developmental status post-gene therapy. Perform age-appropriate neuro-developmental assessments testing (5- 7 years of age) at baseline and 2 years post-gene therapy - Wechsler Scale of Intelligence
Measured: 24 months
Neuro-developmental Outcomes by neurodevelopmental testing (subjects 1 year -42 month of age)
Measure neuro-developmental status post-gene therapy. Perform age-appropriate neuro-developmental assessments testing (1 year to 42 months of age) at baseline and 2 years post-gene therapy - : Bayley Scale of Infant Development
Measured: 24 months
Neuro-developmental Outcomes by Brain Stem Evoked Response (BAER) testing
Measure neuro-developmental status post-gene therapy - Perform Brainstem Auditory Evoked Response test at baseline and at 2 years.
Measured: 24 months
Cessation of immunoglobulin replacement therapy (IgRT).
Record time post-gene therapy that immunoglobulin replacement therapy (IgRT) is stopped based on defined criteria.
Measured: 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

4 Jan 202331 Dec 2027

First posted27 Jun 2022
Study start4 Jan 2023
Primary completion14 Nov 2027 (estimated)
Study completion31 Dec 2027 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Adenosine Deaminase Severe Combined Immune DeficiencyStudies of the same condition, open and under way.
More trials run by University of California, Los AngelesThe sponsor's other registered studies.
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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Prospective, non-randomized Phase I/II clinical trial to assess the safety and efficacy of gene therapy for ADA SCID by transplantation of autologous mPB CD34+ hematopoietic stem and progenitor cells (HSPC) transduced by the EFS-ADA lentiviral vector.
Masking: None (open label)
Enrolment: 20 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05432310
Sponsor's protocol ID: UCLA IRB#21-001814
Status last verified: Aug 2026
First submitted: 4 Jun 2022
Last updated: 20 Aug 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther. 2014 Mar;22(3):607-622. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20.
PubMed 24256635
Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11.
PubMed 33974366

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruiting
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
Enrolling by invitationObservational study
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Immunologic Deficiency Syndromes
Enrolling by invitationObservational study
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
Enrolling by invitationObservational study
Natural History Study of SCID Disorders
Severe Combined Immunodeficiency (SCID), Leaky SCID

All Adenosine Deaminase Severe Combined Immune Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Efficacy and Safety of Cryopreserved Autologous Mobilized Peripheral Blood CD34+ HSPCs Transduced Ex Vivo With the EFS-ADA Lentiviral Vector in Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency
ClinicalTrials.gov ID: NCT05432310
Acronym / sponsor ID: UCLA IRB#21-001814
Conditions: Adenosine Deaminase Severe Combined Immune Deficiency
Keywords: Gene Therapy, Hematopoietic Stem Cell, Lentiviral Vector, Reduced Intensity Conditioning with Busulfan
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Active, not recruiting
Enrolment: 20 participants (estimated)
Lead sponsor: University of California, Los AngelesUniversity, hospital or other
Study start: 4 Jan 2023 (actual)
Primary completion: 14 Nov 2027 (estimated)
Study completion: 31 Dec 2027 (estimated)
First posted: 27 Jun 2022 (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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Prospective, non-randomized Phase I/II clinical trial to assess the safety and efficacy of gene therapy for ADA SCID by transplantation of autologous mPB CD34+ hematopoietic stem and progenitor cells (HSPC) transduced by the EFS-ADA lentiviral vector.

Arm	Type	Receives
Autologous mobilized peripheral blood (mPB) transduced with EFS ADA lentiviral vector	Experimental	Combination Product: A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzymeEvaluate safety and efficacy of this autologous gene therapy

Who may qualify

Ages: 1 month and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (14 listed)

Provision of written informed consent prior to any study related procedures. In this study consent must be provided by the parents/legal guardians and, where applicable according to local laws, a signed assent from the child,
Subjects ≥30 days of age,
With a diagnosis of ADA-SCID based on:
Ineligible for matched family allogeneic bone marrow (BM) transplantation, defined as the absence of a medically eligible HLA-identical sibling or family donor, with normal immune function, who could serve as an allogeneic bone marrow donor.
Females of child-bearing age will be required to provide a negative pregnancy test 30 days prior to Visit 2.

Key exclusion criteria (40 listed)

Subjects will not be eligible for the study if any of the following criteria is fulfilled:
Ineligible for autologous HSCT as per clinical site criteria
Other conditions which in the opinion of the Principal Investigator and/or Co Investigators, contraindicate the mobilization of peripheral blood or the leukapheresis process, the administration of busulfan and the infusion of transduced cells, or which indicate an inability of the subject or subject's parent/legal guardian to comply with the protocol
Hematologic abnormality, defined as:
Pulmonary abnormality, defined as:

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
A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzymeAutologous transplantation of EFS-ADA lentiviral vector transduced, mPB CD34+ cells by central venous infusion, following reduced intensity conditioning with busulfan	Combination product	Autologous mobilized peripheral blood (mPB) transduced with EFS ADA lentiviral vector

Primary outcome

Survival
Time frame: 24 months
The primary study outcome will be to determine survival for all subjects 2 years after gene therapy

9 secondary outcomes

Evaluate Safety from clinical adverse events.
Time frame: 24 months
Evaluate Safety from replication competent lentivirus by quantitative polymerase chain reaction (qPCR) assay.
Time frame: 24 months
Evaluate Safety from vector-related clonal expansion by non-restrictive Linear Amplification Polymerase Chain Reaction (nrLAM-PCR)
Time frame: 24 months
Record event free survival at 24 months
Time frame: 24 months
Determine incidence of Infection over two years after gene therapy
Time frame: 24 months
Neuro-developmental Outcomes by neurodevelopmental testing (subjects 5-7 yeas of age)
Time frame: 24 months
Neuro-developmental Outcomes by neurodevelopmental testing (subjects 1 year -42 month of age)
Time frame: 24 months
Neuro-developmental Outcomes by Brain Stem Evoked Response (BAER) testing
Time frame: 24 months
Cessation of immunoglobulin replacement therapy (IgRT).
Time frame: 24 months

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 20 Aug 2026.
Background: PubMed 24256635Carbonaro DA, Zhang L, Jin X, Montiel-Equihua C, Geiger S, Carmo M, Cooper A, Fairbanks L, Kaufman ML, Sebire NJ, Hollis RP, Blundell MP, Senadheera S, Fu PY, Sahaghian A, Chan RY, Wang X, Cornetta K, Thrasher AJ, Kohn DB, Gaspar HB. Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency. Mol Ther. 2014 Mar;22(3):607-622. doi: 10.1038/mt.2013.265. Epub 2013 Nov 20.
Results publication: PubMed 33974366Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. N Engl J Med. 2021 May 27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11.
Individual participant dataSharing planned: No

Related trials for Adenosine Deaminase Severe Combined Immune Deficiency

Not yet recruiting
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome
Enrolling by invitationObservational study
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Immunologic Deficiency Syndromes
Enrolling by invitationObservational study
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
Enrolling by invitationObservational study
Natural History Study of SCID Disorders
Severe Combined Immunodeficiency (SCID), Leaky SCID

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.

Satiro De Oliveira, MD principal investigator

Assistant Professor

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 14 inclusion and 40 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: under way, not looking for participants, as of the sponsor's last update on 20 Aug 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Survival (time frame: 24 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05432310, which also lists 2 linked publications.

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 20 Aug 2026.