Aplastic AnemiaPhase 2NCT04558736

Haploidentical HCT for Severe Aplastic Anemia

A Phase 2 study testing Anti-Thymocyte Globulin (Rabbit), Fludarabine and Cyclophosphamide in people with Aplastic Anemia and Bone Marrow Failure Syndrome, run by St. Jude Children's Research Hospital at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts

Official record

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Trial summary

Who is it for?: Ages Up to 21 years
Conditions the study is for: Aplastic Anemia, Bone Marrow Failure Syndrome
What is being tested?: Anti-Thymocyte Globulin (Rabbit), Fludarabine and Cyclophosphamide
Type of treatment: Injection or infusion, Medical device, Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 21 participants
Where: 1 siteUnited States
Study dates: 21 Jan 2021to 1 Jul 2030
Phase: Phase 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 Up to 21 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

Age less than or equal to 21 years at time of enrollment.
Confirmed diagnosis of SAA or a single lineage cytopenia
SAA or single lineage cytopenia will be defined as follows:
i. Bone marrow cellularity < 25% or hypocellular marrow for age, AND
ii. One or more of the following (in peripheral blood): (i) Neutrophils < 0.5 x10^9/L (ii) Platelets < 20 x10^9/L, or platelet transfusion dependence (iii) Hemoglobin <8g/dL, or red blood cell transfusion dependence
Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10/10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation.

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Failed at least one trial of immunosuppressive therapy (IST) by being refractory (persistence of severe cytopenias and fulfillment of SAA disease criteria at least 3 months after initial IST) or having relapsed (initial improvement of cytopenias after first-line IST but then a later return to fulfillment of SAA disease criteria when IST is decreased or ceased). IST could have included ATG based regimens, calcineurin inhibitors and/or other higher dose therapy directed at the treatment of primary SAA. Patients with very severe aplastic anemia who are likely not to benefit from IST do not need to have failed a trial of IST and can proceed directly to HCT if they meet the rest of the criteria.
Has a suitable single haplotype matched (≥ 3 of 6) family member donor.
Patient and/or legal guardian must sign informed consent for HCT.
Adequate organ function defined as:
Left ventricular ejection fraction > 40% or shortening fraction ≥ 25%.
Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml/ min/1.73m2.
Forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry
≥ 92% on room air if patient is unable to perform pulmonary function testing.
Karnofsky or Lansky (age-dependent) performance score ≥ 50.
Bilirubin ≤ 3 times the upper limit of normal for age.
Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age.
Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence until after the last dose of chemotherapy has been administered
At least single haplotype matched (≥ 3 of 6) family member.
At least 18 years of age.
HIV negative.
Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).
Not breast feeding.
Related donors must be ruled out for telomere disease by appropriate clinical and diagnostic measures (for example, clinical evaluation, telomere length testing, genetic testing, and/or bone marrow examination).
The HAPLO donor and/or legal guardian must be able to sign informed consent documents.
The potential HAPLO donor must be willing and able to donate PBSCs.

Who cannot join

Diagnosis of Fanconi anemia. Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing.
Known clinical or genetic diagnosis of dyskeratosis congenita
Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre- MDS) or MDS on marrow examination (e.g. Monosomy 7).
Diagnosis of myelodysplastic syndrome (MDS).
Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement- dependent cytotoxicity or flow cytometric testing or the presence of anti- donor HLA antibody to the high expression loci HLA-A, B, C, DRB1, or DPB1 with mean fluorescence intensity (MFI) > 1000 by solid phase immunoassay).
Prior allogeneic hematopoietic cell transplant.

Show all 12Show fewer

Prior solid organ transplant.
Known life-threatening reaction (i.e., anaphylaxis) to ATG that would prohibit use for the patient.
Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as progression or no clinical improvement on appropriate medical treatment.
Female patients who are pregnant (per institutional practice) or breast- feeding.
Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent > 5 years previously will be allowed. Cancer treated with curative intent ≤ 5 years previously will not be allowed unless approved by the PI.
Alemtuzumab or ATG within 2 weeks of enrollment.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Amr Qudeimat, MD contact

866-278-5833

referralinfo@stjude.org

United States1 site, 1 recruiting

St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Amr Qudeimat, MD, 866-278-5833, referralinfo@stjude.org
Amr Qudeimat, MD
Recruiting

Other trials in United States

About this study

Official title: Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia

This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year post-hematopoietic cell transplantation (HCT).

Primary Objectives

To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.
To estimate the overall survival and event free survival at 1-year post transplantation.

Secondary Objectives

To calculate the incidence of acute and chronic GVHD after HCT.
To calculate the rate of secondary graft rejection at 1-year post transplantation
To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus).
To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year.

Exploratory Objectives

To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA.
To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT.
To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation.
To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI.
Quantify donor derived Treg cells at different time points in patients received HCT.
Determine Treg activation status at different stages after HCT.
Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant?
How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient?
Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?

Read the detailed description

Immunosuppressant therapy (IST) is the main treatment for SAA for patients who do not have an HLA-matched sibling donor available for transplant. But some patients with SAA do not respond to IST and some others relapse after IST. HCT using an unrelated but HLA-matched donor is the only curative option for these patients but many patients lack a suitable HLA-matched donor. St Jude is trying to increase donor options for these patients by using novel therapeutic strategies by combining two widely used of GVHD prophylaxis methods: i) selective T cell depletion and ii) use of post-transplant cyclophosphamide. This will allow expansion of the donor pool to include haploidentical donors as well as reduce the risk of GVHD. The goal of this protocol is to test whether combining these GVHD prophylaxis approaches will allow use of haploidentical donors, reduce risk of GVHD, reduce transfusion dependence and improve immune reconstitution.

For this study chemotherapy, antibodies and radiation will be given to prepare the body to receive donor cells. Participants will then be given the donor cell infusion.

Patients will receive two types of donor blood cell products - a progenitor blood cell infusion and then a donor lymphocyte infusion. Both the progenitor blood cell and the donor lymphocyte infusion will be processed in a laboratory at St. Jude using a machine called the CliniMACS™.

In this clinical trial, participants will receive a special type of progenitor blood cells (called TCRαβ- depleted blood cells) from the donor.

After the donor progenitor cells have started to grow within the body (engraftment), participants will receive a second product that contains mature immune cells. These immune cells called CD45RA-depleted lymphocytes or donor lymphocyte infusion (DLI) will help fight infections in the body after the transplant and strengthen the developing immune system.

Conditions: Aplastic Anemia, Bone Marrow Failure Syndrome
Being tested: Anti-Thymocyte Globulin (Rabbit), Fludarabine, Cyclophosphamide, Mesna, G-CSF
Keywords: Haploidentical Transplant
Medical subject headings: Anemia, Aplastic, Bone Marrow Failure Disorders

What participants receive

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

Anti-Thymocyte Globulin (Rabbit)Drug
Also called thymoglobulin, rabbit ATG
Given intravenously (IV)
FludarabineDrug
Also called Fludara Flu Dara
Given intravenously (IV)
CyclophosphamideDrug
Also called CYTOXAN
Given intravenously (IV)
MesnaDrug
Also called MESNEX
Given intravenously (IV)
G-CSFDrug
Also called Neupogen, Filgrastim
Filgrastim is a human granulocyte colony-stimulating factor (G-CSF), produced by recombinant DNA technology. Dosage and Route of Administration: 5mcg/kg subcutaneous or intravenous daily until ANC >2000 for 2 consecutive days, or as clinically indicated
Total Lymphoid Irradiation (TLI)Radiation
Also called TLI
TLI will be given at 800 cGy total dose in 4 fractions.
CliniMACSDevice
Also called Cell Selection System
The mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest, such as CD3+ human T cells.
HPC, A InfusionBiological
Given intravenously Day 0-HPC, A Infusion (TCR αβ+/CD19+-depleted graft)
CD45RA-depleted DLIBiological
CD45RA-depleted DLI will be given at least ONE week after engraftment

Study groups in NCT04558736
Study group	Role	What they receive
Haploidentical HCT	Experimental	To assess the safety and efficacy of haploidentical donor transplantation for patients with severe aplastic anemia who lack an available HLA-matched donor. The goal of this study is to develop a novel, reduced-toxicity, post-transplant pharmacologic immunosuppression (GVHD prophylaxis)- free, highly tolerogenic haploidentical transplant regimen that is associated with few post- transplant complications or late toxicities and is available promptly to all patients, irrespective of matched donor availability. Cells for infusion are prepared using the CliniMACS System. Receives: Drug: Anti-Thymocyte Globulin (Rabbit); Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Mesna; Drug: G-CSF; Radiation: Total Lymphoid Irradiation (TLI); Device: CliniMACS; Biological: HPC, A Infusion; Biological: CD45RA-depleted DLI

What is being measured

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

Engraftment
Rate of patients engrafting at day 30 after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.
Measured: 30 days
Overall and event free survival
Rate of overall survival and event free survival at 1-year post transplantation.
Measured: 1 year

Secondary outcomes (4)

Graft vs host disease
Incidence of acute and chronic GVHD after hematopoietic cell transplant
Measured: 1 year
Graft rejection
Rate of secondary graft rejection at 1-year post transplantation
Measured: 1 year
Viral reactivation
Cumulative incidence of viral reactivation post-transplant (CMV, EBV and adenovirus)
Measured: 1 year
Immune reconstitution
We will record immune reconstitution parameters, including the order and magnitude of recovery of the different subtypes of leukocytes. Results will be summarized by descriptive statistics. The pattern of immune reconstitution will be evaluated using longitudinal approaches such as mixed effect models or generalized estimating equation (GEE) approach.
Measured: 1 year

Timeline

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

21 Jan 20211 Jul 2030

First posted22 Sep 2020
Study start21 Jan 2021
Primary completion1 Jul 2029 (estimated)
Study completion1 Jul 2030 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
More trials run by St. Jude Children's Research HospitalThe 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 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 21 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04558736
Sponsor's protocol ID: HAPSAA
Status last verified: Sep 2026
First submitted: 9 Sep 2020
Last updated: 16 Sep 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: Yes
Individual data sharing: Yes: Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.

Interested in taking part?

Amr Qudeimat, MD

866-278-5833

referralinfo@stjude.org

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 sites and contacts

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All Aplastic Anemia trials

Study at a glance

From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia
ClinicalTrials.gov ID: NCT04558736
Acronym / sponsor ID: HAPSAA
Conditions: Aplastic Anemia, Bone Marrow Failure Syndrome
Keywords: Haploidentical Transplant
Phase: Phase 2
Study type: Interventional
Recruitment status: Recruiting
Enrolment: 21 participants (estimated)
Lead sponsor: St. Jude Children's Research HospitalUniversity, hospital or other
Study start: 21 Jan 2021 (actual)
Primary completion: 1 Jul 2029 (estimated)
Study completion: 1 Jul 2030 (estimated)
First posted: 22 Sep 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.

Primary Objectives

To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.
To estimate the overall survival and event free survival at 1-year post transplantation.

Secondary Objectives

To calculate the incidence of acute and chronic GVHD after HCT.
To calculate the rate of secondary graft rejection at 1-year post transplantation
To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus).
To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year.

Exploratory Objectives

To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA.
To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT.
To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation.
To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI.
Quantify donor derived Treg cells at different time points in patients received HCT.
Determine Treg activation status at different stages after HCT.
Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant?
How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient?
Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?

Detailed description

For this study chemotherapy, antibodies and radiation will be given to prepare the body to receive donor cells. Participants will then be given the donor cell infusion.

In this clinical trial, participants will receive a special type of progenitor blood cells (called TCRαβ- depleted blood cells) from the donor.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Haploidentical HCT	Experimental	Drug: Anti-Thymocyte Globulin (Rabbit), Drug: Fludarabine, Drug: Cyclophosphamide, Drug: Mesna, Drug: G-CSF, Radiation: Total Lymphoid Irradiation (TLI), Device: CliniMACS, Biological: HPC, A Infusion, Biological: CD45RA-depleted DLITo assess the safety and efficacy of haploidentical donor transplantation for patients with severe aplastic anemia who lack an available HLA-matched donor. The goal of this study is to develop a novel, reduced-toxicity, post-transplant pharmacologic immunosuppression (GVHD prophylaxis)- free, highly tolerogenic haploidentical transplant regimen that is associated with few post- transplant complications or late toxicities and is available promptly to all patients, irrespective of matched donor availability. Cells for infusion are prepared using the CliniMACS System.

Who may qualify

Ages: Up to 21 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (26 listed)

Age less than or equal to 21 years at time of enrollment.
Confirmed diagnosis of SAA or a single lineage cytopenia
Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10/10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation.
Failed at least one trial of immunosuppressive therapy (IST) by being refractory (persistence of severe cytopenias and fulfillment of SAA disease criteria at least 3 months after initial IST) or having relapsed (initial improvement of cytopenias after first-line IST but then a later return to fulfillment of SAA disease criteria when IST is decreased or ceased). IST could have included ATG based regimens, calcineurin inhibitors and/or other higher dose therapy directed at the treatment of primary SAA. Patients with very severe aplastic anemia who are likely not to benefit from IST do not need to have failed a trial of IST and can proceed directly to HCT if they meet the rest of the criteria.
Has a suitable single haplotype matched (≥ 3 of 6) family member donor.

Key exclusion criteria (12 listed)

Diagnosis of Fanconi anemia. Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing.
Known clinical or genetic diagnosis of dyskeratosis congenita
Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre- MDS) or MDS on marrow examination (e.g. Monosomy 7).
Diagnosis of myelodysplastic syndrome (MDS).
Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement- dependent cytotoxicity or flow cytometric testing or the presence of anti- donor HLA antibody to the high expression loci HLA-A, B, C, DRB1, or DPB1 with mean fluorescence intensity (MFI) > 1000 by solid phase immunoassay).

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
Anti-Thymocyte Globulin (Rabbit)Also: thymoglobulin, rabbit ATGGiven intravenously (IV)	Drug	Haploidentical HCT
FludarabineAlso: Fludara Flu DaraGiven intravenously (IV)	Drug	Haploidentical HCT
CyclophosphamideAlso: CYTOXANGiven intravenously (IV)	Drug	Haploidentical HCT
MesnaAlso: MESNEXGiven intravenously (IV)	Drug	Haploidentical HCT
G-CSFAlso: Neupogen, FilgrastimFilgrastim is a human granulocyte colony-stimulating factor (G-CSF), produced by recombinant DNA technology. Dosage and Route of Administration: 5mcg/kg subcutaneous or intravenous daily until ANC >2000 for 2 consecutive days, or as clinically indicated	Drug	Haploidentical HCT
Total Lymphoid Irradiation (TLI)Also: TLITLI will be given at 800 cGy total dose in 4 fractions.	Radiation	Haploidentical HCT
CliniMACSAlso: Cell Selection SystemThe mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest, such as CD3+ human T cells.	Device	Haploidentical HCT
HPC, A InfusionGiven intravenously Day 0-HPC, A Infusion (TCR αβ+/CD19+-depleted graft)	Biological	Haploidentical HCT
CD45RA-depleted DLICD45RA-depleted DLI will be given at least ONE week after engraftment	Biological	Haploidentical HCT

Primary outcome

Engraftment
Time frame: 30 days
Rate of patients engrafting at day 30 after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.
Overall and event free survival
Time frame: 1 year
Rate of overall survival and event free survival at 1-year post transplantation.

4 secondary outcomes

Graft vs host disease
Time frame: 1 year
Graft rejection
Time frame: 1 year
Viral reactivation
Time frame: 1 year
Immune reconstitution
Time frame: 1 year

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 16 Sep 2026.
St. Jude Children's Research HospitalLink provided by the sponsor.
Clinical Trials Open at St. JudeLink provided by the sponsor.
Individual participant dataSharing planned: Yes. Individual participant de-identified datasets containing the variables analyzed in the published article will be made available (related to the study primary or secondary objectives contained in the publication). Supporting documents such as the protocol, statistical analyses plan, and informed consent are available through the CTG website for the specific study. Data used to generate the published article will be made available at the time of article publication. Investigators who seek access to individual level de-identified data will contact the computing team in the Department of Biostatistics (ClinTrialDataRequest@stjude.org) who will respond to the data request.

Related trials for Aplastic Anemia

Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
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Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
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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.

Amr Qudeimat, MD contact

866-278-5833

referralinfo@stjude.org

Amr Qudeimat, MD principal investigator

St. Jude Children's Research Hospital

Akshay Sharma, MBBS principal investigator

St. Jude Children's Research Hospital

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 26 inclusion and 12 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: looking for participants now, as of the sponsor's last update on 16 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 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?

Contact Amr Qudeimat, MD (866-278-5833, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Engraftment (time frame: 30 days). 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?

On the ClinicalTrials.gov record for NCT04558736.

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 16 Sep 2026.