NCT05757310|RecruitingPhase 1DMCFDA Drug

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

A Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Severe Aplastic Anemia Patients With COH-MC-17: A Non-Myeloablative/ Reduced-Intensity, Conditioning Regimen and CD4+ T-Cell-Depleted Haploidentical Hematopoietic Transplant

City of Hope Medical Center ClinicalTrials.gov

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Other study identifiers

Sponsor study identifier

23139

Secondary identifiers

NCI-2022-10251

REGISTRY · CTRP (Clinical Trial Reporting Program)

23139

OTHER · City of Hope Medical Center

P30CA033572

NIH

Study Type

interventional

Estimated enrollment

Locations

1 country

Sites

Timeline

Study Start

November 15, 2024

Actual (reported)

Primary Completion

June 18, 2027

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Study Completion

June 18, 2027

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Brief Summary

This phase I trial evaluates the safety and feasibility of using a reduced-intensity regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin prior to a CD4+ T-cell depleted haploidentical hematopoietic cell transplant (haploHCT) for the treatment of patients with severe aplastic anemia that does not respond to treatment (refractory) or that has come back (recurrent). Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid. It may also lower the body's immune response. Pentostatin blocks a protein needed for cell growth. Anti-thymocyte globulin is an immunosuppressive drug can destroy immune cells known as T-cells. HaploHCT transfers blood-forming stem cells from a healthy partially-matched donor to a patient. Administering a regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin before haploHCT may help make room for the new, healthy cells and may reduce the risk of graft versus host disease.

Registry Overview

Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.

Recruitment status: recruiting
Estimated enrollment: 6 participants
Study completion date: June 18, 2027
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.
Listed locations: 1 countries · 1 sites

Trial Relationships

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Some registry-reported names shown below are not yet linked in this graph.

Study Timeline

Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.

First Submitted
January 10, 2023
Actual (reported)
First Posted
March 7, 2023
Actual (reported)
Study Start
November 15, 2024
Actual (reported)
Primary Completion
June 18, 2027
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
June 18, 2027
Estimated
Planned date reported by the registry; this milestone is not confirmed as achieved.

Last Updated: January 5, 2026
Status Verified: December 2025
Last Update Submitted: December 31, 2025

Conditions

Recurrent Severe Aplastic Anemia Refractory Severe Aplastic Anemia

Outcome Measures

Primary Outcomes (3)

Eligibility Criteria

AgeUp to 75 Years

Sexall

Healthy VolunteersYes

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

RECIPIENT: Documented informed consent of the participant.
RECIPIENT: Age: \>= 40 years but =\< 75 years of age at time of enrollment.
RECIPIENT: Failed at least one trial of immunosuppressive therapy (IST) by being refractory (persistence of severe cytopenia and fulfillment of SAA disease criteria at least 3 months after initial IST) or having relapsed (initial improvement of cytopenia 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 anti-thymocyte globulin (ATG) based regimens, calcineuPririn inhibitors and/or other higher dose therapy directed at the treatment of primary SAA.
RECIPIENT: Karnofsky performance score \>= 60%.
RECIPIENT: Confirmed diagnosis of severe aplastic anemia as:
Bone marrow cellularity \< 25% or marrow cellularity \< 50% but with \< 30% residual hematopoietic cells;
Two out of three of the following (in peripheral blood):
Neutrophils \< 0.5 x 10\^9/L;
Platelets \< 20 x 10\^9/L;
Reticulocyte count \< 20 x 10\^9/L (\< 60 x 10\^9/L using an automated analysis).
RECIPIENT: No suitable fully matched related sibling donor (6/6 match for human leukocyte antigen \[HLA\]-A and B at intermediate or high resolution and DRbetaA1 at high resolution using deoxyribonucleic acid \[DNA\]-based typing) available.
RECIPIENT: Available relative of the patient who is a haploidentical match, including biological parents, siblings or half siblings, children, uncles/aunts, first cousins, etc. Eligible haploidentical donors will have 2-4 mismatches if HLA-A, -B, -C, and -DRB1 typing is used; 2-5 mismatches if HLA-A, -B, -C, -DRB1, and -DQB1 typing is used; and 2-6 mismatches if HLA-A, -B, -C, -DRB1, -DQB1, and -DPB1 typing is used. A unidirectional mismatch in either the graft versus host or host versus graft direction is considered a mismatch. The donor and recipient must demonstrate that they are a full haplotype match by being identical at a minimum of one allele (at high resolution DNA-based typing) at the following genetic loci: HLA-A, -B, -C, and DRB1 if 8 allele typing is used; HLA-A, -B, -C, -DRB1, and -DQB1 if 10 allele typing is used; and HLA-A, -B, -C, -DRB1-, DQB1, and -DPB1 is 12 allele typing is used by the local center.
RECIPIENT: Patient and/or legal guardian must sign informed consent for the hematopoietic stem cell transplantation (HSCT).
RECIPIENT: The haplo donor and/or legal guardian must be able to sign informed consent documents.
RECIPIENT: The potential haplo donor must be willing and able to donate bone marrow.
+27 more criteria

You may not qualify if:

RECIPIENT: Inherited and acquired (non-aplastic anemia) bone marrow failure syndromes such as Fanconi anemia must be ruled out according to center standards.
RECIPIENT: Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (e.g. Monosomy 7).
RECIPIENT: Any somatic mutations (including TERT, TERC, DKC1, NOP10) other than PIG-A (PNH) or BCOR mutation.
RECIPIENT: Diagnosis of myelodysplastic syndrome (MDS).
RECIPIENT: 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).

Contact the study team to confirm eligibility.

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (14)

Study Arms (1)

Treatment (conditioning, haploHCT)

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (14)

Study Arms (1)

Treatment (conditioning, haploHCT)

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

Source record

Locations