CompletedPhase 2

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

United States32 participantsStarted 2017-07-03

Plain-language summary

This study is a prospective, multicenter phase II study with patients receiving haploidentical transplantation for Severe Aplastic Anemia (SAA). The primary objective is to assess overall survival (OS) at 1 year post-hematopoietic stem cell transplantation (HSCT).

Who can participate

Age range75 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Patient is \< 75 years of age at time of enrollment.
✓. Confirmed diagnosis of SAA, either from initial diagnosis or follow-up assessments, defined 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 x10\^9/L, Platelets \< 20 x10\^9/L, or Reticulocyte count \< 20 x10\^9/L (\<60 x 10\^9/L using an automated analysis)
✓. No suitable fully matched related sibling donor (6/6 match for human leukocyte antigen (HLA)-A and B at intermediate or high resolution and DRB1 at high resolution using DNA-based typing) available.
✓. Failed at least one trial of immunosuppressive therapy (IST) by being refractory or having relapsed. IST could have included ATG based regimens, calcineurin inhibitors and/or other higher dose therapy directed at the treatment of primary SAA.
✓. 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. See Section 2.4 for additional information.
✓. Patient and/or legal guardian must sign informed consent for HSCT.

What they're measuring

Percentage of Participants With Overall Survival (OS)

Timeframe: 1 year

Trial details

NCT IDNCT02918292

SponsorMedical College of Wisconsin

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2021-08-17

Results submitted2022-04-29

View on ClinicalTrials.gov More Severe Aplastic Anemia trials

Who can participate

Age range75 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Patient is \< 75 years of age at time of enrollment.
✓. Confirmed diagnosis of SAA, either from initial diagnosis or follow-up assessments, defined 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 x10\^9/L, Platelets \< 20 x10\^9/L, or Reticulocyte count \< 20 x10\^9/L (\<60 x 10\^9/L using an automated analysis)
✓. No suitable fully matched related sibling donor (6/6 match for human leukocyte antigen (HLA)-A and B at intermediate or high resolution and DRB1 at high resolution using DNA-based typing) available.
✓. Failed at least one trial of immunosuppressive therapy (IST) by being refractory or having relapsed. IST could have included ATG based regimens, calcineurin inhibitors and/or other higher dose therapy directed at the treatment of primary SAA.
✓. 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. See Section 2.4 for additional information.
✓. Patient and/or legal guardian must sign informed consent for HSCT.

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Exclusion criteria