Denne siden ble automatisk oversatt og nøyaktigheten av oversettelsen er ikke garantert. Vennligst referer til engelsk versjon for en kildetekst.

Peripheral Blood Versus Bone Marrow Plus Peripheral Blood Grafts for Haploidentical Transplantation in Severe Aplastic Anemia (PB-Graft Haplo)

15. september 2026 oppdatert av: Xiao-Jun Huang, Peking University People's Hospital

A Randomized Controlled Trial Comparing Different Graft Sources for Haploidentical Transplantation in Aplastic Anemia

Severe aplastic anemia is a life-threatening bone marrow failure disorder. Haploidentical hematopoietic stem cell transplantation has become an important curative treatment option for patients who do not have an HLA-matched sibling donor.

Traditionally, haploidentical transplantation for severe aplastic anemia uses a graft composed of granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells. However, bone marrow collection is invasive and may increase donor burden. Peripheral blood stem cell collection is simpler and less invasive, but it remains unclear whether using peripheral blood stem cells alone provides similar clinical outcomes without increasing the risk of graft-versus-host disease.

This multicenter, randomized, open-label, non-inferiority trial will compare granulocyte colony-stimulating factor-primed peripheral blood stem cells alone with granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as graft sources for haploidentical transplantation in patients with severe or very severe aplastic anemia. Participants will be randomly assigned in a 1:1 ratio to either graft source group. The primary outcome is the cumulative incidence of grade II-IV acute graft-versus-host disease within 100 days after transplantation. Secondary outcomes include engraftment, chronic graft-versus-host disease, infections, immune reconstitution, survival, and donor safety.

Studieoversikt

Studietype

Intervensjonell

Registrering (Antatt)

180

Fase

  • Ikke aktuelt

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

    • Beijing Municipality
      • Beijing, Beijing Municipality, Kina, 100033
        • Rekruttering
        • Peking University People's Hospital
        • Ta kontakt med:

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen

Tar imot friske frivillige

Nei

Beskrivelse

Inclusion Criteria:

  • Patients with newly diagnosed or relapsed severe aplastic anemia (SAA) or very severe aplastic anemia (vSAA) according to the Camitta criteria.
  • SAA is defined as bone marrow cellularity <25% and at least two of the following peripheral blood criteria: absolute neutrophil count <0.5 × 10^9/L, platelet count <20 × 10^9/L, or reticulocyte count <20 × 10^9/L.
  • vSAA is defined as SAA with an absolute neutrophil count <0.2 × 10^9/L.
  • Age 18 to 40 years.
  • Availability of a haploidentical related donor.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
  • Adequate organ function, defined as follows:
  • Cardiac function: left ventricular ejection fraction ≥50% and no severe arrhythmia.
  • Hepatic function: total bilirubin ≤2 times the upper limit of normal and alanine aminotransferase/aspartate aminotransferase ≤3 times the upper limit of normal.
  • Renal function: creatinine clearance ≥60 mL/min.
  • No active infection.
  • For women of childbearing potential, a negative pregnancy test is required.
  • All patients must agree to use effective contraception during the study period.
  • Written informed consent provided by the patient or legal representative.

Exclusion Criteria:

  • Presence of hematologic malignancy or myelodysplastic syndrome-related chromosomal abnormalities, such as +8 or del(7q).
  • Prior allogeneic hematopoietic stem cell transplantation or prior organ transplantation.
  • Availability of an HLA-matched sibling donor.
  • Active uncontrolled infection, including unresolved invasive fungal disease, active tuberculosis, sepsis, or other uncontrolled infections.
  • Severe comorbidities, defined as a Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score ≥4, or uncontrolled autoimmune disease.
  • Donor-related contraindications, including donor age <12 years or >65 years, donor body mass index <18 or >35 kg/m², contraindications to anesthesia, or contraindications to bone marrow harvest.
  • Psychiatric illness that prevents cooperation with study procedures, or any condition considered by the investigator to make the patient unsuitable for study participation.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: G-CSF-Primed Peripheral Blood Stem Cell Graft
Participants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, and peripheral blood stem cells will be collected by apheresis. Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis.
Haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed peripheral blood stem cells alone as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by peripheral blood stem cell collection by apheresis.
Eksperimentell: G-CSF-Primed Bone Marrow Plus Peripheral Blood Stem Cell Graft
Participants assigned to this arm will undergo haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection. Participants will receive the protocol-specified conditioning regimen and graft-versus-host disease prophylaxis.
Haploidentical hematopoietic stem cell transplantation using granulocyte colony-stimulating factor-primed bone marrow plus peripheral blood stem cells as the graft source. Donors will receive granulocyte colony-stimulating factor mobilization, followed by bone marrow harvest and peripheral blood stem cell collection.

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease Within 100 Days After Transplantation
Tidsramme: Up to 100 Days After Transplantation
The cumulative incidence of newly diagnosed or persistent grade II-IV acute graft-versus-host disease within 100 days after haploidentical hematopoietic stem cell transplantation. Acute graft-versus-host disease will be graded according to the MAGIC criteria and assessed based on skin, gastrointestinal, and liver manifestations. Endpoint adjudication will be performed by an independent blinded endpoint review committee using medical records, photographs, endoscopy reports, and/or biopsy reports, as applicable.
Up to 100 Days After Transplantation

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

15. juni 2026

Primær fullføring (Antatt)

15. april 2027

Studiet fullført (Antatt)

31. desember 2029

Datoer for studieregistrering

Først innsendt

1. juni 2026

Først innsendt som oppfylte QC-kriteriene

1. juni 2026

Først lagt ut (Faktiske)

5. juni 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

16. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

15. september 2026

Sist bekreftet

1. mai 2026

Mer informasjon

Begreper knyttet til denne studien

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Abonnere