Phase 2
N=14
Umbilical Cord Blood Transplantation In Patients With Hematologic Malignancies Using A Myeloablative Preparative Regimen
Hematologic Malignancies · Disorder Related to Transplantation · Hematopoietic Malignancy
Bottom Line
View on ClinicalTrials.gov: NCT01328496 ↗Enrolled (actual)
14
Serious AEs
76.9%
Results posted
May 2017
Primary outcome: Primary: Event Free Survival (EFS) for Research Participants — 4 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Preparative Regimen (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- St. Jude Children's Research Hospital
- Primary completion
- Oct 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Event Free Survival (EFS) for Research Participants |
4 | — |
| SECONDARY Number of Observational Arm Participants Engrafted |
3 | — |
| SECONDARY Number of Observational Arm Patients Who Relapsed |
1 | — |
| SECONDARY Number of Deaths of Observational Arm Patients |
2 | — |
| SECONDARY Number of Observational Arm Patients With Transplant-related Mortality (TRM) |
1 | — |
| SECONDARY Number of Participants With Acute GVHD |
2; 0; 1; 0; 1; 1 | — |
| SECONDARY Number of Participants With Chronic GVHD |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Time to Engraftment of Research Arm Participants |
42.67; 17.11 | — |
| SECONDARY Incidence of Transplant-related Mortality (TRM) |
0; 1 | — |
| SECONDARY The Number of Participants With Transplant-related Morbidity |
8; 3 | — |
Summary
In this study, participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT), who do not have a suitable human leukocyte antigen (HLA)-matched related/sibling donor (MSD), matched unrelated donor (MURD) or killer-immunoglobulin receptors (KIR) ligand mismatched haploidentical donor identified, will receive an umbilical cord blood transplantation (UCBT) using a myeloablative preparative regimen.
The preparative regimen includes fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (10.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Eligibility Criteria
Inclusion Criteria
- Age less than or equal to 21 years old.
- Has a partially HLA-matched single or double UCB product
- High-risk hematologic malignancy.
- High risk ALL in CR1, ALL in High risk CR2, ALL in CR3 or subsequent.
- AML in high risk CR1, AML in CR2 or subsequent
- AML in first relapse with 12mo
- MDS, primary or secondary
- NK cell, biphenotypic, or undifferentiated leukemia in CR1 or subsequent.
- CML in accelerated phase, or in chronic phase with persistent molecular positivity or intolerance to tyrosine kinase inhibitor.
- Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous HCT, or unable to mobilize stem cells for autologous HCT.
- Non-Hodgkin lymphoma in CR2 or subsequent after failure of prior autologous HCT, or unable to mobilize stem cells for autologous HCT.
- JMML
- All patients with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for study.
Patient must fulfill pre-transplant evaluation:
- Cardiac shortening fraction ≥ 26%.
- Creatinine clearance ≥ 70 ml/min/1.73m2.
- Forced vital capacity (FVC) ≥ 50% of predicted value or pulse oximetry ≥ 92% on room air.
- Karnofsky (≥ 16 years) or Lansky (<16 years) performance score ≥ 70
- Bilirubin ≤ 2.5 mg/dL.
- Alanine aminotransferase (ALT) ≤ 5 times the upper limit of normal for age.
- Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age.
Exclusion Criteria
- Patient has a suitable MSD, volunteer MURD, or KIR mismatched haploidentical donor available in the necessary time for stem cell donation.
- Patient has any other active malignancy other than the one for which HCT is indicated.
- Patient had a prior allogeneic HCT
- Patient had an autologous HCT within the previous 12 months.
- Patient is pregnant as confirmed by positive serum or urine pregnancy test within 14 days prior to enrollment.
- Patient is lactating
- Patient has Down Syndrome
- Patient has a current uncontrolled bacterial, fungal, or viral infection per the judgment of the PI.
Data sourced from ClinicalTrials.gov (NCT01328496). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.