Infusion of expanded cord blood cells for treating blood cancers
Pilot Study: Infusion of Off-the-Shelf Ex Vivo Expanded Cryopreserved Progenitor Cells (Dilanubicel) in the Setting of Single Cord Blood Transplantation for Patients With Hematologic Malignancies
This study is testing if a new type of stem cell transplant using expanded umbilical cord blood can help people with blood cancers recover and produce healthy blood cells after chemotherapy and radiation.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 35 (estimated) |
| Ages | 10 Years to 65 Years |
| Sex | All |
| Sponsor | Fred Hutchinson Cancer Center Academic / other |
| Drugs / interventions | chemotherapy, radiation, cyclophosphamide, fludarabine |
| Locations | 1 site (Seattle, Washington) |
| Trial ID | NCT03399773 on ClinicalTrials.gov |
What this trial studies
This phase II trial investigates the effectiveness of donor umbilical cord blood transplant combined with ex-vivo expanded cord blood progenitor cells (dilanubicel) in patients with various types of blood cancer. Participants will undergo chemotherapy and radiation therapy prior to the transplant to prepare their bodies for the infusion of healthy stem cells. The study aims to assess how well these treatments can help restore normal blood cell production and eliminate remaining cancer cells. Patients will be monitored for outcomes over a period of up to two years following treatment.
Who should consider this trial
Good fit: Ideal candidates are patients aged 10 to 65 years with hematologic malignancies who do not have a suitable related donor.
Not a fit: Patients with hematologic malignancies who have a suitable related donor may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could improve survival rates and quality of life for patients with acute leukemia and myelodysplastic syndromes.
How similar studies have performed: Other studies have shown promise with similar approaches, but this specific combination of treatments is being evaluated for the first time.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients 10 to 65 years old with a hematologic malignancy in need of hematopoietic cell transplant who are \> 30 kg and without a suitable related donor * Patient must have hematologic malignancy that meets institutional eligibility requirements for cord blood transplant * Malignancies included are: * Acute leukemia, including acute myeloid leukemia (AML), biphenotypic acute leukemia or mixed-lineage leukemia, acute lymphoblastic leukemia (ALL); all patients must be in complete response (CR) as defined by \< 5% blasts by morphology/flow cytometry in a representative bone marrow sample with adequate cellularity to assess remission status * Myelodysplasia (MDS) International Prognostic Scoring System (IPSS) intermediate (Int)-2 or high risk (i.e., refractory anemia with excess blasts \[RAEB\], refractory anemia with excess blasts in transformation \[RAEBt\]) or refractory anemia with severe pancytopenia or high risk cytogenetics; blasts must be \< 10% in a representative bone marrow aspirate * Chronic myeloid leukemia excluding refractory blast crisis; to be eligible in first chronic phase (CP1) patient must have failed or be intolerant to tyrosine kinase inhibitor therapy * High dose TBI regimen: 10 to =\< 45 years * Intermediate intensity regimen: 10 to =\< 65 years * Patients 10 to =\< 45 years: Lansky (\< 16 years old) or Karnofsky (\>= 16 years old) \>= 70 or Eastern Cooperative Oncology Group (ECOG) 0-1 * Patients \> 45 to =\< 65 years: Karnofsky \>= 70 or ECOG 0-1 and non-age adjusted comorbidity index =\< 5 * Adults: Calculated creatinine clearance must be \> 60 mL and serum creatinine =\< 2 mg/dL * Children (\< 18 years old): Calculated creatinine clearance must be \> 60 mL/min * Total serum bilirubin must be \< 3 mg/dL unless the elevation is thought to be due to Gilbert's disease or hemolysis * Transaminases must be \< 3 x the upper limit of normal per reference values of treating institution * Carbon monoxide diffusing capability (DLCO) corrected \>= 60% normal (may not be on supplemental oxygen) * For pediatric patients unable to perform pulmonary function tests, O2 saturation \> 92% on room air * Left ventricular ejection fraction \>= 50% OR * Shortening fraction \> 26% * Ability of participant or legally authorized representative to understand and the willingness to sign a written informed consent form * DONOR: Minimum requirement: The cord blood (CB) unit must be matched at a minimum at 4/6 HLA-A, B antigens and DRB1 allele with the recipient; therefore, 0-2 mismatches at the A or B or DRB1 loci based on intermediate resolution at HLA-A, B and high resolution allele level typing at HLA- DRB1 are allowed * DONOR: Institutional guidelines for HLA-match may be followed as long as the minimum criteria for HLA-matching as above are met * DONOR: The CB unit selected for transplant must have a MINIMUM of 2.5 x 10\^7 TNC/kg * DONOR: The minimum recommended CD34/kg cell dose is 1.7 x 10\^5 CD34/kg * DONOR: A backup unit must be identified and reserved prior to the start of the treatment plan for possible infusion in the unlikely event of poor post-thaw viability of the primary CB unit. A suitable back up unit will be considered, as follows: * Must be matched at a minimum at 4/6 HLA-A, B, DRBl loci with the recipient. Therefore 0-2 mismatches at the A or B or DRBl loci based on intermediate resolution A, B antigen and DRBl allele typing for determination of HLA-match is allowed (Fred Hutch Protocol 2010). * Must contain a MINIMUM of 1.5 x 10\^7 TNC/kg to ensure the same requirement we use for a standard double CBT per CB selection guideline (Fred Hutch Protocol 2010). Exclusion Criteria: * Uncontrolled viral or bacterial infection at the time of study enrollment * Active or recent (prior 6 month) invasive fungal infection unless cleared by infectious disease (ID) consult * History of human immunodeficiency virus (HIV) infection * Pregnant or breastfeeding * Prior allogeneic transplant * Central nervous system (CNS) leukemic involvement not clearing with intrathecal chemotherapy; diagnostic lumbar puncture is to be performed * \< 30 kg
Where this trial is running
Seattle, Washington
- Fred Hutch/University of Washington Cancer Consortium — Seattle, Washington, United States (Recruiting)
Study contacts
- Principal investigator: Filippo Milano — Fred Hutch/University of Washington Cancer Consortium
- Study coordinator: Filippo Milano
- Email: fmilano@fredhutch.org
- Phone: 206.667.5925
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.