Description:
This is an open-label, safety study of a single ProHema-CB product administered following myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.
Clinical Trials /
A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies
NCT02354417
Related Conditions:
- Acute Myeloid Leukemia
Recruiting Status:
Terminated
Phase:
Phase 1
Trial Eligibility
Document
Title
- Brief Title: A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies
- Official Title: A Phase 1 Trial of a Single ProHema® CB Product (Ex Vivo Modulated Human Cord Blood Cells) Following Myeloablative Conditioning for Pediatric Patients With Hematologic Malignancies
Clinical Trial IDs
- ORG STUDY ID: FT1050-04
- NCT ID: NCT02354417
Conditions
- Hematologic Malignancies
Interventions
| Drug | Synonyms | Arms |
|---|---|---|
| Biological: ProHema-CB | ProHema-CB |
Purpose
This is an open-label, safety study of a single ProHema-CB product administered following
myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.
Detailed Description
A maximum of 18 eligible male and female subjects (1 to 18 years old, inclusive) will be
enrolled and treated in the trial at approximately 3 to 5 centers within the U.S. These 18
subjects will consist of 3 cohorts of 6 subjects each. The cohorts will be defined by age: 1
to 4 years; > 4 to 12 years; and > 12 to 18 years. These cohorts will be enrolled
simultaneously.
All subjects will be admitted to the hospital, per institutional practice, and will receive a
myeloablative conditioning regimen, after which they will receive an HLA-matched or partially
matched ProHema-CB unit on study Day 0.
Trial Arms
| Name | Type | Description | Interventions |
|---|---|---|---|
| ProHema-CB | Experimental | All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour. |
|
Eligibility Criteria
Inclusion Criteria:
1. Male and female subjects aged 1 to 18 years, inclusive.
2. Subjects with hematologic malignancies for whom allogeneic stem cell transplantation
is deemed clinically appropriate.
1. Acute Myelogenous Leukemia (AML) in high risk 1st or subsequent CR
2. Acute Lymphoblastic Leukemia (ALL) in CR
3. NK cell lymphoblastic leukemia in any CR
4. Biphenotypic or undifferentiated leukemia in 1st or subsequent CR
5. Myelodysplastic Syndrome (MDS) at any stage.
6. Chronic Myelogenous Leukemia (CML) All subjects with evidence of CNS leukemia
must be treated and be in CNS CR to be eligible for trial.
3. Lack of 5-6/6 HLA matched related or 8/8 HLA A, B, C, DRß1 matched unrelated donor; or
unrelated donor not available within appropriate timeframe, as determined by the
transplant physician.
4. Availability of suitable primary and secondary umbilical cord blood (UCB) units.
5. Adequate performance status, defined as:
1. Subjects ≥ 16 years: Karnofsky score ≥ 70%.
2. Subjects < 16 years: Lansky score ≥ 70%.
6. Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening
fraction > 26%.
7. Pulmonary:
1. Subjects > 10 years: DLCO (diffusion capacity) > 50% of predicted (corrected for
hemoglobin)
2. FEV1, FVC > 50% of predicted; Note: If unable to perform pulmonary tests, then O2
saturation > 92% on room air.
8. Renal: Serum creatinine within normal range for age, or if serum creatinine outside
normal range for age, then renal function (creatinine clearance or GFR) >
70mL/min/1.73m2.
9. Hepatic: Bilirubin ≤ 2.5 mg/dL (except in the case of Gilbert's syndrome or ongoing
hemolytic anemia); and ALT, AST and Alkaline Phosphatase ≤ 5 × ULN.
10. Signed IRB approved Informed Consent Form (ICF).
Exclusion Criteria:
1. Female subjects that are pregnant or breastfeeding.
2. Evidence of HIV infection or HIV positive serology.
3. Current uncontrolled bacterial, viral or fungal infection.
4. Prior allogeneic hematopoietic stem cell transplant.
5. Autologous transplant < 12 months prior to enrollment.
6. Prior autologous transplant for the disease for which the UCB transplant is being
performed.
7. Active malignancy other than the one for which the UCB transplant is being performed
within 12 months of enrollment.
8. Inability to receive TBI.
9. Requirement of supplemental oxygen.
10. HLA-matched related donor able to donate.
11. Use of an investigational drug within 30 days prior to screening.
12. Subject is unlikely to comply with the protocol requirements, instructions and
study-related restrictions
| Maximum Eligible Age: | 18 Years |
| Minimum Eligible Age: | 1 Year |
| Eligible Gender: | All |
| Healthy Volunteers: | No |
Primary Outcome Measures
| Measure: | Safety Profile, Primarily Assessed by Neutrophil Engraftment |
| Time Frame: | Neutrophil engraftment by Day 42 |
| Safety Issue: | |
| Description: | To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment. |
Details
| Phase: | Phase 1 |
| Primary Purpose: | Interventional |
| Overall Status: | Terminated |
| Lead Sponsor: | Fate Therapeutics |
Last Updated
October 10, 2018
