A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies

NCT02354417 | Terminated Phase 1 Results DMC

• 1 other identifier: FT1050-04
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 2

Brief Summary

This is an open-label, safety study of a single ProHema-CB product administered following myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.

Conditions

Hematologic Malignancies

Outcome Measures

Primary Outcomes (1)

• Safety Profile, Primarily Assessed by Neutrophil Engraftment

  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.

  Neutrophil engraftment by Day 42

Study Arms (1)

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.

Biological: Biological: ProHema-CB

Interventions

Biological: ProHema-CB BIOLOGICAL

Each subject will receive one administration of ProHema-CB unit transplant.

ProHema-CB

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male and female subjects aged 1 to 18 years, inclusive.
• Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate.
• Acute Myelogenous Leukemia (AML) in high risk 1st or subsequent CR
• Acute Lymphoblastic Leukemia (ALL) in CR
• NK cell lymphoblastic leukemia in any CR
• Biphenotypic or undifferentiated leukemia in 1st or subsequent CR
• Myelodysplastic Syndrome (MDS) at any stage.
• Chronic Myelogenous Leukemia (CML) All subjects with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for trial.
• 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.
• Availability of suitable primary and secondary umbilical cord blood (UCB) units.
• Adequate performance status, defined as:
• Subjects ≥ 16 years: Karnofsky score ≥ 70%.
• Subjects \< 16 years: Lansky score ≥ 70%.
• Cardiac: Left ventricular ejection fraction at rest must be \> 40%, or shortening fraction \> 26%.
• Pulmonary:

You may not qualify if:

• Female subjects that are pregnant or breastfeeding.
• Evidence of HIV infection or HIV positive serology.
• Current uncontrolled bacterial, viral or fungal infection.
• Prior allogeneic hematopoietic stem cell transplant.
• Autologous transplant \< 12 months prior to enrollment.
• Prior autologous transplant for the disease for which the UCB transplant is being performed.
• Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment.
• Inability to receive TBI.
• Requirement of supplemental oxygen.
• HLA-matched related donor able to donate.
• Use of an investigational drug within 30 days prior to screening.
• Subject is unlikely to comply with the protocol requirements, instructions and study-related restrictions

Sponsors & Collaborators

• Fate Therapeutics: lead

Study Sites (2)

City of Hope

Duarte, California, 91010, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115-5450, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Chris Storgard, Chief Medical Officer
• Organization: Fate Therapeutics, Inc.

chris.storgard@fatetherapeutics.com

866-875-1833

Study Officials

• Chris Storgard, MD

  Fate Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 25, 2014
• First Posted: February 3, 2015
• Study Start: December 1, 2014
• Primary Completion: December 1, 2016
• Study Completion: February 1, 2017
• Last Updated: October 10, 2018
• Results First Posted: October 10, 2018

Record last verified: 2018-02

Locations

US (2)