Trial Outcomes & Findings for A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies (NCT NCT02354417)

Last Updated: 2018-10-10

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

3 participants

Primary outcome timeframe

Neutrophil engraftment by Day 42

Results posted on

2018-10-10

Participant Flow

Participant milestones

Participant milestones
Measure	ProHema-CB 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: ProHema-CB: Each subject will receive o
Overall Study STARTED	3
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	ProHema-CB 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: ProHema-CB: Each subject will receive o
Overall Study Physician Decision	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ProHema-CB n=3 Participants 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: ProHema-CB: Each subject will receive one administration of ProHema-CB unit transplant.
Age, Continuous	15.7 Years STANDARD_DEVIATION 3.2 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants
Weight (kg)	73.4 kg STANDARD_DEVIATION 28.8 • n=5 Participants

PRIMARY outcome

Timeframe: Neutrophil engraftment by Day 42

Outcome measures

Outcome measures
Measure	ProHema-CB n=3 Participants 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: ProHema-CB: Each subject will receive one administration of ProHema-CB unit transplant.
Safety Profile, Primarily Assessed by Neutrophil Engraftment	3 Participants

Adverse Events

ProHema-CB

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	ProHema-CB n=3 participants at risk 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: ProHema-CB: Each subject will receive o
General disorders Mucosal inflammation	33.3% 1/3 • During the First Year of Transplant Serious Adverse Events and Adverse Events assessed as possibly or probably related to ProHema are included below.
Vascular disorders Hypertension	33.3% 1/3 • During the First Year of Transplant Serious Adverse Events and Adverse Events assessed as possibly or probably related to ProHema are included below.

Additional Information

Chris Storgard, Chief Medical Officer

Fate Therapeutics, Inc.

Phone: 866-875-1833

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place