NCT03001414

Study of Angiogenic Cell Therapy for Progressive Pulmonary Hypertension

Study of Angiogenic Cell Therapy for Progressive Pulmonary Hypertension: Intervention With Repeat Dosing of eNOS-enhanced EPCs

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Northern Therapeutics · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The SAPPHIRE clinical trial seeks to establish the efficacy and safety of repeated monthly dosing of autologous EPCs transfected with human eNOS (heNOS) in patients with symptomatic severe PAH on available PAH-targeted medical therapy.

Detailed description

SAPPHIRE will use autologous progenitor cell-based gene delivery to enhance lung microvascular repair and regeneration in patients with severe symptomatic PAH. A total of 45 patients will be enrolled in this multi-centre, late phase, randomized, double-blind, placebo-controlled, 3-arm protocol. Up to nine centres across Canada will participate. Consented study participants who meet all eligibility criteria during the screening period will be scheduled to undergo apheresis. Following successful apheresis collection and receipt of the cell samples by the cell manufacturing facility, randomization will take place though a web-based system. Manufacturing of the cell therapy product will then be performed by the cell manufacturing facility according to the assigned treatment allocation: Arm 1: Placebo (Plasma-Lyte A; 4 monthly IV infusions) in Course 1 (1st 6 months) followed by Autologous EPCs transfected with human eNOS in Course 2 (2nd 6 months; 4 monthly IV infusions) Arm 2: Autologous EPCs transfected with human eNOS in Course 1 (1st 6 months; 4 monthly IV infusions) followed by Placebo (Plasma-Lyte A) in Course 2 (2nd 6 months; 4 monthly IV infusions) Arm 3: Autologous EPCs transfected with human eNOS in Course 1 (1st 6 months; 4 monthly IV infusions) followed by a repeat dosing with Autologous EPCs transfected with human eNOS in Course 2 (2nd 6 months; 4 monthly IV infusions) Approximately 5-9 days later, the study product will be transported to the investigative site where the initial treatment will be delivered to the study participant in an outpatient setting which is equipped for continuous monitoring of vital signs and oxygen saturation. Participants will subsequently be monitored for a minimum of 1 hour and discharged from the clinic once judged by the study investigator to be clinically stable. Treatment and follow-up assessments will take place over a 12-month period (11 study visits in total). Once the 12-month trial data collection is completed, the trial will convert to a registry with the goal of collecting long-term safety information through annual telephone contacts for 10 years. Participants will be permitted to enroll in other clinical trials during the registry period.

Conditions

• Hypertension,Pulmonary

Interventions

Type	Name	Description
BIOLOGICAL	Placebo followed by Autologous EPCs transfected with human eNOS	4 doses of placebo in first 6 months followed by 4 doses of autologous EPCs transfected with eNOS in second 6 months
BIOLOGICAL	Autologous EPCs transfected with human eNOS followed by Placebo	4 doses of autologous EPCs transfected with eNOS in first 6 months followed by 4 doses of placebo in second 6 months
BIOLOGICAL	Autologous EPCs transfected with human eNOS	4 doses of autologous EPCs transfected with eNOS in first 6 months which is repeated in second 6 months

Timeline

• Start date: 2017-09-28
• Primary completion: 2023-05-04
• Completion: 2023-11-01
• First posted: 2016-12-23
• Last updated: 2025-01-31
• Results posted: 2025-01-31

Locations

4 sites across 1 country: Canada