Ignite Creation Date:
2024-05-06 @ 9:31 AM
Last Modification Date:
2024-10-26 @ 12:15 PM
Study NCT ID:
NCT03001414
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-02-01
First Post:
2016-12-15
Brief Title:
Study of Angiogenic Cell Therapy for Progressive Pulmonary Hypertension Intervention With Repeat Dosing of eNOS-enhanced EPCs
Sponsor:
Northern Therapeutics
Organization:
Northern Therapeutics
Study Overview
Official Title:
A Multicentre Late Phase Clinical Trial to Establish the Efficacy and Safety of Repeat Dosing of Autologous Endothelial Progenitor Cells EPCs Transfected With Human Endothelial NO-synthase eNOS in Patients With Pulmonary Arterial Hypertension PAH on Top of Conventional Treatments
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SAPPHIRE
Brief 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
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None