Angiogenic Cell Therapy for Pulmonary Hypertension

(SAPPHIRE Trial)

Recruiting at 6 trial locations

Overseen By Kim Danovitch, CCRP

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Northern Therapeutics

Must be taking: PAH-targeted therapy

Must not be taking: Investigational drugs

Disqualifiers: Pregnancy, Hypertension, Diabetes, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 21

22 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Duncan J Stewart, MD FRCPC

Principal Investigator

Northern Therapeutics, Inc.

Eligibility Criteria

Adults aged 18-80 with severe Pulmonary Arterial Hypertension (PAH) due to conditions like scleroderma or congenital heart defects, not participating in other trials or pulmonary rehab recently. Must be on stable PAH therapy for at least 3 months, able to walk unassisted, and meet specific criteria on diagnostic tests.

Inclusion Criteria

My PAH is classified as moderate to severe, and I've been on stable treatment for over 3 months.

I had surgery to fix a heart defect more than a year ago.

I can walk between 125 and 440 meters in 6 minutes.

Exclusion Criteria

I do not have a disease that severely limits my ability to walk.

You have three or more specific risk factors for heart failure with preserved ejection fraction (HFpEF) mentioned in the AMBITION study.

Unable to provide informed consent and comply with the visit schedule

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Apheresis and Randomization

Participants undergo apheresis for cell collection and are randomized to treatment arms

1 week

1 visit (in-person)

Treatment Course 1

Participants receive 4 monthly IV infusions according to their assigned treatment arm

6 months

4 visits (in-person)

Treatment Course 2

Participants receive another 4 monthly IV infusions according to their assigned treatment arm

6 months

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

11 visits (in-person)

Long-term Registry

Participants are followed for long-term safety information through annual telephone contacts

10 years

Treatment Details

Interventions

• Autologous EPCs transfected with human eNOS

Trial Overview The SAPPHIRE trial is testing the safety and effectiveness of monthly doses of autologous EPCs enhanced with human eNOS gene against a placebo in improving symptoms of PAH. Participants will receive either the treatment first followed by placebo or vice versa.

Participant Groups

Group I: Placebo followed by Autologous EPCs transfected with eNOS Experimental Treatment 1 Intervention

4 monthly IV injections of Placebo (Plasma-Lyte A) during Course 1 followed by 4 monthly IV injections of Autologous EPCs transfected with human eNOS (total of 80 million cells) during Course 2

Group II: Autologous EPCs transfected with eNOS followed by Placebo Experimental Treatment 1 Intervention

4 monthly IV injections of Autologous EPCs transfected with human eNOS (total of 80 million cells) during Course 1 followed by 4 monthly IV injections of Placebo (Plasma-Lyte A) during Course 2

Group III: Autologous EPCs transfected with eNOS Experimental Treatment 1 Intervention

4 monthly IV injections of Autologous EPCs transfected with human eNOS in Course 1 followed by 4 monthly injections of Autologous EPCs transfected with human eNOS in Course 2 (total of 160 million cells)

Who Is Running the Clinical Trial?

Northern Therapeutics

Lead Sponsor

Trials

3

Recruited

40+

Ottawa Hospital Research Institute

Collaborator

Trials

585

Recruited

3,283,000+