← Back to Search

Angiogenic Cell Therapy

Angiogenic Cell Therapy for Pulmonary Hypertension (SAPPHIRE Trial)

Phase 2 & 3

Waitlist Available

Research Sponsored by Northern Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

6-Minute Walk Distance (6MWD) of ≥ 125 meters and ≤ 440 meters on two consecutive tests

Scleroderma associated PAH (limited or diffuse);

Must not have

Significant musculoskeletal disease or any other disease that would significantly limit ambulation

Unrepaired or recently repaired congenital systemic-to-pulmonary shunt other than patent foramen ovale

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 and 9 months

Summary

This trial is testing whether a treatment involving a person's own cells, transfected with human eNOS, is effective and safe for patients with severe PAH who are already on other PAH-targeted therapies.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions at the injection site, immune responses to modified cells, or complications from underlying health issues exacerbated by treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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

Select...

My condition is scleroderma with associated pulmonary arterial hypertension.

Select...

I have not used appetite suppressants or been exposed to toxins.

Select...

My PAH is caused by genetics, scleroderma, drugs/toxins, or heart defects.

Select...

My tests show I don't have blood clots in my lungs.

Select...

I am between 18 and 80 years old.

Select...

I can walk by myself, even if I use oxygen.

Select...

My pulmonary arterial hypertension is either idiopathic or inherited.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

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

Select...

I have a heart defect that hasn't been fixed or was recently repaired, except for a patent foramen ovale.

Select...

My PAH is not caused by conditions listed in the trial's inclusion criteria.

Select...

I have moderate to severe lung disease.

Select...

My liver condition is severe.

Select...

My kidney function is very low or I need dialysis.

Select...

I have had a procedure to create a hole in the wall between the upper chambers of my heart.

Select...

I have a history of serious heart artery problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 and 9 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 and 9 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 and 9 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change in 6 Minute Walk Distance (6MWD) from Baseline

Change in Echocardiography Right Ventricular (RV) Function Measures from Baseline

Change in Magnetic Resonance Imaging Right Ventricular (RV) Function Measures from Baseline

+3 more

Trial Design

3Treatment groups

Experimental Treatment

Group I: Placebo followed by Autologous EPCs transfected with eNOSExperimental Treatment1 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 PlaceboExperimental Treatment1 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 eNOSExperimental Treatment1 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)

0 / 16Eligibility criteria met