Sildenafil for Bronchopulmonary Dysplasia

(SILDI-SAFE Trial)

Recruiting at 29 trial locations

Overseen By Project Leader

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Christoph Hornik

Must not be taking: Vasopressors, Inhaled nitric oxide

Disqualifiers: Pulmonary hypertension, Congenital heart defect, others

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

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 2

Duration: 4 weeks

122 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a multicenter, randomized, placebo-controlled, sequential dose-escalating, double-masked, safety study of sildenafil in premature infants (inpatient in Neonatal Intensive Care Units (NICUs)) with severe bronchopulmonary dysplasia (BPD).

Research Team

Christoph Hornik, MD

Principal Investigator

Duke UMC

Matt Laughon, MD

Principal Investigator

UNC

Eligibility Criteria

This trial is for premature infants in NICUs with severe bronchopulmonary dysplasia. Eligible babies are 32-44 weeks postmenstrual age, born before 29 weeks gestation, and need respiratory support. Parents must consent to participate. Infants can't join if they've had certain heart defects, sickle cell disease, recent vasopressors or nitric oxide use, high liver enzymes, or known sildenafil allergy.

Inclusion Criteria

Documented informed consent from parent or guardian, prior to study procedures

I am on a breathing machine or CPAP depending on my baby's age in weeks.

I was born before reaching 29 weeks of pregnancy.

Exclusion Criteria

I have not taken milrinone in the last 24 hours.

I have sickle cell disease.

Participant will be reassessed prior to dosing to reconfirm eligibility criteria.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

Treatment

Participants receive sildenafil or placebo every 8 hours for 28 days

4 weeks

Daily monitoring in NICU

Weaning

Participants are weaned off the study drug following the treatment period

1 week

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

2 visits (in-person)

Treatment Details

Interventions

• Placebo
• Sildenafil

Trial Overview The study tests the safety of Sildenafil compared to a placebo in helping premature infants with severe lung problems (BPD). It's randomized (babies are put into groups by chance), double-masked (neither doctors nor parents know which treatment the baby gets), and dose levels increase over time.

Participant Groups

Group I: Cohort 1, sildenafil Active Control 1 Intervention

Sildenafil (0.5 mg/kg IV or 1 mg/kg enteral) every 8 hours for 28 days

Group II: Cohort 3, sildenafil Active Control 1 Intervention

Sildenafil (2 mg/kg IV or 4 mg/kg enteral) every 8 hours for 28 days

Group III: Cohort 2, sildenafil Active Control 1 Intervention

Sildenafil (1 mg/kg IV or 2 mg/kg enteral) every 8 hours for 28 days

Group IV: Cohort 2, placebo Placebo Group 1 Intervention

Placebo (IV or enteral) every 8 hours for 28 days

Group V: Cohort 3, placebo Placebo Group 1 Intervention

Placebo (IV or enteral) every 8 hours for 28 days

Group VI: Cohort 1, placebo Placebo Group 1 Intervention

Placebo (IV or enteral) every 8 hours for 28 days

Sildenafil is already approved in United States, European Union, Canada, Japan for the following indications:

Approved in United States as Viagra for:

• Erectile dysfunction
• Pulmonary arterial hypertension

Approved in European Union as Viagra for:

• Erectile dysfunction
• Pulmonary arterial hypertension

Approved in United States as Revatio for:

• Pulmonary arterial hypertension

Approved in Canada as Sildenafil for:

• Erectile dysfunction
• Pulmonary arterial hypertension

Approved in Japan as Sildenafil for:

• Erectile dysfunction
• Pulmonary arterial hypertension

Who Is Running the Clinical Trial?

Christoph Hornik

Lead Sponsor

Trials

1

Recruited

120+

Christoph P Hornik, MD MPH

Lead Sponsor

Trials

3

Recruited

280+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

University of North Carolina, Chapel Hill

Collaborator

Trials

1,588

Recruited

4,364,000+