Chronic Sildenafil for Severe Diaphragmatic Hernia

NCT00133679 | Terminated Phase 4 Results DMC

• 1 other identifier: K23HL079922-01
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 3

Brief Summary

The purpose of this study is to test if sildenafil is effective in the treatment of infants with severe congenital diaphragmatic hernia (determined by the presence of prolonged pulmonary hypertension or prolonged oxygen supplementation on mechanical ventilation), as measured by the estimated pulmonary artery systolic pressure following treatment.

Conditions

Hernia, Diaphragmatic; Hypertension, Pulmonary; Hypoplasia, Pulmonary

Keywords

Chronic lung disease; Nitric oxide; cyclic guanosine monophosphate; phosphodiesterase inhibitor

Outcome Measures

Primary Outcomes (1)

• Estimated Systolic Pulmonary Arterial Pressure at Final Echocardiogram

  7 weeks

Secondary Outcomes (5)

• Systemic Levels of Vasoactive Substances at the Time of Final Echocardiogram

  7 weeks

• Adverse Effects of Sildenafil Therapy

  2 years

• Somatic Growth at 1 and 2 Years of Age

  2 years

• Neurodevelopmental Outcome at 1 and 2 Years of Age

  2 years

• Respiratory Status at 1 and 2 Years of Age

  2 years

Study Arms (2)

1

EXPERIMENTAL

Sildenafil x 45 days

Drug: sildenafil

2

PLACEBO COMPARATOR

Placebo x 45 d

Drug: Placebo

Interventions

sildenafil DRUG

Sildenafil 0.5 mg/kg every 6 hours orally x 45 d

Also known as: Viagra, Revatio

1

Placebo DRUG

Placebo suspension (equal volume to experimental drug) x 45 days

2

Eligibility Criteria

• Age: 10 Days - 42 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Congenital diaphragmatic hernia
• days (d) of age
• Significant illness severity as demonstrated by:
• Receiving assisted ventilation and
• FiO2 \>= 0.40 at 10-14d of age, or
• FiO2 \>= 0.40 for \>=48hours at 15-27d of age, or
• FiO2 \>= 0.35 at 28-42d of age
• Or, need for extracorporeal support at \>=10d of age
• Or, estimated pulmonary arterial or right ventricular systolic pressure of \>= 2/3 systemic pressure at 14-42d of age

You may not qualify if:

• Structural congenital heart disease (other than patent ductus arteriosus or patent foramen ovale/atrial septal defect \[ASD\] or non-hemodynamically significant ventricular septal defect \[VSD\])
• Sildenafil contraindicated (until condition resolves):
• Unable to absorb oral medication, or
• Unstable systemic blood pressure, or
• Receiving a drug that may interfere with sildenafil metabolism, or
• Renal insufficiency
• Hepatic insufficiency Previous use of sildenafil

Sponsors & Collaborators

• University of California, San Francisco: lead
• Children's Hospital Los Angeles: collaborator
• Ann & Robert H Lurie Children's Hospital of Chicago: collaborator

Study Sites (3)

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

University of California San Francisco Children's Hospital

San Francisco, California, 94143, United States

Location

Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

MeSH Terms

Conditions

Hernia, Diaphragmatic; Hypertension, Pulmonary

Interventions

Sildenafil Citrate

Condition Hierarchy (Ancestors)

Internal Hernia; Hernia; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Sulfonamides; Amides; Organic Chemicals; Sulfones; Sulfur Compounds; Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Roberta Keller, MD
• Organization: University of California, San Francisco

roberta.keller@ucsf.edu

(415) 476-1888

Study Officials

• Roberta L Keller, MD

  University of California, San Francisco

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2005
• First Posted: August 23, 2005
• Study Start: February 1, 2006
• Primary Completion: July 1, 2013
• Study Completion: October 1, 2013
• Last Updated: January 13, 2021
• Results First Posted: January 13, 2021

Record last verified: 2021-01

Locations

US (3)