Fetal Tracheal Balloon Study in Diaphragmatic Hernia

This study is currently recruiting participants.
Verified September 2012 by Rhode Island Hospital
Sponsor:
Information provided by (Responsible Party):
Francois Luks, Rhode Island Hospital
ClinicalTrials.gov Identifier:
NCT00966823
First received: August 26, 2009
Last updated: September 24, 2012
Last verified: September 2012
  Purpose

The purpose of this phase 2 limited study is to examine whether prenatal intervention correct the lung underdevelopment associated with severe diaphragmatic hernia.


Condition Intervention Phase
Diaphragmatic Hernia
Lung Disease
Device: Fetal tracheal obstruction with detachable balloon (device)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase 2 Fetal Tracheal Balloon (IDE G080077) Study in Diaphragmatic Hernia

Resource links provided by NLM:


Further study details as provided by Rhode Island Hospital:

Primary Outcome Measures:
  • Survival at birth [ Time Frame: Newborn period (1 day) ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Survival at 30 days [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]
  • Maternal complications [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]
  • Fetal morbidity [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]
  • In utero lung growth (LHR) [ Time Frame: 30 days ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 15
Study Start Date: September 2008
Estimated Study Completion Date: September 2013
Estimated Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Detachable balloon
Fetuses treated with endoscopic tracheal occlusion
Device: Fetal tracheal obstruction with detachable balloon (device)
  • Endoscopic placement of a detachable balloon in the fetal trachea at 28-30 weeks gestation.
  • Ultrasound-guided puncture of balloon or, if not feasible, repeat endoscopic tracheoscopy with puncture and retrieval of the balloon at 34 weeks gestation.
Other Name: Goldvalve Balloon, nFocus Neuromedical, Inc.

Detailed Description:

Congenital diaphragmatic hernia (CDH) has traditionally been associated with very high mortality rates. Most infants died of pulmonary hypoplasia and severe pulmonary hypertension. This led to correction of CDH and pulmonary hypoplasia before birth. Unfortunately, maternal morbidity of open fetal surgery was significant and fetal mortality was very high (>60%). Moreover, the results of postnatal therapy for CDH improved dramatically, from less than 20% survival several decades ago to more than 70% today.

Fetal intervention has evolved as well, to a minimally invasive approach that involves a single endoscopic port and occlusion of the fetal trachea. While this has considerably decreased the morbidity and fetal mortality of the in utero procedure, its results do not exceed the overall (i.e., non-stratified) results of contemporary postnatal treatment. Most recently, a multicentric cooperative study under (Eurofoetus) has conducted a clinical trial comparing postnatal treatment with endoscopic fetal tracheal occlusion for the most severe forms of CDH. Results of the Eurofoetus trial and of a recent retrospective review involving European and North-American centers have shown the following: 1) It is possible to identify a specific subgroup of fetuses with CDH in whom survival can be predicted to be less than 10%, despite all current methods of postnatal treatment, 2) Survival of fetuses with predicted postnatal survival of 8% was >50% following endoscopic fetal tracheal occlusion, and 3) Fetal tracheal occlusion in that group resulted in an increase in lung size (LHR), from an average of 0.7 pre-intervention, to 1.7 post-intervention.

Based on the available research literature, the results of the Eurofoetus trial, and this institution's experience with endoscopic fetal surgery, we hypothesize that in the highest risk group of fetuses with congenital diaphragmatic hernia, where chances of survival is estimated at less than 10%, endoscopic fetal tracheal occlusion in late second trimester, with reversal of occlusion in mid-third trimester, allows catch-up lung growth and maturation and converts the condition into one with intermediate to good prognosis (predicted survival 50-60%). We propose to offer this form of treatment, under an FDA-approved Investigational Device Exemption (G080077), to eligible patients, on a case-by-case basis, after discussion before a multidisciplinary board.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Singleton pregnancies
  • Isolated congenital diaphragmatic hernia
  • Normal karyotype (amniocentesis)
  • Initial diagnosis before 26 weeks gestation
  • Competent cervix
  • Severity of CDH: lung-to-head ratio (LHR) ≤0.8 at 22-26 weeks gestation
  • Liver herniation in the chest
  • Informed consent

Exclusion Criteria:

  • Preterm labor, premature rupture of membranes or amniotic leak
  • Significant maternal morbidity
  • Minor (<18 years)
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00966823

Contacts
Contact: Francois I Luks, MD, PhD 401-228-0556 Francois_Luks@brown.edu

Locations
United States, Rhode Island
Rhode Island Hospital/Women & Infants' Hospital of Rhode Island Recruiting
Providence, Rhode Island, United States, 02903
Contact: Francois I Luks, MD, PhD    401-228-0556    Francois_Luks@brown.edu   
Contact: Deb Watson-Smith, RN    401-421-1939    dwsmith@usasurg.org   
Principal Investigator: Francois I Luks, MD, PhD         
Sub-Investigator: Stephen R Carr, MD         
Sub-Investigator: Christopher S Muratore, MD         
Sub-Investigator: Barbara M O'Brien, MD         
Sponsors and Collaborators
Rhode Island Hospital
Investigators
Principal Investigator: Francois I Luks, MD, PhD Rhode Island Hospital
  More Information

Additional Information:
Publications:

Responsible Party: Francois Luks, Francois I. Luks, MD, PhD, Rhode Island Hospital
ClinicalTrials.gov Identifier: NCT00966823     History of Changes
Other Study ID Numbers: G080077
Study First Received: August 26, 2009
Last Updated: September 24, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Rhode Island Hospital:
Diaphragm
Trachea
Lung development
Lung growth
Tracheal occlusion
ECMO
Neonatal death

Additional relevant MeSH terms:
Hernia
Hernia, Diaphragmatic
Hernia, Hiatal
Lung Diseases
Pathological Conditions, Anatomical
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on April 16, 2014