Fetal Tracheal Balloon Study in Diaphragmatic Hernia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00966823
Recruitment Status : Terminated (Device no longer available)
First Posted : August 27, 2009
Results First Posted : May 30, 2017
Last Update Posted : May 30, 2017
Information provided by (Responsible Party):
Francois Luks, Rhode Island Hospital

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

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

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Fetal Tracheal Balloon (IDE G080077) Study in Diaphragmatic Hernia
Study Start Date : September 2008
Actual Primary Completion Date : January 2015
Actual Study Completion Date : January 2015

Arm Intervention/treatment
Experimental: Detachable balloon
Intervention: Fetuses treated with endoscopic tracheal occlusion
Device: Fetal tracheal obstruction with detachable balloon (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.

Primary Outcome Measures :
  1. Newborn Survival at Birth [ Time Frame: Newborn period (1 day) ]

Secondary Outcome Measures :
  1. Newborn Survival at 30 Days [ Time Frame: 30 days ]
  2. Maternal Complications [ Time Frame: Intervention to 30 days postpartum ]
  3. Fetal Morbidity [ Time Frame: Intervention to delivery ]
    Fetal morbidity, fetal mortality

  4. Number of Participants With In Utero Lung Growth (LHR) >1.4 [ Time Frame: Intervention to 2 weeks post-intervention ]

    Inclusion criterion for the study is LHR<0.9 (extreme pulmonary hypoplasia). Given that LHR is relatively constant during 2nd and 3rd trimester of gestation, "In utero lung growth" is defined as LHR>1.4 (definition of mild/moderate pulmonary hypoplasia) within 2 weeks of intervention.

    Outcome measure = number of participants with LHR>1.4 at 2 weeks post-intervention

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00966823

United States, Rhode Island
Rhode Island Hospital/Women & Infants' Hospital of Rhode Island
Providence, Rhode Island, United States, 02903
Sponsors and Collaborators
Rhode Island Hospital
Principal Investigator: Francois I Luks, MD, PhD Rhode Island Hospital

Additional Information:

Responsible Party: Francois Luks, Francois I. Luks, MD, PhD, Rhode Island Hospital Identifier: NCT00966823     History of Changes
Other Study ID Numbers: G080077
First Posted: August 27, 2009    Key Record Dates
Results First Posted: May 30, 2017
Last Update Posted: May 30, 2017
Last Verified: May 2017

Keywords provided by Francois Luks, Rhode Island Hospital:
Lung development
Lung growth
Tracheal occlusion
Neonatal death

Additional relevant MeSH terms:
Lung Diseases
Hernia, Diaphragmatic
Hernias, Diaphragmatic, Congenital
Pathological Conditions, Anatomical
Respiratory Tract Diseases
Congenital Abnormalities