Prenatal Steroids for Treatment of Congenital Cystic Adenomatoid Malformations (CCAM)

Study Overview
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Brief Summary
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Congenital cystic adenomatoid malformations (CCAMs) are theorized to be growing immature lung tissue. Administration of maternal steroids in the mid-trimester may stop the growth or decrease the size of the CCAM, thus increasing normal lung tissue and improving survival in fetuses with large CCAMs. This is a prospective, blinded, randomized trial comparing administration of a single course of antenatal steroids (Betamethasone) to control (i.e., placebo). The primary outcome variable will be incidence of hydrops. One month postnatal survival and relative size of the CCAM as determined by CCAM volume:head circumference ratio (CVR) between treatment/no treatment groups will be secondary outcome variables. Change in size of CCAM will be serially followed for both groups with individual growth curves being plotted prenatally and these will be compared with pathology weigh and volume to evaluate treatment effect. Other prenatal data collected will include: incidence of polyhydramnios, incidence of premature rupture of membranes, incidence of material complications. We will also compare mode of delivery, postnatal respiratory compromise, need for resection in the first week of life, and occurrence of complications during newborn administration

Official Title
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Investigation of Prenatal Steroids for Treatment of Prenatally Diagnosed CCAMs

Conditions
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Congenital Cystic Adenomatoid Malformation

Intervention / Treatment
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* Drug: Betamethasone
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: GA < 26 weeks Maternal age > 18 years of age Singleton pregnancy Normal chromosomes CCAM volume to head circumference ratio (CVR) > 1.4 No maternal medical/surgical contraindications No evidence of hydrops Not previously randomization Exclusion Criteria: Maternal diabetes or use of insulin Preterm labor Multiple congenital anomalies with CCAM Chromosomal anomaly with CCAM Multiple gestation pregnancy with CCAM Not willing to be randomized Unable or unwilling to return to UCSF for second dose of drug or placebo CVR < 1.4

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Female

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Active Study Group<br>STEROID: Betamethasone; 12 mg intramuscularly x 2 doses 24 hours apart | Drug: Betamethasone<br>* 12 mg intramuscularly x 2 doses 24 hours apart<br>|
| Placebo Comparator: Placebo Group<br>PLACEBO: IM x 2 doses 24 hours apart | Drug: Placebo<br>* PLACEBO: IM x 2 doses 24 hours apart<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Hydrops Fetalis |  | Delivery, up to approximately 20 weeks post-enrollment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Comparison of CCAM Size in Mid-trimester Fetuses (Study/Administration vs Control/Placebo) |  | Baseline, Delivery (up to approximately 20 weeks post-enrollment) | 
| Survival at One-month Between Study and Control Groups. | Status of neonate survival 30 days after delivery | 30 days after delivery (up to approximately 24 weeks post-enrollment) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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prenatal steroids, hydrops, congenital cystic adenomatoid malformation of the lung, prenatal intervention, betamethasone, prenatal diagnosis