Congenital Cystic Adenomatoid Malformation
Conditions
Keywords
prenatal steroids, hydrops, congenital cystic adenomatoid malformation of the lung, prenatal intervention, betamethasone, prenatal diagnosis
Brief summary
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
Interventions
DRUGBetamethasone
12 mg intramuscularly x 2 doses 24 hours apart
DRUGPlacebo
PLACEBO: IM x 2 doses 24 hours apart
Sponsors
Children's Hospital Medical Center, Cincinnati
Children's Hospital of Philadelphia
University of California, San Francisco
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
FEMALE
Age
18 Years to No maximum
Healthy volunteers
Yes
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of Hydrops Fetalis | Delivery, up to approximately 20 weeks post-enrollment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. | 30 days after delivery (up to approximately 24 weeks post-enrollment) | Status of neonate survival 30 days after delivery |
Countries
United States
Participant flow
Recruitment details
Only one participant was enrolled to the study before it was terminated; no participants were enrolled to the control arm
Participants by arm
| Arm | Count |
|---|---|
| Active Study Group STEROID: Betamethasone; 12 mg intramuscularly x 2 doses 24 hours apart
Betamethasone: 12 mg intramuscularly x 2 doses 24 hours apart | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | Active Study Group |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Region of Enrollment United States | 1 participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 1 |
| serious Total, serious adverse events | 0 / 1 |
Outcome results
Primary
Incidence of Hydrops Fetalis
Time frame: Delivery, up to approximately 20 weeks post-enrollment
Population: Only one participant was enrolled to the study before it was terminated; no participants were enrolled to the control arm
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Active Study Group | Incidence of Hydrops Fetalis | 0 participants |
Secondary
Comparison of CCAM Size in Mid-trimester Fetuses (Study/Administration vs Control/Placebo)
Time frame: Baseline, Delivery (up to approximately 20 weeks post-enrollment)
Population: Study was terminated without enrollment to control arm; therefore, no comparison was made
Secondary
Survival at One-month Between Study and Control Groups.
Status of neonate survival 30 days after delivery
Time frame: 30 days after delivery (up to approximately 24 weeks post-enrollment)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Active Study Group | Survival at One-month Between Study and Control Groups. | 1 participants |