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Birth Defects We Treat

Since FTC was founded we have treated more than 15,000 birth defects, and of those 7% were CCAM

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Congenital Cystic Adenomatoid Malformation Prenatal Steroid Trial

Trial description
Patient Enrollment and Screening
- Inclusion Criteria
- Exclusion Criteria
What happens after enrollment?
Trial Coordinator Contact Information

Investigation of Prenatal Steroids for Treatment of Prenatally Diagnosed CCAMs

Preliminary evidence has shown that two doses of betamethasone, a type of corticosteroid commonly given in pregnancy, may help fetuses with large CCAM's (Tsao KJ et al. J Pediatr Surg 2003 Mar;38(3):508-10). It is hypothesized that betamethasone will stop the growth or shrink the size of the abnormal lung tissue, but this has not been well proven scientifically Therefore, our goal is further investigate this promising therapy, including its potential adverse effects. We are conducting a clinical trial for which we are currently enrolling patients. To take part in this study, you must be pregnant with a fetus with a large congenital cystic adenomatoid malformation (CCAM), (lung mass), and meet other requirements (Inclusion Criteria) listed below.

This is a randomized, double-blinded trial in which half of the women will be given betamethasone, and the other half will be given a placebo (an inactive substance). "Randomized" means who gets which treatment is determined by chance—neither the study investigators nor the patients will choose which treatment is given to patient. Furthermore, "double-blinded' means neither the investigators nor the patients will know which specific treatment the patients are receiving.

Patient Enrollment and Screening

UCSF will enroll 56 women divided into the two groups, Treatment and Placebo. Other medical institutions may join UCSF in this study. You will need to be screened by the doctors before you can enroll in the study. Screening will include:

Review of your medical chart
An ultrasound (sonogram)
fetal echocardiogram (an ultrasound of just the fetus's heart)
physical exam
consultation with a perinatologist
consultation with a pediatric surgeon

Inclusion Criteria	Exclusion Criteria
Gestational age < 26 weeks at randomization Maternal age ≥ 18 years of age Singleton pregnancy Normal karyotype (if available) Microcystic CCAM ≥ CVR 1.4 No maternal medical/surgical contradictions to steroid use No additional fetal anomalies Willingness to be randomized into either group	Gestational age > 26 weeks at randomization Maternal age < 18 years of age Other congenital anomalies Abnormal karyotype Fetal hydrops Microcystic CCAM CVR < 1.4 Maternal medical or surgical contraindications Refusal to accept randomization Maternal diabetes or use of insulin Preterm labor Multiple congenital anomalies with CCAM Multiple gestation Broncho-pulmonary sequestration or systemic feeder vessel Unable or unwilling to return to Center for second dose Prior randomization to this study Unable to understand consent process Failure to meet one or more of the inclusion criteria

Inclusion Criteria

Exclusion Criteria

Gestational age < 26 weeks at randomization
Maternal age ≥ 18 years of age
Singleton pregnancy
Normal karyotype (if available)
Microcystic CCAM ≥ CVR 1.4
No maternal medical/surgical contradictions to steroid use
No additional fetal anomalies
Willingness to be randomized into either group

Gestational age > 26 weeks at randomization
Maternal age < 18 years of age
Other congenital anomalies
Abnormal karyotype
Fetal hydrops
Microcystic CCAM CVR < 1.4
Maternal medical or surgical contraindications
Refusal to accept randomization
Maternal diabetes or use of insulin
Preterm labor
Multiple congenital anomalies with CCAM
Multiple gestation
Broncho-pulmonary sequestration or systemic feeder vessel
Unable or unwilling to return to Center for second dose
Prior randomization to this study
Unable to understand consent process
Failure to meet one or more of the inclusion criteria

What happens after enrollment?

If after the screening exams and procedures the study investigators find you eligible to enroll in the study and you choose to continue, you will then be randomized (placed into a group by chance). You will either be given betamethasone or a placebo. Neither you nor the doctor can choose which group you will be in. Regardless of which group you are in, you will receive 2 injections of either betamethasone or the placebo 24 hours apart. Neither you nor the doctor will know what group you are in.

Once enrolled in the study, follow up will include:

A fetal MRI at 30 weeks gestation
At least one ultrasound every week until you are 30 weeks gestation.
At least one ultrasound every two weeks once you are past 30 weeks gestation until delivery.

Your follow-up ultrasounds do not need to be performed at UCSF; they may be done at an OB office near your home.

This study will help doctors learn more about treatment for congenital cystic adenomatoid malformation, and it is hoped that this information will help in the treatment of future patients with CCAM.