Congenital Cystic Adenomatoid MalformationPhase 1/2CCAM SteroidsNCT00670956
Prenatal Steroids for Treatment of Congenital Cystic Adenomatoid Malformations (CCAM)
A Phase 1/2 study testing Betamethasone in people with Congenital Cystic Adenomatoid Malformation, run by University of California, San Francisco at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older, women onlyHealthy volunteers can take part
- Conditions the study is for
- Congenital Cystic Adenomatoid Malformation
- What is being tested?
- Betamethasone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 1 participants
- Where
- 3 sitesUnited States
- Study dates
- Apr 2008to Sep 2011
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Women only
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229-3039
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of California, San Francisco Fetal Treatment Center
San Francisco, California, 94143
About this study
Official title: Investigation of Prenatal Steroids for Treatment of Prenatally Diagnosed CCAMs
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
- Being tested
- Betamethasone, Placebo
- Keywords
- prenatal steroids, hydrops, congenital cystic adenomatoid malformation of the lung, prenatal intervention, betamethasone, prenatal diagnosis
- Medical subject headings
- Cystic Adenomatoid Malformation of Lung, Congenital, Edema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BetamethasoneDrug
12 mg intramuscularly x 2 doses 24 hours apart
PlaceboDrug
PLACEBO: IM x 2 doses 24 hours apart
| Study group | Role | What they receive |
|---|---|---|
| Active Study Group | Active comparator | STEROID: Betamethasone; 12 mg intramuscularly x 2 doses 24 hours apart Receives: Drug: Betamethasone |
| Placebo Group | Placebo comparator | PLACEBO: IM x 2 doses 24 hours apart Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Hydrops Fetalis
Measured: Delivery, up to approximately 20 weeks post-enrollment
Secondary outcomes (2)
Comparison of CCAM Size in Mid-trimester Fetuses (Study/Administration vs Control/Placebo)
Measured: 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
Measured: 30 days after delivery (up to approximately 24 weeks post-enrollment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2008 (estimated)
- Study startApr 2008
- Primary completionSep 2011
- Study completionSep 2011
Why it stopped: Recruitment has been poor. Study drug is being offered as part of standard care of women carrying a pregnancy diagnosed with CCAM
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (11)Papers the sponsor has linked to this study.
- Other trials for Congenital Cystic Adenomatoid MalformationStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Timothy M Crombleholme, MD, Principal investigator, Children's Hospital of CincinnatiDouglas Wilson, MD, Principal investigator, Children's Hospital of PhiladelphiaHanmin Lee, MD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00670956
- Sponsor's protocol ID
- 10-03705
- Acronym
- CCAM Steroids
- Status last verified
- Mar 2015
- First submitted
- 30 Apr 2008
- Last updated
- 30 Mar 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Vu L, Tsao K, Lee H, Nobuhara K, Farmer D, Harrison M, Goldstein RB. Characteristics of congenital cystic adenomatoid malformations associated with nonimmune hydrops and outcome. J Pediatr Surg. 2007 Aug;42(8):1351-6. doi: 10.1016/j.jpedsurg.2007.03.039.
PubMed 17706495Schumacher A, Sidor J, Buhling KJ. [Continuous glucose monitoring using the glucose sensor CGMS in metabolically normal pregnant women during betamethasone therapy for fetal respiratory distress syndrome]. Z Geburtshilfe Neonatol. 2006 Oct;210(5):184-90. doi: 10.1055/s-2006-951743. German.
PubMed 17099841Peltoniemi OM, Kari MA, Tammela O, Lehtonen L, Marttila R, Halmesmaki E, Jouppila P, Hallman M; Repeat Antenatal Betamethasone Study Group. Randomized trial of a single repeat dose of prenatal betamethasone treatment in imminent preterm birth. Pediatrics. 2007 Feb;119(2):290-8. doi: 10.1542/peds.2006-1549.
PubMed 17272618Roberts D, Dalziel S. Antenatal corticosteroids for accelerating fetal lung maturation for women at risk of preterm birth. Cochrane Database Syst Rev. 2006 Jul 19;(3):CD004454. doi: 10.1002/14651858.CD004454.pub2.
PubMed 16856047Neilson JP. Antenatal corticosteroids for accelerating fetal lung maturation for women at risk of preterm birth. Obstet Gynecol. 2007 Jan;109(1):189-90. doi: 10.1097/01.aog.0000251610.51286.b1. No abstract available.
PubMed 17233108Tsao K, Hawgood S, Vu L, Hirose S, Sydorak R, Albanese CT, Farmer DL, Harrison MR, Lee H. Resolution of hydrops fetalis in congenital cystic adenomatoid malformation after prenatal steroid therapy. J Pediatr Surg. 2003 Mar;38(3):508-10. doi: 10.1053/jpsu.2003.50089.
PubMed 12632377Arca MJ, Teich S. Current controversies in perinatal care: fetal versus neonatal surgery. Clin Perinatol. 2004 Sep;31(3):629-48. doi: 10.1016/j.clp.2004.03.016.
PubMed 15325542Wilson RD, Baxter JK, Johnson MP, King M, Kasperski S, Crombleholme TM, Flake AW, Hedrick HL, Howell LJ, Adzick NS. Thoracoamniotic shunts: fetal treatment of pleural effusions and congenital cystic adenomatoid malformations. Fetal Diagn Ther. 2004 Sep-Oct;19(5):413-20. doi: 10.1159/000078994.
PubMed 15305098Knox EM, Kilby MD, Martin WL, Khan KS. In-utero pulmonary drainage in the management of primary hydrothorax and congenital cystic lung lesion: a systematic review. Ultrasound Obstet Gynecol. 2006 Oct;28(5):726-34. doi: 10.1002/uog.3812.
PubMed 17001747Davenport M, Warne SA, Cacciaguerra S, Patel S, Greenough A, Nicolaides K. Current outcome of antenally diagnosed cystic lung disease. J Pediatr Surg. 2004 Apr;39(4):549-56. doi: 10.1016/j.jpedsurg.2003.12.021.
PubMed 15065026Miller JA, Corteville JE, Langer JC. Congenital cystic adenomatoid malformation in the fetus: natural history and predictors of outcome. J Pediatr Surg. 1996 Jun;31(6):805-8. doi: 10.1016/s0022-3468(96)90138-4.
PubMed 8783108
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigation of Prenatal Steroids for Treatment of Prenatally Diagnosed CCAMs
- ClinicalTrials.gov ID
- NCT00670956
- Acronym / sponsor ID
- CCAM Steroids / 10-03705
- Keywords
- prenatal steroids, hydrops, congenital cystic adenomatoid malformation of the lung, prenatal intervention, betamethasone, prenatal diagnosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Recruitment has been poor. Study drug is being offered as part of standard care of women carrying a pregnancy diagnosed with CCAM
- Enrolment
- 1 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- Children's Hospital Medical Center, Cincinnati, Children's Hospital of Philadelphia
- Study start
- Apr 2008
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 2 May 2008 (estimated)
- Results
- Posted 30 Mar 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active Study Group | Active comparator | Drug: BetamethasoneSTEROID: Betamethasone; 12 mg intramuscularly x 2 doses 24 hours apart |
| Placebo Group | Placebo comparator | Drug: PlaceboPLACEBO: IM x 2 doses 24 hours apart |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- GA < 26 weeks
- Maternal age > 18 years of age
- Singleton pregnancy
- Normal chromosomes
- CCAM volume to head circumference ratio (CVR) > 1.4
Key exclusion criteria (8 listed)
- Maternal diabetes or use of insulin
- Preterm labor
- Multiple congenital anomalies with CCAM
- Chromosomal anomaly with CCAM
- Multiple gestation pregnancy with CCAM
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Betamethasone12 mg intramuscularly x 2 doses 24 hours apart | Drug | Active Study Group |
| PlaceboPLACEBO: IM x 2 doses 24 hours apart | Drug | Placebo Group |
Primary outcome
Incidence of Hydrops Fetalis
Time frame: Delivery, up to approximately 20 weeks post-enrollment
2 secondary outcomes
Comparison of CCAM Size in Mid-trimester Fetuses (Study/Administration vs Control/Placebo)
Time frame: Baseline, Delivery (up to approximately 20 weeks post-enrollment)
Survival at One-month Between Study and Control Groups.
Time frame: 30 days after delivery (up to approximately 24 weeks post-enrollment)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 17706495Vu L, Tsao K, Lee H, Nobuhara K, Farmer D, Harrison M, Goldstein RB. Characteristics of congenital cystic adenomatoid malformations associated with nonimmune hydrops and outcome. J Pediatr Surg. 2007 Aug;42(8):1351-6. doi: 10.1016/j.jpedsurg.2007.03.039.
- Background: PubMed 17099841Schumacher A, Sidor J, Buhling KJ. [Continuous glucose monitoring using the glucose sensor CGMS in metabolically normal pregnant women during betamethasone therapy for fetal respiratory distress syndrome]. Z Geburtshilfe Neonatol. 2006 Oct;210(5):184-90. doi: 10.1055/s-2006-951743. German.
- Background: PubMed 17272618Peltoniemi OM, Kari MA, Tammela O, Lehtonen L, Marttila R, Halmesmaki E, Jouppila P, Hallman M; Repeat Antenatal Betamethasone Study Group. Randomized trial of a single repeat dose of prenatal betamethasone treatment in imminent preterm birth. Pediatrics. 2007 Feb;119(2):290-8. doi: 10.1542/peds.2006-1549.
- Background: PubMed 16856047Roberts D, Dalziel S. Antenatal corticosteroids for accelerating fetal lung maturation for women at risk of preterm birth. Cochrane Database Syst Rev. 2006 Jul 19;(3):CD004454. doi: 10.1002/14651858.CD004454.pub2.
- Background: PubMed 17233108Neilson JP. Antenatal corticosteroids for accelerating fetal lung maturation for women at risk of preterm birth. Obstet Gynecol. 2007 Jan;109(1):189-90. doi: 10.1097/01.aog.0000251610.51286.b1. No abstract available.
- Background: PubMed 12632377Tsao K, Hawgood S, Vu L, Hirose S, Sydorak R, Albanese CT, Farmer DL, Harrison MR, Lee H. Resolution of hydrops fetalis in congenital cystic adenomatoid malformation after prenatal steroid therapy. J Pediatr Surg. 2003 Mar;38(3):508-10. doi: 10.1053/jpsu.2003.50089.
- Background: PubMed 15325542Arca MJ, Teich S. Current controversies in perinatal care: fetal versus neonatal surgery. Clin Perinatol. 2004 Sep;31(3):629-48. doi: 10.1016/j.clp.2004.03.016.
- Background: PubMed 15305098Wilson RD, Baxter JK, Johnson MP, King M, Kasperski S, Crombleholme TM, Flake AW, Hedrick HL, Howell LJ, Adzick NS. Thoracoamniotic shunts: fetal treatment of pleural effusions and congenital cystic adenomatoid malformations. Fetal Diagn Ther. 2004 Sep-Oct;19(5):413-20. doi: 10.1159/000078994.
- Background: PubMed 17001747Knox EM, Kilby MD, Martin WL, Khan KS. In-utero pulmonary drainage in the management of primary hydrothorax and congenital cystic lung lesion: a systematic review. Ultrasound Obstet Gynecol. 2006 Oct;28(5):726-34. doi: 10.1002/uog.3812.
- Background: PubMed 15065026Davenport M, Warne SA, Cacciaguerra S, Patel S, Greenough A, Nicolaides K. Current outcome of antenally diagnosed cystic lung disease. J Pediatr Surg. 2004 Apr;39(4):549-56. doi: 10.1016/j.jpedsurg.2003.12.021.
- Background: PubMed 8783108Miller JA, Corteville JE, Langer JC. Congenital cystic adenomatoid malformation in the fetus: natural history and predictors of outcome. J Pediatr Surg. 1996 Jun;31(6):805-8. doi: 10.1016/s0022-3468(96)90138-4.
- Fetal Care Center of Cincinnati Children's HospitalLink provided by the sponsor.
- Fetal Treatment Center at the University of California, San FranciscoLink provided by the sponsor.
Related trials for Congenital Cystic Adenomatoid Malformation
Recruiting
Atomized Inhalation ICG for Treatment of Congenital Lung Malformations
Pulmonary Sequestration, Congenital Pulmonary Airway Malformation
Recruiting
The COllaborative Neonatal Network for the First CPAM Trial
Congenital Pulmonary Airway Malformation
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Timothy M Crombleholme, MD principal investigator
Children's Hospital of Cincinnati
Douglas Wilson, MD principal investigator
Children's Hospital of Philadelphia
Hanmin Lee, MD principal investigator
University of California, San Francisco
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Betamethasone in people with Congenital Cystic Adenomatoid Malformation, run by University of California, San Francisco at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Mar 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Hydrops Fetalis (time frame: Delivery, up to approximately 20 weeks post-enrollment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00670956, which also lists 11 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2015.