Congenital pulmonary airway malformation

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2Active trials59Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Congenital pulmonary airway malformation (CPAM), formerly known as congenital cystic adenomatoid malformation (CCAM), is a rare developmental abnormality of the lung in which an abnormal mass of disorganized lung tissue forms during fetal development. The malformation consists of cystic or solid lesions that arise from abnormal branching of the developing airways and replace normal lung parenchyma. CPAMs are classified into several types (Types 0 through 4) based on the size of the cysts and the tissue of origin, with Type 1 (large cysts) and Type 2 (smaller cysts) being the most common. The condition primarily affects the respiratory system, typically involving one lobe of the lung, though bilateral involvement can rarely occur. CPAM is most often detected prenatally on routine ultrasound or presents in the neonatal period with respiratory distress, including tachypnea, cyanosis, and difficulty breathing. Some cases may remain asymptomatic and be discovered incidentally later in childhood or even adulthood, sometimes presenting with recurrent pulmonary infections. In severe prenatal cases, large lesions can cause mediastinal shift, compression of the normal lung, and fetal hydrops, which carries a poor prognosis without intervention. Smaller lesions may spontaneously regress during pregnancy, though they typically persist after birth. The primary treatment for symptomatic CPAM is surgical resection, usually lobectomy, which is generally curative. For asymptomatic cases detected prenatally or in infancy, the management approach remains debated, though many centers recommend elective surgical removal due to the risk of recurrent infections and a small but recognized risk of malignant transformation (particularly pleuropulmonary blastoma in Type 4 lesions). In severe fetal cases with hydrops, prenatal interventions such as thoracoamniotic shunting or fetal surgery may be considered. Outcomes after surgical resection are generally excellent, with most children achieving normal lung function and development.

Also known as:

Clinical phenotype terms— hover any for plain English:

Abnormal pleura morphologyHP:0002103
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

FDA & Trial Timeline

6 events
Mar 2026Optimising Breathing Support at Extubation in Very Preterm Infants: A Clinical Study

Murdoch Childrens Research Institute — NA

TrialNOT YET RECRUITING
Sep 2025"Effectiveness of Delivery Room Bubble CPAP in Preterm Infants With Respiratory Distress

Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh — NA

TrialENROLLING BY INVITATION
Mar 2024Atomized Inhalation ICG for Treatment of Congenital Lung Malformations

Jiexiong Feng — NA

TrialRECRUITING
Jan 2023The COllaborative Neonatal Network for the First CPAM Trial

Erasmus Medical Center — NA

TrialRECRUITING
Sep 2021Prospective Study of Pregnancy in Women With Cystic Fibrosis

Amalia Magaret

TrialACTIVE NOT RECRUITING
Jul 2011Impact of Early Lung Physiology, Viral Infections and the Microbiota on the Development and Progression of Lung Disease in Children With Cystic Fibrosis

Insel Gruppe AG, University Hospital Bern

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Congenital pulmonary airway malformation.

2 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

2 recruitingView all trials with filters →
N/A2 trials
The COllaborative Neonatal Network for the First CPAM Trial
N/A
Actively Recruiting
PI: Rene MH Wijnen, professor (Head of department Pediatric Surgery, Erasmus MC S) · Sites: Nijmegen, Gelderlanf; Rotterdam, South Holland · Age: 01 yrs
Atomized Inhalation ICG for Treatment of Congenital Lung Malformations
N/A
Actively Recruiting
PI: Tianqi Zhu, MD (Tongji Hospital) · Sites: Wuhan, Hubei · Age: 014 yrs

Specialists

Showing 25 of 59View all specialists →
AM
Arjunan Ganesh, MD
PHILADELPHIA, PA
Specialist
PI on 1 active trial1 Congenital pulmonary airway malformation publication
AP
Antonius Mulder, MD, PhD,prof
Specialist
PI on 1 active trial
SP
Stijn L Verhulst, MD, PhD,prof
Specialist
PI on 1 active trial
GM
Georg Schmolzer, MD,PhD
Specialist
PI on 1 active trial
ML
M Lacher
Specialist
1 Congenital pulmonary airway malformation publication
OM
Olivia McKinney
PARKESBURG, PA
Specialist
1 Congenital pulmonary airway malformation publication
OA
O Aubert
Specialist
1 Congenital pulmonary airway malformation publication
SS
S Schweitzer
Specialist
1 Congenital pulmonary airway malformation publication
DL
Dan Liu
Specialist
1 Congenital pulmonary airway malformation publication
MD
Mark Davenport
Specialist
1 Congenital pulmonary airway malformation publication
MS
M Stiller
Specialist
1 Congenital pulmonary airway malformation publication
QC
Qiang Chen
VERONA, NJ
Specialist
2 Congenital pulmonary airway malformation publications
SP
Shailesh B Patel
Specialist
1 Congenital pulmonary airway malformation publication
NA
Niyi Ade-Ajayi
Specialist
1 Congenital pulmonary airway malformation publication
ZH
Zhi Hu
FLUSHING, NY
Specialist
1 Congenital pulmonary airway malformation publication
AM
Ancuta Muntean
Specialist
1 Congenital pulmonary airway malformation publication
PB
Pietro Bagolan
Specialist
2 Congenital pulmonary airway malformation publications
LB
Liliana-Elena Banias
Specialist
1 Congenital pulmonary airway malformation publication
RW
René M H Wijnen
Specialist
2 Congenital pulmonary airway malformation publications
AF
Andres Floto
Cambridge, Cambridgeshire
Specialist

Rare Disease Specialist

PI on 3 active trials
AM
Avroy A. Fanaroff, MD
CLEVELAND, OH
Specialist
PI on 3 active trials
CM
Charles R. Bauer, MD
ST GEORGE, UT
Specialist
PI on 4 active trials
WP
W. Kenneth Poole, PhD
Specialist
PI on 2 active trials
WM
Waldemar A. Carlo, MD
BIRMINGHAM, AL
Specialist
PI on 5 active trials
NM
Neil N. Finer, MD
SAN DIEGO, CA
Specialist
PI on 4 active trials

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Congenital pulmonary airway malformation.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Congenital pulmonary airway malformation

Disease timeline:

New recruiting trial: Impact of Sinus Surgery on Individuals With Cystic Fibrosis

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: Circadian Rhythm Disorders in Children With Cystic Fibrosis Under CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Modulators

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: CFTR Modulators in Pregnancy and Postpartum

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: e-Health Program to Prevent Exacerbations in the Cystic Fibrosis Population

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: Genetics of Insulin and Incretins in Cystic Fibrosis

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: A Study of Females With CF Throughout Pregnancy and Post-partum, and Follow up of Their Offspring

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: Muscle Function and Physical Activity in the Modern Era of Cystic Fibrosis

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: Oral Health Status of Cystic Fibrosis Patients. An Online Survey in Collaboration With the Vaincre la Mucoviscidose Patient Association.

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

New recruiting trial: Safety, Tolerability, and Pharmacokinetics of Multiple Dose Combinations of SION-451 and Complementary Modulators SION-2222 and SION-109 in Healthy Participants.

A new clinical trial is recruiting patients for Congenital pulmonary airway malformation

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Congenital pulmonary airway malformation

What is Congenital pulmonary airway malformation?

Congenital pulmonary airway malformation (CPAM), formerly known as congenital cystic adenomatoid malformation (CCAM), is a rare developmental abnormality of the lung in which an abnormal mass of disorganized lung tissue forms during fetal development. The malformation consists of cystic or solid lesions that arise from abnormal branching of the developing airways and replace normal lung parenchyma. CPAMs are classified into several types (Types 0 through 4) based on the size of the cysts and the tissue of origin, with Type 1 (large cysts) and Type 2 (smaller cysts) being the most common. The c

How is Congenital pulmonary airway malformation inherited?

Congenital pulmonary airway malformation follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Congenital pulmonary airway malformation typically begin?

Typical onset of Congenital pulmonary airway malformation is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for Congenital pulmonary airway malformation?

Yes — 2 recruiting clinical trials are currently listed for Congenital pulmonary airway malformation on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Congenital pulmonary airway malformation?

25 specialists and care centers treating Congenital pulmonary airway malformation are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.