Malformation Clinical Trials

87 recruitingLast updated: May 13, 2026

There are 87 actively recruiting malformation clinical trials across 22 countries. Studies span Not Applicable, Phase 2, Phase 3, Phase 1. Top locations include Paris, France, Boston, Massachusetts, United States, Beijing, Beijing Municipality, China. Updated daily from ClinicalTrials.gov.


Malformation Trials at a Glance

87 actively recruiting trials for malformation are listed on ClinicalTrialsFinder across 6 cities in 22 countries. The largest study group is Not Applicable with 24 trials, with the heaviest enrollment activity in Paris, Boston, and Beijing. Lead sponsors running malformation studies include Fondation Ophtalmologique Adolphe de Rothschild, Beijing Tiantan Hospital, and Fondazione Policlinico Universitario Agostino Gemelli IRCCS.

Browse malformation trials by phase

Treatments under study

About Malformation Clinical Trials

Looking for clinical trials for Malformation? There are currently 2 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Malformation trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Malformation clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 87 trials

Recruiting
Phase 2Phase 3

Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations Associated With a PIK3CA Mutation.

Lymphatic Malformations
Novartis Pharmaceuticals232 enrolled56 locationsNCT05948943
Recruiting
Phase 2

A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation

PIK3CA MutationVascular MalformationsPIK3CA-Related Overgrowth Spectrum (PROS)+4 more
Relay Therapeutics, Inc.277 enrolled31 locationsNCT06789913
Recruiting
Phase 2

A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations

PI3K Gene MutationKRAS G12DVascular Anomalies+15 more
Murdoch Childrens Research Institute50 enrolled2 locationsNCT05983159
Recruiting

MEHMO Natural History and Biomarkers

ObesityEpilepsyHypogonadisms+3 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)150 enrolled1 locationNCT06019182
Recruiting

Cera™ Vascular Plug System Post-Market Clinical Follow-Up

Arteriovenous FistulaAneurysmPortal Hypertension+3 more
Lifetech Scientific (Shenzhen) Co., Ltd.132 enrolled9 locationsNCT06099015
Recruiting

Lymphatic Anomalies Registry for the Assessment of Outcome Data

Lymphatic MalformationCLOVES SyndromeKlippel Trenaunay Syndrome+7 more
Boston Children's Hospital1,000 enrolled1 locationNCT02399527
Recruiting

Pulmonary Embolism and Right-to-Left Shunts

Pulmonary Embolism (PE)StrokePulmonary Arteriovenous Malformation+3 more
Tufts Medical Center256 enrolled1 locationNCT07519876
Recruiting

Neurosurgical Outcome Network

DepressionTrigeminal NeuralgiaObsessive-Compulsive Disorder+20 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta4,500 enrolled27 locationsNCT06724029
Recruiting
Not Applicable

Spatiotemporal Entrainment Neuromodulation Targeting Cerebello-Cerebral Circuits to Enhance Cognitive and Social Rehabilitation in Cerebellar Diseases

Cerebellar DegenerationCerebellar Malformation
I.R.C.C.S. Fondazione Santa Lucia60 enrolled2 locationsNCT07500103
Recruiting

Characterization and Contribution of Genome-wide DNA Methylation (DNA Methylation Episignatures) in Rare Diseases With Prenatal Onset

Congenital MalformationRare Fetal Genetic Diseases
Assistance Publique - Hôpitaux de Paris63 enrolled1 locationNCT06475651
Recruiting

Outcomes in ARMs: Comparison Between Surgical Techniques in Patients With Perineal or Vestibular Fistula - a Multicenter Italian Study

Anorectal Malformations
Meyer Children's Hospital IRCCS500 enrolled21 locationsNCT07438691
Recruiting
Not Applicable

PHIL® Embolic System Pediatric IDE

Intracranial Arteriovenous MalformationsArterio-venous Fistula
Alejandro Berenstein15 enrolled1 locationNCT03731000
Recruiting

Respiratory and Psychological Impact of Elective Surgery of Congenital Lung Malformations

Congenital Lung Malformations
Assistance Publique - Hôpitaux de Paris434 enrolled1 locationNCT06424392
Recruiting

Pregnancy in Women With Rare Multisystemic Vascular Diseases: COGRare5 Study

Arteriovenous MalformationsVascular AnomalyOsler Rendu Disease+3 more
Hospices Civils de Lyon400 enrolled36 locationsNCT04194619
Recruiting
Phase 2

Weekly Sirolimus Therapy

Lymphatic MalformationVenous Malformation
Medical University of South Carolina24 enrolled1 locationNCT04861064
Recruiting

Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies

Noonan SyndromeCardiofaciocutaneous SyndromeCostello Syndrome+3 more
National Cancer Institute (NCI)500 enrolled2 locationsNCT04888936
Recruiting
Phase 2

Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)

Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)
Centre Hospitalier Universitaire Dijon20 enrolled13 locationsNCT05577754
Recruiting
Not Applicable

A Prospective Study on the Treatment of cLM Based on ICG Imaging

Lymphatic Malformation
Nanjing Children's Hospital110 enrolled1 locationNCT06275022
Recruiting
Not Applicable

Efficacy & Safety of LOBO™ Vascular Occlusion Device for Embolization of Pulmonary Arteriovenous Malformations

Pulmonary Arteriovenous Malformations
University of North Carolina, Chapel Hill30 enrolled1 locationNCT06735976
Recruiting

Baylor Continence Scale Validity in Anorectal Malformation

Anorectal Malformations
Izmir Bakircay University230 enrolled1 locationNCT07029984