Malformation Clinical Trials

92 recruitingLast updated: July 25, 2026

There are 92 actively recruiting malformation clinical trials across 24 countries. Studies span Not Applicable, Phase 2, Phase 3, Phase 1. Top locations include Paris, France, Boston, Massachusetts, United States, Tours, France. Updated daily from ClinicalTrials.gov.


Malformation Trials at a Glance

92 actively recruiting trials for malformation are listed on ClinicalTrialsFinder across 6 cities in 24 countries. The largest study group is Not Applicable with 27 trials, with the heaviest enrollment activity in Paris, Boston, and Tours. 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 92 trials

Recruiting
Not Applicable

A Phase II Trial of Fetal Embolization for Vein of Galen Malformation

Vein of Galen Malformations
Darren Orbach20 enrolled1 locationNCT07483255
Recruiting
Phase 3

A Study to Investigate Efficacy and Safety of KP-001 Compared With Placebo in Patients Aged ≥2 Years With Common VM, Common LM, or KTS/CLOVES Syndrome

Venous malformationsLymphatic MalformationsCLOVES Syndrome+1 more
Kaken Pharmaceutical150 enrolled1 locationNCT07285005
Recruiting

Comprehensive HHT Outcomes Registry of the United States (CHORUS)

Gastrointestinal BleedingTelangiectasiaArteriovenous Malformations+4 more
Cure HHT10,000 enrolled16 locationsNCT06259292
Recruiting
Phase 2Phase 3

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

Lymphatic Malformations
Novartis Pharmaceuticals232 enrolled60 locationsNCT05948943
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
Not Applicable

Exploratory Study of Cerebral Perfusion and Metabolic Alterations and Neurocognitive Disorders in Patients With Unruptured Arteriovenous Malformations Before and After Surgical Treatment

Unruptured Brain Arteriovenous Malformation
University Hospital, Montpellier10 enrolled1 locationNCT07569887
Recruiting
Phase 1Phase 2

Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)

PIK3CA-Related Overgrowth Spectrum (PROS)PIK3CA-related Vascular Malformations (PRVM)
Haihe Biopharma Co., Ltd.141 enrolled15 locationsNCT06975618
Recruiting
Not Applicable

CORRECTION OF EAR DEFORMITIES IN NEWBORNS BY MODELING, COMPARISON OF TWO PROTOCOLS

Ear Malformation
University Hospital, Caen134 enrolled1 locationNCT06431698
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
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 (The ReInspire Study)

PIK3CA MutationVascular AnomaliesVascular Malformations+5 more
Relay Therapeutics, Inc.277 enrolled34 locationsNCT06789913
Recruiting

Orthognathic Speech Pathology: Phonetic Contrasts of Patients With Dental Discrepancies Pre- and Post-Treatment Analyses

Speech DisordersDentofacial DeformitiesSpeech Sound Disorder+7 more
University of North Carolina, Chapel Hill180 enrolled1 locationNCT04117360
Recruiting

Treatment of Low-flow Venous Malformations With Electrosclerotherapy. Prospective Observational Study

Vascular Malformations
Istituto Ortopedico Rizzoli65 enrolled1 locationNCT06189092
Recruiting
Phase 2

Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations

PediatricLymphatic Malformation
University Hospital, Lille28 enrolled2 locationsNCT03243019
Recruiting
Phase 2

Safety and Efficacy Study of Intracystic TARA-002 for the Treatment of Lymphatic Malformations in Participants 6 Months to Less Than 18 Years of Age

Lymphatic Malformation
Protara Therapeutics38 enrolled10 locationsNCT05871970
Recruiting
Phase 2

Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations

Arteriovenous Malformations
Centre Hospitalier Universitaire, Amiens50 enrolled13 locationsNCT02042326
Recruiting

Patient-specific Planning of Minimally Invasive Brain Interventions Based on Vascular-hemodynamic Mapping

Intracranial AneurysmArteriovenous Malformations, Cerebral
University Hospital, Ghent500 enrolled1 locationNCT06588543
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

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