Clinical trials: Lymphatic Malformations, active
Hereditary Hemorrhagic Telangiectasia, HHT
CHORUS - Comprehensive HHT Outcomes Registry of the United States (Formerly OUR HHT Registry)
The goal of this study is to better understand HHT, the symptoms and complications it causes ("outcomes") and how the disease impacts people's lives.
- Conditions
- Hereditary Hemorrhagic Telangiectasia, HHT, Arteriovenous Malformation of Brain
- Trial name
- NCT04150822
- Testing
- Registry and Saliva sample
- Where
- Canada
- Volunteers
- 296 people
- Sponsor
- Unity Health Toronto
Tuberous Sclerosis, Lymphangioleiomyomatosis
TSC Biosample Repository and Natural History Database
The TSC Biosample Repository collects and stores samples of blood, DNA, and tissues that scientists can request to use in their research. The samples we collect are all linked to clinical data in the TSC Natural History Database.
- Trial name
- NCT05676099
- Testing
- Phlebotomy, Buccal (cheek) swab, Genetic Testing
- Where
- 26 sites in United States, Canada
- Volunteers
- 5,000 people
- Sponsor
- National Tuberous Sclerosis Association
Lymphatic Abnormality
Injection Sclerotherapy With Bleomycin VS Doxycycline in Patients With Lymphatic Malformations
This is a randomized clinical trial comparing the safety and effectiveness of injection sclerotherapy using bleomycin versus doxycycline in children under 12 years with lymphatic malformations.
- Trial name
- NCT07320001
- Testing
- Bleomycin, doxycycline (tetracycline)
- Volunteers
- 92 people
- Sponsor
- Basharat Hussain
Hereditary Hemorrhagic Telangiectasia
Cardiac Evaluation in Hereditary Hemorrhagic Telangiectasia
Hereditary haemorrhagic telangiectasia (HHT) is a rare autosomal dominant genetic disease associated with mutations in genes encoding proteins of the transforming growth factor β (TGF-β) family, i.e.
- Trial name
- CARDI-HHT (NCT07101575)
- Testing
- Transthoracic echocardiography
- Volunteers
- 380 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA)
Lymphatic Anomalies Registry for the Assessment of Outcome Data
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical…
- Conditions
- Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA), Central Conducting Lymphatic Anomaly, CLOVES Syndrome
- Trial name
- NCT02399527
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Boston Children's Hospital
Vascular Anomaly, Generalized Lymphatic Anomaly
Identification of Biomarkers for Patients with Vascular Anomalies
The study will use blood (serum and plasma) and tissue obtained from participants undergoing prescribed surgical resection of vascular anomalies of interest proposed in this study.
- Conditions
- Vascular Anomaly, Generalized Lymphatic Anomaly, Kaposiform Hemangioendothelioma, Kaposiform Lymphangiomatosis
- Trial name
- NCT03001180
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Cavernous Malformation, Cerebral
Daratumumab for Familial Cerebral Cavernous Malformations: A Single-Arm Safety and Efficacy Study
Cerebral cavernous malformation (CCM) is a common vascular abnormality of the brain, affecting 0.1%-0.5% of people. It often causes recurrent brain hemorrhages, epilepsy, and neurological impairments, with surgery being the main…
- Trial name
- CCM-DS01 (NCT07026604)
- Testing
- Daratumumab
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Beijing Tiantan Hospital
Hereditary Hemorrhagic Telangiectasia, Pulmonary Arterial Hypertension
DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia
This is a Phase 1/2, randomized, double-blind, placebo-controlled, first-in-human study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of subcutaneously administered DIAG723 in adult patients with…
- Trial name
- DIAMOND (NCT07623525)
- Testing
- DIAG723
- Where
- 11 sites in Australia, Canada and 1 more
- Volunteers
- 93 people
- Sponsor
- Diagonal Therapeutics, Inc.
Telangiectasia, Hereditary Hemorrhagic
Molecular Studies on Hereditary Haemorrhagic Telangiectasia Families
This study will examine genes involved in the vascular dysplasia Hereditary haemorrhagic telangiectasia i(HHT)
- Trial name
- NCT00230620
- Where
- United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Imperial College London
Hereditary Haemorrhagic Telangiectasia, PFO - Patent Foramen Ovale
Non-Invasive Detection of Pulmonary Right-to-Left Shunts Using the SONAS Ultrasound Device
The goal of this clinical trial is to learn how well the SONAS ultrasound device can detect right-to-left shunts of adults with hereditary hemorrhagic telangiectasia (HHT) who are already scheduled for a contrast bubble…
- Conditions
- Hereditary Haemorrhagic Telangiectasia, PFO - Patent Foramen Ovale, Pulmonary Arteriovenous Malformation
- Trial name
- SONAS (NCT07414446)
- Testing
- Transcranial Ultrasound Device
- Where
- Netherlands
- Volunteers
- 40 people
- Sponsor
- St. Antonius Hospital
Tuberous Sclerosis Complex, Lymphangioleiomyomatosis
Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)
This study is an observational registry designed to gather information about Tuberous Sclerosis Complex (TSC) and Lymphangioleiomyomatosis (LAM) in pregnant women and their child.
- Trial name
- NCT06160310
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- David M. Ritter
Velocardiofacial Syndrome, Williams Syndrome
The Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome
The purpose of this study is to investigate the Psychiatric and Cognitive Phenotypes in Velocardiofacial Syndrome (VCFS), Williams Syndrome (WS)and Fragile X Syndrome Characterization, Treatment and Examining the Connection to…
- Conditions
- Velocardiofacial Syndrome, Williams Syndrome, Fragile X Syndrome
- Trial name
- VCFS (NCT00768820)
- Testing
- methylphenidate, fluoxetin, risperidone
- Where
- Israel
- Volunteers
- 400 people
- Sponsor
- The Chaim Sheba Medical Center
Cerebral Cavernous Malformation, Cavernous Angioma
Biomarkers of CASH
The project aims to develop prognostic and diagnostic blood tests for symptomatic brain hemorrhage in patients diagnosed with cavernous angiomas, a critical clinical challenge in a disease affecting more than a million Americans.
- Conditions
- Cerebral Cavernous Malformation, Cavernous Angioma, Hemorrhagic Microangiopathy
- Trial name
- NCT04467489
- Testing
- observational
- Where
- 4 sites in United States
- Volunteers
- 1,040 people, healthy volunteers welcome
- Sponsor
- University of Chicago
Telangiectasia, Hereditary Hemorrhagic
Case Notes Review on Patients With Hereditary Haemorrhagic Telangiectasia
This observational study is for individuals with Hereditary haemorrhagic telangiectasia and pulmonary arteriovenous malformations that are reviewed at the Hammersmith Hospital, London.
- Trial name
- NCT00230685
- Where
- United Kingdom
- Volunteers
- 2,000 people
- Sponsor
- Imperial College London
Vascular Anomaly, Osler Rendu Disease
Pregnancy in Women With Rare Multisystemic Vascular Diseases: COGRare5 Study
There are no prospective studies of pregnancies for the diseases studied here in (Heredity Hemorrhagic Telangiectasia, Marfan syndrome or related, primary lower limb lymphedema, superficial arteriovenous malformations, and…
- Conditions
- Vascular Anomaly, Osler Rendu Disease, Marfan Syndrome or Related, Lymphedema Primary
- Trial name
- COGRare5 (NCT04194619)
- Testing
- Questionnaire
- Where
- 37 sites in France
- Volunteers
- 400 people
- Sponsor
- Hospices Civils de Lyon
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines.
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of
- Trial name
- NCT06092346
- Where
- United States
- Volunteers
- 999 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Hereditary Hemorrhagic Telangiectasia, Rendu Osler Disease
Prospective Study of Antiplatelet and Anticoagulation Therapy in Hereditary Haemorrhagic Telangiectasia
The goal of this clinical trial is to evaluate in real life, in patients with Hereditary Hemorrhagic Telangiectasia (HHT), the tolerance of the strategy of use of anticoagulant and/or antiplatelet, by comparing a new exposure…
- Trial name
- PROPLACO-Tel (NCT05641142)
- Testing
- monitoring the use of anticoagulant and/or antiplatelet therapy in patients with osler rendering disease
- Where
- 18 sites in France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Clermont-Ferrand
Hereditary Hemorrhagic Telangiectasia
A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
The primary objective of this trial is to assess the efficacy of engasertib 40 mg once daily (QD) in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of double-blind treatment in participants with…
- Trial name
- NCT07743671
- Testing
- Engasertib
- Where
- 4 sites in United States
- Volunteers
- 240 people
- Sponsor
- Vaderis Therapeutics AG
Lymphatic Malformation, Pediatric
Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations
To evaluate the efficacy of Rapamycin in extended cervicofacial lymphatic malformations in pediatric patients. Rapamycin is administered oral for a 6 month period.
- Trial name
- RAPAMALYMPH (NCT03243019)
- Testing
- rapamycin, MRI, Rapamycin dosage
- Where
- 2 sites in France
- Volunteers
- 28 people
- Sponsor
- University Hospital, Lille
Hereditary Haemorrhagic Telangiectasia (HHT)
Quality of Life in Pediatric Participants With HHT
This observational study evaluates health-related quality of life (HR-QoL) in pediatric and young adult patients aged 2-25 years with Hereditary Hemorrhagic Telangiectasia (HHT).
- Trial name
- NCT07474428
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Ashley Nelson