Clinical trials: Lymphatic Malformations
Hereditary Hemorrhagic Telangiectasia
Phase III Randomized, Placebo-Controlled, Crossover Study of Soy Protein Isolate for Hereditary Hemorrhagic Telangiectasia
OBJECTIVES: I. Evaluate migraine prophylaxis with soy protein isolate in patients with hereditary hemorrhagic telangiectasia. II.
- Trial name
- NCT00004654
- Testing
- soy protein isolate
- Volunteers
- 60 people
- Sponsor
- National Center for Research Resources (NCRR)
Leiomyomatosis
Official Record of Patients Diagnosed With Lymphangioleiomyomatosis (LAM)
Pulmonary lymphoangioleiomyomatosis (LAM) is a rare destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy for the disease and the prognosis is poor.
- Trial name
- NCT00001869
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Lymphatic Diseases, Lymphatic Abnormalities
A Prospective Natural History Study of Lymphatic Anomalies
Background: The lymphatic system is a network of vessels that carry a clear fluid called lymph through the body. Problems in the lymphatic system can cause pain, fluid buildup, and issues with immunity.
- Trial name
- NCT05731141
- Where
- 2 sites in United States
- Volunteers
- 1,200 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Cerebral Cavernous Malformations
Effect of Oral Propranolol on mRNA Expresssion in Symptomatic Cavernous Malformation
This is a single center, randomized, trial that will enroll twenty (n=20) patients with a diagnosis of symptomatic cavernous malformations who are planned candidates for surgical resection by one of the investigators, and who…
- Trial name
- NCT03474614
- Testing
- Propranolol, DNA and RNA Analysis
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Joseph's Hospital and Medical Center, Phoenix
Cerebral Cavernous Malformation
Atorvastatin Treatment of Cavernous Angiomas With Symptomatic Hemorrhage Exploratory Proof of Concept (AT CASH EPOC) Trial
This phase I/II randomized, placebo-controlled, double-blinded, single-site clinical trial is designed to investigate the effect of a prolonged course of atorvastatin versus placebo on CCM lesional iron deposition assessed by…
- Trial name
- AT CASH EPOC (NCT02603328)
- Testing
- Atorvastatin
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- University of Chicago
Hemorrhagic Hereditary Telangiectasia (HHT)
Efficacy and Safety of a 0.1% Tacrolimus Nasal Ointment as a Treatment for Epistaxis in Hemorrhagic Hereditary Telangiectasia (HHT)
The recognized manifestations of HHT are all due to abnormalities in vascular structure. Epistaxis due to telangiectases formation is spontaneous, very variable, recurrent in 90% of patients, and associated with severe anemia in…
- Trial name
- TACRO (NCT03152019)
- Testing
- Protopic® (Tacrolimus) 0.1% ointment
- Where
- 3 sites in France
- Volunteers
- 50 people
- Sponsor
- Hospices Civils de Lyon
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
Hereditary Hemorrhagic Telangiectasia, HHT
Genotype-phenotype Association in Hereditary Hemorrhagic Telangiectasia
The present project aims to study the inflammatory and endothelial responses involved in the differences in clinical events related to both genotypes (ENG vs. ACVRL1) in HHT.
- Conditions
- Hereditary Hemorrhagic Telangiectasia, HHT, Rendu Osler Weber Disease
- Trial name
- Generate-HHT (NCT05550376)
- Testing
- Registry
- Where
- Spain
- Volunteers
- 85 people
- Sponsor
- Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
Pulmonary Arteriovenous Malformation
Microvascular Plug (MVP) for the Treatment of Pulmonary ArterioVenous Malformations (PAVMs)
The purpose of this study is to compare the use of the Micro Vascular Plug (MVP) system and other embolic devices in treatment of PAVMs.
- Trial name
- NCT04396041
- Testing
- Microvascular Plug (MVP), Amplatzer Vascular Plug (AVP), Detachable coils
- Where
- United States
- Volunteers
- 223 people
- Sponsor
- Johns Hopkins University
Prader-Willi Syndrome, PWS-like Syndrome
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome.
- Conditions
- Prader-Willi Syndrome, PWS-like Syndrome, Silver Russel Syndrome, Congenital Hypopituitarism
- Trial name
- GROW UR GENES (NCT04463316)
- Testing
- Retrospective file studies
- Where
- Netherlands
- Volunteers
- 600 people
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Vascular Malformations, Arteriovenous Malformations
A Study to Evaluate the Efficacy and Safety of Everolimus in Patients With Teratment-refractory Vascular Anomalies
Background and Objectives Vascular anomalies are a heterogeneous group of disorders classified into vascular tumors and vascular malformations according to the ISSVA classification.
- Conditions
- Vascular Malformations, Arteriovenous Malformations, Venous Malformation, Lymphangioma
- Trial name
- NCT07477548
- Testing
- Everolimus
- Volunteers
- 67 people
- Sponsor
- Yonsei University
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Vasculitic Neuropathy
Polyneuropathy, Impairments and Physical Activity - The PolyImPAct Study
The project aims to investigate the validity, and reliability of outcome measures of muscle strength, functioning (gait, balance, and fine motor skills), physical activity, and patient-reported outcome measures of functioning…
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), Vasculitic Neuropathy, POEMS Syndrome, Multifocal Motor Neuropathy
- Trial name
- PolyImPAct (NCT06040567)
- Testing
- Validity and reliability of outcome measures in polyneuropathy, Healthy controls
- Where
- Denmark
- Volunteers
- 520 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Williams Syndrome, Angelman Syndrome
Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes
OBJECTIVES: I. Investigate phenotype and genotype correlations in patients with Smith-Magenis syndrome (SMS) associated with del(17p11.2). II.
- Conditions
- Williams Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Shprintzen Syndrome
- Trial name
- NCT00004351
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Osler Rendu Disease, Hereditary Hemorrhagic Telangiectasia
Evaluation of Obstetrical and Neonatal Complications in Hereditary Haemorrhagic Telangiectasia (HHT)
Hereditary Haemorrhagic Telangiectasia (HHT) is a rare inherited genetic disease of autosomal dominant inheritance with a prevalence of about 1/5000.
- Trial name
- CONCERTO (NCT03691142)
- Testing
- Questionnaire
- Where
- France
- Volunteers
- 207 people
- Sponsor
- Hospices Civils de Lyon
Hereditary Hemorrhagic Telangiectasia, Gastrointestinal Hemorrhage
Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber
The purpose of this study is to determine whether long-acting octreotide is safe and effective in the treatment of patients with Rendu-Osler-Weber (e.g. HHT).
- Conditions
- Hereditary Hemorrhagic Telangiectasia, Gastrointestinal Hemorrhage, Anemia
- Trial name
- ROW (NCT02874326)
- Testing
- Octreotide LAR
- Where
- 2 sites in Netherlands
- Volunteers
- 15 people
- Sponsor
- Radboud University Medical Center
HHT, Hereditary Hemorrhagic Telangiectasia
Intranasal Bevacizumab for HHT-Related Epistaxis
This is a randomized, controlled, double-blind, placebo-controlled trial of intranasal Avastin (bevacizumab) injection versus saline control for control of HHT-related epistaxis when used in conjunction with bipolar…
- Conditions
- HHT, Hereditary Hemorrhagic Telangiectasia, Epistaxis, Nose Bleeds
- Trial name
- NCT02389959
- Testing
- Bevacizumab
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Stanford University
Cavernous Angioma, Familial, Cerebral Cavernous Malformations
Modifiers of Disease Severity in Cerebral Cavernous Malformations
Cerebral cavernous malformations (CCMs) are clusters of abnormal blood vessels in the brain and spine. CCMs can bleed and cause strokes, seizures, and headaches.
- Conditions
- Cavernous Angioma, Familial, Cerebral Cavernous Malformations, Cerebral Cavernous Hemangioma
- Trial name
- NCT01764529
- Where
- 7 sites in United States
- Volunteers
- 789 people
- Sponsor
- University of California, San Francisco
Hereditary Hemorrhagic Telangiectasia
Bevacizumab In Hereditary Hemorrhagic Telangiectasia
This research study is studying to see whether bevacizumab may treat chronic bleeding and iron deficiency anemia in Hereditary Hemorrhagic Telangiectasia (HHT).
- Trial name
- TrUST-HHT (NCT04404881)
- Testing
- Bevacizumab (Avastin®)
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Hanny Al-Samkari, MD
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
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