Clinical trials: Lymphatic Malformations
Telangiectasia, Hereditary Hemorrhagic
Pomalidomide for the Treatment of Bleeding in HHT
This is a Phase II placebo-controlled double-blind study of pomalidomide in patients with hereditary hemorrhagic telangiectasia (HHT) with moderate to severe epistaxis who have anemia and/or require parenteral iron infusions or…
- Trial name
- PATH-HHT (NCT03910244)
- Testing
- Pomalidomide Oral Product
- Where
- 14 sites in United States
- Volunteers
- 145 people
- Sponsor
- The Cleveland Clinic
Hereditary Hemorrhagic Telangiectasia, Epistaxis
Efficacy of Thalidomide in the Treatment of Hereditary Hemorrhagic Telangiectasia
Hereditary hemorrhagic telangiectasia (HHT) (OMIM 187300 and 600376), also known as Rendu-Osler-Weber syndrome, is an autosomal dominant disease and has a prevalence between 1:5000 and 1:8000 in different populations.
- Trial name
- THALI-HHT (NCT01485224)
- Testing
- Thalidomide
- Where
- Italy
- Volunteers
- 31 people
- Sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
Complete DiGeorge Anomaly, DiGeorge Syndrome
Thymus Transplantation Safety-Efficacy
Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into educated T cells, which fight infection.
- Conditions
- Complete DiGeorge Anomaly, DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- NCT01220531
- Testing
- Cultured Thymus Tissue
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Hereditary Hemorrhagic Telangiectasia
Prospective Descriptive Study of the Angiogenic T Cell Population in Subjects With Hereditary Hemorrhagic Telangiectasia (HHT)
Hereditary hemorrhagic telangiectasia (HHT) results from genetic deregulation of angiogenesis. It is characterized by mucocutaneous telangiectasia responsible for recurrent epistaxis affecting quality of life (anaemia, iron…
- Trial name
- TangRO (NCT03572556)
- Testing
- Blood samples, Epistaxis charts
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire Dijon
DiGeorge Syndrome, Congenital Heart Defects
Infection in DiGeorge Following CHD Surgery
We propose a retrospective review of patients with DiGeorge syndrome having undergone cardiac surgery to evaluate the incidence of blood stream and/or surgical site infection.
- Trial name
- NCT00278005
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- Children's Healthcare of Atlanta
MPN (Myeloproliferative Neoplasms)
A Study of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis
The purpose of this study is to evaluate the safety, tolerability, and efficacy of the combination of parsaclisib and ruxolitinib in subjects with myelofibrosis.
- Trial name
- NCT02718300
- Testing
- Parsaclisib, Ruxolitinib
- Where
- 39 sites in United States
- Volunteers
- 74 people
- Sponsor
- Incyte Corporation
22q11 Deletion Syndrome
NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
This is a Phase 2, randomized, placebo-controlled crossover trial to assess the safety and efficacy of NB-001 in children and adolescents with 22q11DS that manifest commonly associated neuropsychiatric symptoms.
- Trial name
- NCT05290493
- Testing
- NB-001
- Where
- 4 sites in United States, Canada
- Volunteers
- 37 people
- Sponsor
- Nobias Therapeutics, Inc.
Hereditary Haemorrhagic Telangiectasia
Hereditary Haemorrhagic Telangiectasia Flight Safety Study
Hereditary Haemorrhagic Telangiectasia (HHT) is a condition in which sufferers have abnormal blood vessels which makes them more likely to bleed than other people, particularly in the lungs, which results in low blood oxygen…
- Trial name
- Flying and HHT (NCT01590121)
- Testing
- Aeroplane flight in the past- no active intervention for study, Questionnaire
- Where
- United Kingdom
- Volunteers
- 156 people
- Sponsor
- Imperial College London
Telangiectasia, Hereditary Hemorrhagic
Studies of Hereditary Hemorrhagic Telangiectasia
OBJECTIVES: I. Examine screening modalities for pulmonary vascular arteriovenous malformation (PAVM) in individuals with endoglin mutations. II.
- Trial name
- NCT00004648
- Volunteers
- 65 people
- Sponsor
- National Center for Research Resources (NCRR)
Hereditary Hemorrhagic Telangiectasia
Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept
Hereditary hemorrhagic telangiectasia (HHT) is a hereditary vascular condition characterized by the development of abnormal connections between arteries and veins throughout the body, called vascular malformations.
- Trial name
- NCT01752049
- Testing
- Topical timolol maleate
- Where
- Canada
- Volunteers
- 5 people
- Sponsor
- Unity Health Toronto
Telangiectasia, Hereditary Hemorrhagic
Study of Endothelial Cells in Patients With Hereditary Haemorrhagic Telangiectasia
Hereditary Haemorrhagic Telangiectasia (HHT, also known as Osler-Weber-Rendu Syndrome) is an disease that leads to the development of dilated and fragile blood vessels.
- Trial name
- NCT00733629
- Where
- United Kingdom
- Sponsor
- Imperial College London
Hemangiomas, Vascular Malformations
Diagnosis of Hemangiomas and Vascular Malformations
To develop a non-invasive, simple and affordable, measurement technique to differentiate hemangiomas from subtypes of VMS. The purpose of this study is to determine the best office based diagnositc instrument for differentiating…
- Conditions
- Hemangiomas, Vascular Malformations, Venular Malformations, Venous Malformations
- Trial name
- NCT00577213
- Volunteers
- 56 people
- Sponsor
- University of Arkansas
Neurodevelopmental Disorders, Autism
Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders
There are currently no approved medications for the treatment of anxiety in children and youth with neurodevelopmental disorders (NDDs), both common and rare.
- Conditions
- Neurodevelopmental Disorders, Autism, Autism Spectrum Disorder, Fragile X Syndrome
- Trial name
- CALM (NCT06081348)
- Testing
- Sertraline
- Where
- 7 sites in Canada
- Volunteers
- 130 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Telangiectasia, Hereditary Hemorrhagic, Rendu Osler Disease
Diagnostic Value of Ultra-low Dose Thoracic Scanner for the Pulmonary Arteriovenous Malformation Detection in HHT Patient
Hereditary hemorrhagic telangiectasia (HHT) is linked to a dysregulation of angiogenesis leading to the formation of arteriovenous malformations (AVM): cutaneo-mucous telangiectasia and visceral shunts.
- Trial name
- ULD OSLER (NCT04108052)
- Testing
- Low CT scanner without injection dose, Ultra-low dose CT scanner without injection
- Where
- France
- Volunteers
- 45 people
- Sponsor
- Hospices Civils de Lyon
Non-Hodgkin's Lymphoma
Study of Chiauranib in Relapsed/Refractory Non-Hodgkin's Lymphoma
Chiauranib may stop the growth of tumor cells by blocking Aurora kinase B（Aurora B）、VEGFR/PDGFR/c-Kit、CSF-1R targets. This clinical trial is studying the efficacy and safety of chiauranib works in treating patients with relapsed…
- Trial name
- NCT03074825
- Testing
- Chiauranib
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Chipscreen Biosciences, Ltd.
Lymphoma
A Study of INCB050465 in Relapsed or Refractory Follicular Lymphoma
The purpose of this study is to assess the objective response rate of parsaclisib treatment in participants with relapsed or refractory follicular lymphoma.
- Trial name
- CITADEL-203 (NCT03126019)
- Testing
- Parsaclisib
- Where
- 83 sites in United States, Australia and 11 more
- Volunteers
- 126 people
- Sponsor
- Incyte Corporation
Hereditary Hemorrhagic Telangiectasia
Body Donation in HHT
The possibility of body donation in hereditary hemorrhagic telangiectasia (HHT) means that human tissue samples with and without vascular malformations can be analyzed. The aim is to gain insights into the disease mechanism.
- Trial name
- NCT07826845
- Testing
- body donation in HHT
- Where
- Germany
- Volunteers
- 5 people
- Sponsor
- University Hospital, Essen
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
22q11 Deletion Syndrome, DiGeorge Syndrome
SNP-based Microdeletion and Aneuploidy RegisTry (SMART)
This multi-center prospective observational study is designed to track birth outcomes and perinatal correlates to the Panorama prenatal screening test in the general population among ten thousand women who present clinically and…
- Conditions
- 22q11 Deletion Syndrome, DiGeorge Syndrome, Trisomy 21, Trisomy 18
- Trial name
- SMART (NCT02381457)
- Where
- 21 sites in United States, Australia and 4 more
- Volunteers
- 20,960 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Venous Malformation, Lymphatic Malformation
First in Human Trial of Topical VT30 in Pts With Venous/Lymphatic Malformations Assoc With PIK3CA or TEK Gene Mutations
VT30-101 is a 2-part first-in-human trial of topically administered VT30 to subjects with cutaneous venous malformations, lymphatic malformations, or mixed venolymphatic malformations associated with PIK3CA or TEK mutations.
- Conditions
- Venous Malformation, Lymphatic Malformation, Venolymphatic Malformation
- Trial name
- NCT04409145
- Testing
- VT30
- Where
- 15 sites in United States
- Volunteers
- 15 people
- Sponsor
- Venthera, Inc., a BridgeBio company