Clinical trials: Lymphatic Malformations
22Q11 Deletion Syndrome
Mindfulness Program for Adolescents With 22q11DS
The goal of this study is to evaluate the effectiveness of the Aware Program, an online mindfulness education program, with adolescents with 22q11DS and their parents.
- Trial name
- NCT05849441
- Testing
- Aware Program
- Where
- United States
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Innovation Research & Training
Hereditary Hemorrhagic Telangiectasia, Nosebleeds
Sirolimus for Nosebleeds in HHT
This pilot study is to determine the safety and efficacy of oral sirolimus (blood trough level 6-10ng/ml) in patients with HHT that are experiencing moderate or severe epistaxis.
- Conditions
- Hereditary Hemorrhagic Telangiectasia, Nosebleeds, Epistaxis
- Trial name
- NCT05269849
- Testing
- Sirolimus
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Unity Health Toronto
DiGeorge Anomaly, DiGeorge Syndrome
Thymus Transplantation Dose in DiGeorge #932
One purpose of this study is to determine whether the amount of cultured thymus tissue implanted into DiGeorge anomaly infants has any effect on the immune outcome.
- Conditions
- DiGeorge Anomaly, DiGeorge Syndrome, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- NCT00576836
- Testing
- Cultured Thymus Tissue Implantation (CTTI), Cultured Thymus Tissue Implantation with Parathyroid Transplantation
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
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
Follicular Lymphoma
Study of Duvelisib in Combination With Rituximab vs Rituximab in Subjects With Previously Treated Follicular Lymphoma
A study to evaluate the safety and efficacy of duvelisib administered in combination with rituximab vs placebo in combination with rituximab in patients with previously treated CD20-positive follicular lymphoma who are not…
- Trial name
- DYNAMO + R (NCT02204982)
- Testing
- Duvelisib, Rituximab
- Where
- 5 sites in Australia, France and 2 more
- Volunteers
- 13 people
- Sponsor
- SecuraBio
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.
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
- Trial name
- EXIST-2 (NCT00790400)
- Testing
- Everolimus (RAD001)
- Where
- 25 sites in United States, Canada and 9 more
- Volunteers
- 118 people
- Sponsor
- Novartis Pharmaceuticals
Lymphatic Malformations
OK432 (Picibanil) in the Treatment of Lymphatic Malformations
Standard of care for Lymphatic Malformations has been surgical excision. We have been using OK432/Picibanil (generously supplied by Chugai Pharmaceuticals in Japan) since 1992 with great success for macrocystic disease.
- Trial name
- NCT03427619
- Testing
- OK432
- Where
- 14 sites in United States
- Volunteers
- 275 people, healthy volunteers welcome
- Sponsor
- Richard JH Smith
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
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
Velo-cardio-facial Syndrome, Psychosis
Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with…
- Trial name
- NCT01127503
- Testing
- Metyrosine
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Bausch Health Americas, Inc.
Lymphangioma
A Study to Evaluate Sildenafil for the Treatment of Lymphatic Malformations
There is an unsatisfied medical need for a first-line treatment of lymphatic malformations with a good benefit/risk profile. Based on a patient experience in the institution, the investigators plan to verify whether or not the…
- Trial name
- NCT01290484
- Testing
- Sildenafil
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Stanford University
Telangiectasia, Hereditary Hemorrhagic, Epistaxis
North American Study of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of the NOSE Study is to carefully examine the efficacy and safety of 3 nasal sprays (bevacizumab, estriol, and tranexamic acid), compared to placebo, for the treatment of HHT related nosebleeds.
- Trial name
- NOSE (NCT01408030)
- Testing
- Sterile saline, Bevacizumab, Estriol
- Where
- 6 sites in United States
- Volunteers
- 123 people
- Sponsor
- James Gossage
Epistaxis, Hereditary Hemorrhagic Telangiectasia
Office-sclerotherapy for Epistaxis Due to Hereditary Hemorrhagic Telangiectasia
The purpose of this study is to test a novel and tolerable office-based treatment method, sclerotherapy with sodium tetradecyl sulfate, for recurrent epistaxis (nosebleeds) related to Hereditary Hemorrhagic Telangiectasia (HHT)…
- Trial name
- NCT01408732
- Testing
- Sclerotherapy, Standard Treatment
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- University of Minnesota
Hereditary Hemorrhagic Telangiectasia (HHT)
Topical Bevacizumab for the Management of Recurrent Epistaxis in Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
Terence M. Davidson, MD is conducting a research study to find out more about the topical application of Avastin (proper chemical name is bevacizumab) in the treatment of epistaxis (nose bleeding) in patients with Hereditary…
- Trial name
- NCT01397695
- Testing
- Bevacizumab
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, San Diego
Epistaxis
NOrth American Study for the Treatment of Recurrent epIstaxis With DoxycycLine: The NOSTRIL Trial
The purpose of this research study is to determine whether Doxycycline can be used to control nosebleeds (epistaxis) for patients with Hereditary Hemorrhagic Telangiectasia (HHT).
- Trial name
- NOSTRIL (NCT04167085)
- Testing
- Doxycycline
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- University of California, Los Angeles
DiGeorge Anomaly, Complete DiGeorge Anomaly
Phase I/II Thymus Transplantation With Immunosuppression #950
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and…
- Conditions
- DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- #950 (NCT00579527)
- Testing
- Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
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
Hypoparathyroidism, DiGeorge Syndrome
Effects of PTH Replacement on Bone in Hypoparathyroidism
Hypoparathyroidism is a rare condition associated with a low level of parathyroid hormone (PTH) in the blood. Hypoparathyroidism can be genetic and show up in childhood, or it can occur later in life.
- Trial name
- NCT00395538
- Testing
- PTH 1-34
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Hereditary Haemorrhagic Telangiectasia (HHT)
Diet and Hereditary Haemorrhagic Telangiectasia
Hereditary Haemorrhagic Telangiectasia (HHT) affects 1 in 5,000 people. The purpose of this study is to provide data regarding the diet and nosebleed frequency using a questionnaire. This will be filled in by people with HHT.
- Trial name
- NCT01692015
- Testing
- Questionnaire on dietary history, Questionnaire on nosebleed severity, One week food diary generated by weighing foods
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- Imperial College London