Clinical trials: Lymphatic Malformations
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
Acute Leukemia, Acute Myeloid Leukemia
A Study of Withdrawal of Immunosuppression and Donor Lymphocyte Infusions Following Allogeneic Transplant for Pediatric Hematologic Malignancies
There is no curative therapy once acute leukemia patients relapse after transplant. Patients who develop clinically significant graft versus host disease (GVHD) have a lower rate of relapse than those who do not develop GVHD.
- Conditions
- Acute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Biphenotypic Leukemia
- Trial name
- NCT01036009
- Testing
- Withdrawal of immunosuppression and donor lymphocyte infusion
- Where
- 2 sites in United States
- Volunteers
- 43 people
- Sponsor
- University of California, San Francisco
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
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
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Feasibility of [11C]Acetate-PET in LAM and TSC
This study aims to assess [11C]acetate positron emission tomography (PET)/computed tomography (CT) as a biomarker for renal angiomyolipomas and pulmonary lymphangioleiomyomatosis (LAM) and an early biomarker of response to…
- Trial name
- NCT05467397
- Testing
- [11C]acetate
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Brigham and Women's Hospital
Hereditary Hemorrhagic Telangiectasia (HHT), Epistaxis
Prospective Pilot Study of Floseal for the Treatment of Anterior Epistaxis in Patients With (HHT)
Current management strategies for severe and recurrent epistaxis secondary to Hereditary hemorrhagic telangiectasia (HHT) include surgical procedures, and uncomfortable nasal packing, both of which are often only short-term…
- Trial name
- NCT02638012
- Testing
- Floseal, Packing
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- Unity Health Toronto
Hereditary Hemorrhagic Telangiectasia
Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia
This study is a double-blinded, randomized controlled trial to evaluate the efficacy of an intranasal topical timolol gel in the care for epistaxis in adults with hereditary hemorrhagic telangiectasia.
- Trial name
- ETIC-HHT (NCT04139018)
- Testing
- Timolol Gel
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Washington University School of Medicine
Telangiectasia, Hereditary Hemorrhagic
Evaluation of Pazopanib on Bleeding in Subjects With Hereditary Haemorrhagic Telangiectasia
This study will investigate whether pazopanib can reduce epistaxis and improve anaemia in subjects with hereditary haemorrhagic telangiectasia (HHT) at a dose that is well tolerated. The study will have 2 parts.
- Trial name
- NCT02204371
- Testing
- Pazopanib
- Where
- 6 sites in United States, Canada
- Volunteers
- 7 people
- Sponsor
- GlaxoSmithKline
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
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
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
DiGeorge Syndrome, Complete Typical DiGeorge Anomaly
Thymus Transplantation in DiGeorge Syndrome #668
The study purpose is to determine whether cultured thymus tissue implantation (CTTI) is effective in treating typical complete DiGeorge syndrome.
- Trial name
- NCT00576407
- Testing
- Cultured Thymus Tissue for Implantation (CTTI)
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
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
Kaposiform Hemangioendotheliomas, Tufted Angioma
Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
The purpose of this study is to determine if the use of sirolimus in the treatment of children and young adults with complicated vascular anomalies will prove to be safe and provide objective response resulting in improved…
- Conditions
- Kaposiform Hemangioendotheliomas, Tufted Angioma, Capillary Venous Lymphatic Malformation, Venous Lymphatic Malformation
- Trial name
- NCT00975819
- Testing
- sirolimus
- Where
- 2 sites in United States
- Volunteers
- 61 people
- Sponsor
- Denise Martin Adams
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
Kaposiform Hemangioendothelioma (KHE), Kasabach-Merritt Syndrome
A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors
In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk vascular tumors such as Tufted Angioma…
- Conditions
- Kaposiform Hemangioendothelioma (KHE), Kasabach-Merritt Syndrome, Tufted Angioma
- Trial name
- NCT02110069
- Testing
- Vincristine, Sirolimus
- Where
- 7 sites in United States
- Volunteers
- 4 people
- Sponsor
- Boston Children's Hospital
Renal Angiomyolipoma
Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.
The purpose of this study was to assess the safety and efficacy of everolimus (Afinitor®) in Chinese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC).
- Trial name
- NCT03525834
- Testing
- Everolimus
- Where
- 5 sites in China
- Volunteers
- 40 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
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)
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