Clinical trials: Lymphatic Malformation
Chronic Iron Overload
Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation
Extend evaluation of deferasirox film-coated tablet (FCT) formulation
- Trial name
- NCT02720536
- Testing
- Deferasirox
- Where
- 14 sites in Austria, Greece and 1 more
- Volunteers
- 53 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Gerofit Exercise Intervention for Older Adults With Sickle Cell Disease (SICKLE-FIT Study)
The purpose of this study to assess the feasibility, acceptability, and safety of a personalized exercise training program adapted from Gerofit to improve physical health and quality of life for adults with SCD
- Trial name
- SICKLE-FIT (NCT05714098)
- Testing
- Gerofit Exercise Program
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Duke University
Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age)…
- Conditions
- Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis
- Trial name
- NCT04935879
- Testing
- Inclacumab
- Where
- 58 sites in United States, Brazil and 10 more
- Volunteers
- 241 people
- Sponsor
- Pfizer
Sickle Cell Disease
Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
- Trial name
- NCT01090323
- Testing
- ICL670
- Where
- 39 sites in United States, Canada and 3 more
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Hemophilia A
Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia.
- Trial name
- NCT00950170
- Testing
- Laboratory Tests
- Where
- 11 sites in France, Germany and 5 more
- Volunteers
- 23 people
- Sponsor
- Pfizer
Sickle Cell Anemia
Carbon Monoxide Measurement to Screen for Sickle Cell Disease
Modify the design of the CoSense device (Model C20112, currently cleared by the FDA for ETCO (end-tidal carbon monoxide) monitoring to improve accuracy and consistency under temperature conditions encountered in countries with…
- Trial name
- SCDCO (NCT02530242)
- Testing
- End-tidal Carbon Monoxide Subjects, End-tidal Carbon Monoxide Controls
- Volunteers
- 32 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Hemophilia B
Pivotal Study (Pharmacokinetics, Efficacy, Safety) of BAX 326 (rFIX) in Hemophilia B Patients
The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK) parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in previously treated patients (PTPs) with severe and…
- Trial name
- NCT01174446
- Testing
- BAX 326, BeneFIX
- Where
- 25 sites in Argentina, Brazil and 12 more
- Volunteers
- 86 people
- Sponsor
- Baxalta now part of Shire
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
Hemophilia A
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously…
- Trial name
- NCT02962765
- Where
- 39 sites in United States, Argentina and 11 more
- Volunteers
- 80 people
- Sponsor
- Octapharma
Leukemia
S0919 Idarubicin, Cytarabine, and Pravastatin in Treating Patients With Relapsed Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as idarubicin and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00840177
- Testing
- cytarabine, idarubicin, pravastatin sodium
- Where
- 185 sites in United States
- Volunteers
- 115 people
- Sponsor
- SWOG Cancer Research Network
Hemophilia A or B With Inhibitors, Hemophilia A
Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor
The purpose of the study was to determine the efficacy, safety, and health-related quality of life benefits with FEIBA NF prophylactic treatment as compared with on-demand treatment.
- Conditions
- Hemophilia A or B With Inhibitors, Hemophilia A, Hemophilia B
- Trial name
- NCT00851721
- Testing
- Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
- Where
- 17 sites in United States, Brazil and 8 more
- Volunteers
- 52 people
- Sponsor
- Baxalta now part of Shire
Reversal of Anticoagulant Treatment
Study of Octaplex (Human Prothrombin Complex Concentrate) and Fresh Frozen Plasma in Patients Under Vitamin K Therapy Antagonist Needing Urgent Surgery or Invasive Procedures
The purpose of this study is to determine whether Octaplex (human prothrombin complex concentrate) can reverse the effects of anticoagulants when compared to the standard treatment of fresh frozen plasma (FFP).
- Trial name
- NCT00618098
- Testing
- Octaplex (human prothrombin complex concentrate), Fresh frozen plasma
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Octapharma
Factor XIII Deficiency
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00945906
- Testing
- FXIII Concentrate (Human) (FXIII)
- Where
- 25 sites in United States
- Volunteers
- 61 people
- Sponsor
- CSL Behring
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
Congenital Fibrinogen Deficiency, Afibrinogenemia
Pharmacokinetic, Efficacy, and Safety Study of Octafibrin Compared to Haemocomplettan/Riastap
The purpose of this study is to investigate pharmacokinetic properties, surrogate efficacy and safety of Octafibrin compared to Haemocomplettan® P/RiaSTAPTM in patients with congenital fibrinogen deficiency
- Trial name
- NCT01575756
- Testing
- Octafibrin, Haemocomplettan® P or RiaSTAPTM
- Where
- 10 sites in United States, Bulgaria and 4 more
- Volunteers
- 22 people
- Sponsor
- Octapharma
Aplastic Anemia, Pure Red Cell Aplasia
A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure
Participants in this study are suffering from rare and serious blood disorders. In aplastic anemia, the bone marrow stops producing red blood cells, platelets, and white blood cells.
- Conditions
- Aplastic Anemia, Pure Red Cell Aplasia, Diamond Blackfan Anemia
- Trial name
- NCT00001962
- Testing
- Daclizumab
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fanconi Anemia, Squamous Cell Carcinoma
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous…
- Trial name
- NCT03476330
- Testing
- Quercetin (dietary supplement)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD
Related donor stem cell transplantation using the alemtuzumab/ TBI platform has been shown to be a safe strategy to cure severe sickle cell disease.
- Trial name
- SCD-Haplo (NCT02013375)
- Testing
- Haploidentical Transplant, Alemtuzumab, Total Body Irradiation
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Damiano Rondelli, MD
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
Hemophilia A
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
- Trial name
- GENEr8-3 (NCT04323098)
- Testing
- valoctocogene roxaparvovec
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 22 people
- Sponsor
- BioMarin Pharmaceutical