Clinical trials: Lymphatic Malformation
Haemophilia A
A Study to Compare the Blood Levels of Mim8 in Healthy Men When Administered With the DV3407-C1 Pen Injector or a Syringe and Cartridge
Novo Nordisk is developing the study medicine Mim8 for the treatment of haemophilia A. The study aims to show similar levels of Mim8 in blood when using a new pen injector, called DV3407-C1 pen injector, and when using a syringe…
- Trial name
- NCT05681845
- Testing
- NNC0365-3769 (Mim8)
- Where
- Germany
- Volunteers
- 205 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Anemia, Sickle Cell
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor…
- Trial name
- NCT04091737
- Testing
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- CSL Behring
Hemophilia B
BeneFIX Drug Use Results Survey [All-Case Surveillance]
The survey is intended to investigate the following matters, etc. under the actual use status after marketing in all patients who are administered this drug for a certain period of time after the launch. 1.
- Trial name
- NCT01154231
- Testing
- Nonacog Alfa (Genetical Recombination)
- Where
- Japan
- Volunteers
- 314 people
- Sponsor
- Pfizer
Cancer, Pancreatic Cancer
9-ING-41 in Patients With Advanced Cancers
GSK-3β is a potentially important therapeutic target in human malignancies. The Actuate 1801 Phase 1/2 study is designed to evaluate the safety and efficacy of 9-ING-41, a potent GSK-3β inhibitor, as a single agent and in…
- Conditions
- Cancer, Pancreatic Cancer, Sarcoma, Renal Cancer
- Trial name
- NCT03678883
- Testing
- 9-ING-41, Gemcitabine - 21 day cycle, Doxorubicin.
- Where
- 66 sites in United States, Belgium and 5 more
- Volunteers
- 350 people
- Sponsor
- Actuate Therapeutics Inc.
Sickle Cell Disease, Sickle Cell Anemia
A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy
This trial is a follow-up companion study to Protocol ICA-17043-10, a Phase III, multi-center, efficacy and safety study of ICA-17043.
- Trial name
- NCT00294541
- Testing
- ICA-17043
- Where
- 31 sites in United States
- Volunteers
- 51 people
- Sponsor
- Icagen
Hemophilia B
BAX 326 Pediatric Study
The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.
- Trial name
- NCT01488994
- Testing
- BAX326
- Where
- 11 sites in India, Poland and 4 more
- Volunteers
- 23 people
- Sponsor
- Baxalta now part of Shire
Tumor or Lymphoma
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of JNJ-42756493 in Adult Participants With Advanced or Refractory Solid Tumors or Lymphoma
The purpose of this study is to evaluate the safety, pharmacokinetics (study of what the body does to a drug), and pharmacodynamics (study of what a drug does to the body) of JNJ-42756493, a pan-fibroblast growth factor receptor…
- Trial name
- NCT01703481
- Testing
- JNJ-42756493: Part 1, JNJ-42756493: Part 2, JNJ-42756493: Part 3
- Where
- 37 sites in United States, France and 1 more
- Volunteers
- 188 people
- Sponsor
- Janssen Research & Development, LLC
Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Congenital Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with congenital hemophilia A.
- Trial name
- NCT01434511
- Testing
- OBI-1
- Where
- 3 sites in United States, South Africa and 1 more
- Volunteers
- 1 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency
The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors （Part A）and to evaluate the…
- Conditions
- Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT07644832
- Testing
- SR604
- Where
- 9 sites in China
- Volunteers
- 76 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Severe Haemophilia A
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
The rationale of this study is to further fine-tune and individualize prophylactic treatment of patients with severe Haemophilia A with the goal of keeping the trough FVIII level above 1% between doses.
- Trial name
- NCT02256917
- Testing
- Human cl rhFVIII
- Where
- 30 sites in United States, Canada and 7 more
- Volunteers
- 58 people
- Sponsor
- Octapharma
Hemophilia A, Hemophilia A and B
A Phase III Clinical Trial Evaluating SR604 Injection for Prophylactic Treatment in Patients With Hemophilia
This is a single-arm, multicenter Phase III clinical trial designed to evaluate the efficacy and safety of prophylactic treatment with SR604 Injection in hemophilia A/B patients with or without inhibitors.
- Conditions
- Hemophilia A, Hemophilia A and B, Hemophilia B
- Trial name
- NCT07876674
- Testing
- SR604
- Volunteers
- 90 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Hemophilia A
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A.
- Trial name
- NCT04418414
- Testing
- Gene therapy, Biological
- Volunteers
- 7 people
- Sponsor
- Expression Therapeutics, LLC
Sphingomyelin Lipidosis
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa.
- Trial name
- NCT02004704
- Testing
- GZ402665
- Where
- 9 sites in United States, Belgium and 5 more
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
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
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A
This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal…
- Trial name
- guardian™ 5 (NCT02035384)
- Testing
- turoctocog alfa
- Where
- 41 sites in United States, Austria and 13 more
- Volunteers
- 69 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H803 injection in severe Hemophilia A subjects.
- Trial name
- NCT06111638
- Testing
- Single dose intravenous injection of BBM-H803
- Where
- 10 sites in China
- Volunteers
- 55 people
- Sponsor
- Shanghai Xinzhi BioMed Co., Ltd.
Sickle Cell Disease
A Study of IMR-687 in Subjects With Sickle Cell Disease
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
- Trial name
- NCT04474314
- Testing
- IMR-687
- Where
- 49 sites in United States, Ghana and 11 more
- Volunteers
- 115 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Von Willebrand Disease (VWD)
A Phase II Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of SR604 Injection in Patients With Von Willebrand Disease
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in patients with von Willebrand disease.
- Trial name
- NCT07640893
- Testing
- SR604
- Where
- 9 sites in China
- Volunteers
- 24 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Severe Hemophilia A
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
This Phase 1/2 study will be a dose escalation study in adults in 5 cohorts (named cohorts 1, 2, 3, 5 and 6), with the main purpose to assess the safety of subcutaneous injection of OCTA101 (a human-cl rhFVIII and recombinant…
- Trial name
- NCT04046848
- Testing
- OCTA101
- Where
- Bulgaria
- Volunteers
- 36 people
- Sponsor
- Octapharma
PTCH1 or SMO Activated Solid and Hematologic Tumors
LDE225 for Patients With PTCH1 or SMO Mutated Tumors
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
- Trial name
- SIGNATURE (NCT02002689)
- Testing
- LDE225
- Where
- 10 sites in United States
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals