Clinical trials: Hematologic Disorder
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
Antithrombin III Deficiency
Safety, Pharmacokinetics and Efficacy of an ATIII Concentrate
To assess the safety, pharmacokinetics and efficacy of a plasma-derived AT-III concentrate in the treatment of subjects with congenital AT-III deficiency.
- Trial name
- NCT00319228
- Testing
- Plasma-derived AT-III concentrate
- Volunteers
- 3 people
- Sponsor
- Instituto Grifols, S.A.
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC
Elranatamab, Myeloma
A Study of Elranatamab (PF-06863135) in Chinese Participants With Refractory Multiple Myeloma.
The purpose of this study is to understand the study medicine (called Elranatamab, or PF-06863135) as potential treatment for refractory multiple myeloma.
- Conditions
- Elranatamab, Myeloma, Multiple Myeloma, Relapsed Multiple Myeloma
- Trial name
- NCT05228470
- Testing
- Elranatamab
- Where
- 15 sites in China
- Volunteers
- 39 people
- Sponsor
- Pfizer
Myelofibrosis, Leukemia, Myelocytic, Acute
A Phase 1/2 Study of CPI-0610 With and Without Ruxolitinib in Patients With Hematologic and Myeloproliferative Malignancies
Phase 1 Part: This was an open-label, sequential dose escalation study of pelabresib (CPI-0610) in patients who had previously been treated for Acute Leukemia, Myelodysplastic/Myeloproliferative Neoplasms.
- Conditions
- Myelofibrosis, Leukemia, Myelocytic, Acute, Myelodysplastic/Myeloproliferative Neoplasm, Myelodysplastic Syndrome (MDS)
- Trial name
- NCT02158858
- Testing
- Pelabresib, Ruxolitinib
- Where
- 48 sites in United States, Belgium and 7 more
- Volunteers
- 336 people
- Sponsor
- Constellation Pharmaceuticals
Myeloid Leukemia, Chronic, Chronic Phase
Ponatinib in Participants With Resistant Chronic Phase Chronic Myeloid Leukemia (CP-CML) to Characterize the Efficacy and Safety of a Range of Doses
The purpose of this study is to characterize the efficacy of ponatinib administered in 3 starting doses (45 mg, 30 mg, and 15 mg daily) in participants with CP-CML who are resistant to prior tyrosine-kinase inhibitor (TKI)…
- Trial name
- OPTIC (NCT02467270)
- Testing
- Ponatinib
- Where
- 86 sites in United States, Argentina and 19 more
- Volunteers
- 283 people
- Sponsor
- Takeda
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
Hemophilia A, Gene Therapy
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe…
- Conditions
- Hemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder
- Trial name
- GENEr8-AAV5+ (NCT03520712)
- Testing
- Valoctocogene Roxaparvovec
- Where
- 9 sites in South Africa, South Korea and 2 more
- Volunteers
- 3 people
- Sponsor
- BioMarin Pharmaceutical
Severe Haemophilia A
Gene Therapy Study in Severe Haemophilia A Patients (270-201)
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, dose escalation study in order to determine the safety and efficacy of valoctocogene roxaparvovec (an Adenovirus-Associated Virus based gene therapy…
- Trial name
- NCT02576795
- Testing
- valoctocogene roxaparvovec
- Where
- 5 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy evaluation period (EEP)…
- Trial name
- BMN 270-301 (NCT03370913)
- Testing
- valoctocogene roxaparvovec
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 144 people
- Sponsor
- BioMarin Pharmaceutical
Multiple Myeloma
ACY-1215 (Ricolinostat) in Combination With Pomalidomide and Low-dose Dex in Relapsed-and-Refractory Multiple Myeloma
Phase 1b: To evaluate the side effects and determine the best dose of ACY-1215 in combination with Pomalidomide and low-dose dexamethasone in patients with relapsed-and-refractory multiple myeloma.
- Trial name
- NCT01997840
- Testing
- ACY-1215 (Ricolinostat) in combination with pomalidomide and dexamethasone
- Where
- 2 sites in United States
- Volunteers
- 103 people
- Sponsor
- Celgene
Hemophilia A
A Gene Transfer Study for Hemophilia A
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
- Trial name
- NCT03003533
- Testing
- SPK-8011
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Hematologic Malignancies, Solid Tumor
A Trial of PF-07901800 (TTI-621) for Patients With Hematologic Malignancies and Selected Solid Tumors
Multicenter, open-label, phase 1a/1b trial of PF-07901800 (TTI-621) in subjects with relapsed or refractory hematologic malignancies and selected solid tumors.
- Trial name
- NCT02663518
- Testing
- PF-0791800 (TTI-621), PF-07901800 (TTI-621) plus Rituximab, PF-07901800 (TTI-621) plus Nivolumab
- Where
- 51 sites in United States, Canada
- Volunteers
- 249 people
- Sponsor
- Pfizer
Multiple Myeloma
A Study of KW-2478 in Combination With Bortezomib in Subjects With Relapsed and/or Refractory Multiple Myeloma
The purpose of this study is to assess the safety and benefits of the investigational study drug, KW-2478, when given with bortezomib (Velcade®), a drug approved for the treatment of Multiple Myeloma (MM).
- Trial name
- NCT01063907
- Testing
- KW-2478, Bortezomib
- Where
- 26 sites in United States, Philippines and 1 more
- Volunteers
- 95 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis
A Study of Momelotinib Versus Danazol in Symptomatic and Anemic Myelofibrosis Participants (MOMENTUM)
MOMENTUM is a randomized, double-blind, active control Phase 3 trial intended to confirm the differentiated clinical benefits of the investigational drug momelotinib (MMB) versus danazol (DAN) in symptomatic and anemic…
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Trial name
- NCT04173494
- Testing
- Momelotinib, Danazol
- Where
- 167 sites in United States, Australia and 20 more
- Volunteers
- 195 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Idiopathic Thrombocytopenic Purpura, Thrombocytopenia
An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP
This protocol will provide open label romiplostim to adult thrombocytopenic subjects. Romiplostim will be administered by subcutaneous injection once per week.
- Conditions
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenic Purpura
- Trial name
- NCT00508820
- Testing
- Romiplostim
- Volunteers
- 407 people
- Sponsor
- Amgen
Idiopathic Thrombocytopenic Purpura, Thrombocytopenia
Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura
This is a phase 3b, multi-center, randomized, Standard of Care (SOC)-controlled, open-label, 52-week treatment study to compare romiplostim to medical SOC for Idiopathic Thrombocytopenia Purpura (ITP), with a 6-month Safety…
- Conditions
- Idiopathic Thrombocytopenic Purpura, Thrombocytopenia, Thrombocytopenia in Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP), Thrombocytopenic Purpura
- Trial name
- NCT00415532
- Testing
- Medical Standard of Care for ITP, Romiplostim
- Volunteers
- 234 people
- Sponsor
- Amgen
Cancer, Hematologic Malignancies
Denosumab Compared to Zoledronic Acid in the Treatment of Bone Disease in Patients With Multiple Myeloma
The purpose of this study is to determine if denosumab is non-inferior to zoledronic acid in the treatment of bone disease from multiple myeloma.
- Conditions
- Cancer, Hematologic Malignancies, Multiple Myeloma, Oncology
- Trial name
- NCT01345019
- Testing
- Denosumab, Zoledronic acid, Denosumab (for the open-label treatment phase)
- Where
- 309 sites in United States, Australia and 27 more
- Volunteers
- 1,718 people
- Sponsor
- Amgen