Clinical trials: Bleeding Diathesis
Factor XIII Deficiency
A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) 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
- NCT00885742
- Testing
- FXIII Concentrate (Human)
- Where
- 23 sites in United States, Spain
- Volunteers
- 41 people
- Sponsor
- CSL Behring
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
Primary Objective: To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to…
- Trial name
- NCT00768287
- Testing
- IB1001, nonacog alfa
- Where
- 23 sites in United States, France and 5 more
- Volunteers
- 77 people
- Sponsor
- Medexus Pharma, Inc.
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
Hemophilia A, Hemophilia B
Study of Ataluren (PTC124) in Hemophilia A and B
Hemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blood clotting.
- Trial name
- NCT00947193
- Testing
- Ataluren
- Where
- 13 sites in United States, Canada and 2 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics
Hemophilia B
A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.
- Trial name
- NCT01496274
- Testing
- rIX-FP
- Where
- 30 sites in United States, Austria and 8 more
- Volunteers
- 63 people
- Sponsor
- CSL Behring
Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Myelomonocytic Leukemia
Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders
This phase II trial studies the side effects and best dose of total-body irradiation when given together with fludarabine phosphate followed by a donor peripheral stem cell transplant in treating patients with myelodysplastic…
- Conditions
- Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndrome, Essential Thrombocythemia
- Trial name
- NCT00397813
- Testing
- Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- 3 sites in United States
- Volunteers
- 77 people
- Sponsor
- Fred Hutchinson Cancer Center
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of…
- Trial name
- paradigm™ 2 (NCT01333111)
- Testing
- nonacog beta pegol
- Where
- 43 sites in United States, Canada and 13 more
- Volunteers
- 74 people
- Sponsor
- Novo Nordisk A/S
Primary Thrombocytopenia
Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
NewGam is a newly developed human normal immunoglobulin solution for intravenous administration (IGIV). This study will evaluate the safety and efficacy of NewGam 10% in patients with primary immune thrombocytopenia.
- Trial name
- NCT01349790
- Testing
- NewGam
- Where
- Germany
- Volunteers
- 40 people
- Sponsor
- Octapharma
Hemophilia B
Dose Confirmation Trial of AAV5-hFIXco-Padua
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase.
- Trial name
- NCT03489291
- Testing
- AAV5-hFIXco-Padua (AMT-061)
- Where
- 4 sites in United States
- Volunteers
- 3 people
- Sponsor
- CSL Behring
Immune Thrombocytopenia (ITP)
A Phase 2a Study Evaluating BIVV020 in Adults With Persistent/Chronic Immune Thrombocytopenia (ITP)
Primary Objective: To evaluate the effect of BIVV020 on the durability of platelet response in participants with persistent/chronic immune thrombocytopenia (ITP) Secondary Objectives: To assess the safety and tolerability of…
- Trial name
- NCT04669600
- Testing
- SAR445088 (BIVV020)
- Where
- 9 sites in United States, Czechia and 4 more
- Volunteers
- 12 people
- Sponsor
- Bioverativ, a Sanofi company
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
Thrombocytopenia in Pediatric Subjects With Immune Idiopathic Thrombocytopenic Purpura ITP
A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura
This is an extension study designed to assess the safety and durability of platelet count increases with romiplostim treatment of thrombocytopenic patients with immune (Idiopathic) thrombocytopenia purpura.
- Trial name
- NCT01071954
- Testing
- Romiplostim
- Where
- 31 sites in United States, Australia and 2 more
- Volunteers
- 66 people
- Sponsor
- Amgen
Thrombocythemia, Essential
Hematology, IMG-7289, LSD1 (Lysine-Specific Demethylase 1) Inhibitor, Essential Thrombocythemia (ET), Ph 2
This is a single-center, open-label investigator-initiated trial evaluating the effects of IMG-7289 administered orally once daily in patients with essential thrombocythemia.
- Trial name
- NCT04081220
- Testing
- IMG-7289
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
Immune Thrombocytopenia
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
This was a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in…
- Trial name
- LUNA 3 (NCT04562766)
- Testing
- Rilzabrutinib
- Where
- 155 sites in United States, Argentina and 24 more
- Volunteers
- 232 people
- Sponsor
- Principia Biopharma, a Sanofi Company
Hemophilia A
Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq
Uncontrolled, multi-centre, non-interventional study with a prospective and a retrospective cohort, to evaluate the efficacy of Wilate or Nuwiq in achieving complete or partial immune tolerance induction (ITI) success in severe…
- Trial name
- PREVAIL (NCT03344003)
- Testing
- Wilate or Nuwiq
- Where
- 3 sites in Canada
- Volunteers
- 14 people
- Sponsor
- Octapharma
Immune Thrombocytopenia (ITP)
A Study to Assess Efficacy and Safety of Eltrombopag in Combination With a Short Course of Dexamethasone in Patients With Newly Diagnosed ITP
The purpose of this study was to compare the ability of eltrombopag in combination with a short course of high-dose dexamethasone to induce sustained response off treatment in patients with newly-diagnosed ITP versus 1-3 cycles…
- Trial name
- XPAG-ITP (NCT04346654)
- Testing
- Eltrombopag, Dexamethasone
- Where
- 10 sites in Germany
- Volunteers
- 26 people
- Sponsor
- Novartis Pharmaceuticals
High Risk Polycythemia Vera, High Risk Essential Thrombocythemia
Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea in Polycythemia Vera (PV) and Essential Thrombocythemia (ET)
This research is looking at two conditions, Essential Thrombocythemia (ET) and Polycythemia Vera (PV). ET causes people to produce too many blood cells called platelets and PV causes too many platelets and red blood cells to be…
- Trial name
- NCT01259856
- Testing
- PEGASYS, Hydroxyurea, Aspirin
- Where
- 25 sites in United States, France and 2 more
- Volunteers
- 168 people
- Sponsor
- Ronald Hoffman
Hemophilia A
Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
The primary purpose of this study is to describe the outcome of Immune Tolerance Induction (ITI) treatment performed with rFVIIIFc within a timeframe of 60 weeks in patients with haemophilia A who have failed previous attempts at…
- Trial name
- ReITIrate (NCT03103542)
- Testing
- Recombinant coagulation factor (rFVIIIFc)
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 16 people
- Sponsor
- Swedish Orphan Biovitrum
Immune Thrombocytopenic Purpura
Open Label Study of R788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
The primary objective of this study was to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP
- Trial name
- NCT02077192
- Testing
- Fostamatinib Disodium
- Where
- 55 sites in United States, Australia and 13 more
- Volunteers
- 123 people
- Sponsor
- Rigel Pharmaceuticals
Idiopathic Thrombocytopenic Purpura
A Study of MabThera (Rituximab) in Patients With Idiopathic Thrombocytopenic Purpura.
This single arm study will evaluate the efficacy and safety of MabThera monotherapy in patients with refractory, relapsing or chronic idiopathic thrombocytopenic purpura (ITP).
- Trial name
- NCT00475423
- Testing
- rituximab [MabThera/Rituxan]
- Where
- 12 sites in Australia
- Volunteers
- 122 people
- Sponsor
- Hoffmann-La Roche