Clinical trials: Blood Platelet Disorder
Primary Immune Thrombocytopenia
A Study to Evaluate the Efficacy and Safety of Orelabrutinib in Adult Patients With Immune Thrombocytopenia
The study is designed to be a randomized, open, multi-center, phase IIa/IIb seamless adaptive trial. Phase IIa: The study consists of a screening period, a core treatment period, an open label extension period, and a safety…
- Trial name
- NCT05232149
- Testing
- Orelabrutinib( lower dose), Orelabrutinib( higher dose)
- Where
- 9 sites in China
- Volunteers
- 30 people
- Sponsor
- Beijing InnoCare Pharma Tech Co., Ltd.
Myelodysplastic Syndromes, Thrombocytopenia
A Safety and Efficacy Study to Evaluate AMG 531 Treatment in Subject With Myelodysplastic Syndrome Receiving Revlimid
This is a dose and schedule finding study of AMG 531 designed to assess the activity of AMG 531 to reduce the rate of clinically significant bleeding and blood transfusions in subjects with myelodysplastic syndrome (MDS)…
- Trial name
- NCT00418665
- Testing
- AMG 531
- Volunteers
- 39 people
- Sponsor
- Amgen
Healthy Volunteers
Safety and Tolerability of ZE74-0282 in Healthy Volunteers
This first-in-human, randomized, double-blind, placebo-controlled Phase 1 study evaluates the safety, tolerability, and pharmacokinetics of single, split, and multiple oral doses of ZE74-0282 under fasted or fed conditions in…
- Trial name
- NCT07527221
- Where
- Australia
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Eilean Therapeutics AU Pty Ltd
MPN (Myeloproliferative Neoplasms)
Myelofibrosis and Essential Thrombocythemia Observational Study (MOST)
The purpose of this prospective, longitudinal, noninterventional study is to describe clinical characteristics, evolution of disease burden, and treatment patterns in patients with select subcategories of essential…
- Trial name
- NCT02953704
- Where
- 116 sites in United States, Puerto Rico
- Volunteers
- 1,469 people
- Sponsor
- Incyte Corporation
Thrombotic Thrombocytopenic Purpura (TTP)
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body.
- Trial name
- NCT04683003
- Testing
- TAK-755
- Where
- 27 sites in United States, Austria and 9 more
- Volunteers
- 77 people
- Sponsor
- Takeda
Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplant
A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Transplant (HSCT).
The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and…
- Trial name
- ALX-TMA-502 (NCT07732361)
- Where
- 43 sites in United States, Belgium and 8 more
- Volunteers
- 307 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Thrombotic Thrombocytopenic Purpura (TTP)
A Study to Learn More About the Treatment of People With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Who Received Recombinant ADAMTS13 (rADAMTS13) as Part of the Early Access Program
Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by inherited changes in the ADAMTS13 gene that reduce the body's ability to produce the ADAMTS13 enzyme.
- Trial name
- NCT07429942
- Testing
- No Intervention
- Where
- 8 sites in Germany, Poland
- Volunteers
- 94 people
- Sponsor
- Takeda
Thrombocytopenia Associated With Chronic Liver Disease
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once-daily Oral Avatrombopag in Japanese Subjects With Chronic Liver Diseases and Thrombocytopenia
This is a phase 2, randomized, double-blind, placebo-controlled, parallel group study using avatrombopag for Japanese subjects with thrombocytopenia associated with chronic liver disease.
- Trial name
- NCT02227693
- Testing
- avatrombopag
- Where
- 14 sites in Japan
- Volunteers
- 39 people
- Sponsor
- Eisai Co., Ltd.
Immune Thrombocytopenic Purpura
A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
The purpose of this study is to determine whether fostamatinib is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP).
- Trial name
- FIT (NCT02076399)
- Testing
- Fostamatinib disodium
- Where
- 52 sites in United States, Australia and 6 more
- Volunteers
- 76 people
- Sponsor
- Rigel Pharmaceuticals
Autoimmune Hemolytic Anemia, Immune Thrombocytopenia
A Phase 2 Study to Evaluate the Safety and Efficacy of KZR-616 in Patients With AIHA and ITP
This is a Phase 2 randomized, dose-blind, multicenter study designed to evaluate the safety, tolerability, efficacy, Pharmacokinetics (PK), and Pharmacodynamics (PD) of treatment with KZR-616 in patients with active Autoimmune…
- Trial name
- MARINA (NCT04039477)
- Testing
- KZR-616
- Where
- 22 sites in United States, Australia and 3 more
- Sponsor
- Kezar Life Sciences, Inc.
Aplastic Anemia
A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment
This research study is testing a medicine called avatrombopag in adults with aplastic anemia, a rare condition where the bone marrow does not produce enough blood cells.
- Trial name
- NCT07786922
- Testing
- Avatrombopag 20 mg Oral Tablet
- Volunteers
- 26 people
- Sponsor
- Swedish Orphan Biovitrum
Idiopathic Thrombocytopenic Purpura
A Study in Healthy Subjects to Evaluate Bioavailability of 4 Formulations of E5501
This is a study in healthy subjects. There are two parts to the study. In the first part of the study each subject will receive a single 10mg dose of each of the four formulations of E550.
- Trial name
- NCT01549054
- Testing
- 10-mg dose of E5501 2G tablet, 10-mg dose of E5501 cyclodextrin oral solution, 10-mg dose of E5501-P21% powder
- Where
- United Kingdom
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Eisai Inc.
Polycythemia Vera
A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV
A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients with PV
- Trial name
- NCT05481151
- Testing
- P1101 (Ropeginterferon alfa-2b-njft), Ropeginterferon alfa-2b-njft (P1101)
- Where
- 23 sites in United States, Canada
- Volunteers
- 111 people
- Sponsor
- PharmaEssentia
Thrombocythemia, Essential, Primary Myelofibrosis
A Study to Evaluate Safety and Efficacy of Bomedemstat (MK-3543-017)
The primary purpose of the study is to transition participants into an extension study to collect long-term safety and efficacy data.
- Conditions
- Thrombocythemia, Essential, Primary Myelofibrosis, Myelofibrosis, Post-polycythemia Vera Myelofibrosis
- Trial name
- NCT06351631
- Testing
- Bomedemstat
- Where
- 21 sites in United States, Australia and 4 more
- Volunteers
- 400 people
- Sponsor
- Merck Sharp & Dohme LLC
Atypical Hemolytic Uremic Syndrome
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
- Trial name
- NCT03303313
- Testing
- Cemdisiran
- Where
- 12 sites in Bosnia and Herzegovina, Canada and 8 more
- Sponsor
- Alnylam Pharmaceuticals
Myelodysplastic Syndrome
Eltrombopag Treatment of Thrombocytopenia in Subjects With Advanced Myelodysplastic Syndrome (MDS) or Secondary Acute Myeloid Leukemia After MDS (sAML/MDS)
This study will evaluate the safety and tolerability of eltrombopag in the treatment of low platelet counts in adult subjects with advanced myelodysplastic syndrome (MDS), secondary acute myeloid leukemia after MDS (sAML/MDS), or…
- Trial name
- NCT00903422
- Testing
- eltrombopag olamine
- Where
- 65 sites in United States, Brazil and 9 more
- Volunteers
- 98 people
- Sponsor
- GlaxoSmithKline
Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis
Safety and Tolerability Study of Oral NS-018 in Patients With Primary Myelofibrosis (MF), Post-polycythemia Vera MF or Post-essential Thrombocythemia MF
The purpose of this study is to determine the safety and tolerability of orally administered NS-018 in patients with Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (post-PV MF), or Post-essential…
- Conditions
- Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis
- Trial name
- NCT01423851
- Testing
- NS-018
- Where
- 9 sites in United States
- Volunteers
- 77 people
- Sponsor
- NS Pharma, Inc.
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Atypical Hemolytic Uremic Syndrome
The Burden of Atypical Hemolytic Uremic Syndrome and The Clinical Characteristics of Patients in Egyptian Hospitals A Multicenter, Observational, Retrospective Cohort Study in Egypt
Atypical hemolytic uremic syndrome (aHUS) is a rare, progressive, and life-threatening disease that occurs at any age, with incidence rate of 0.75 to 2.0 cases per million population per year.
- Trial name
- REACH (NCT07218536)
- Where
- 6 sites in Egypt
- Volunteers
- 200 people
- Sponsor
- AstraZeneca
Cancer
BCX-1777 in Treating Patients With Refractory Cancer
RATIONALE: BCX-1777 may stop the growth of cancer cells by blocking the enzymes necessary for their growth. PURPOSE: Phase I trial to study the effectiveness of BCX-1777 in treating patients who have refractory cancer.
- Trial name
- NCT00073944
- Testing
- forodesine hydrochloride
- Where
- United States
- Sponsor
- BioCryst Pharmaceuticals