Clinical trials: Bone Marrow Disease
Infantile Malignant Osteopetrosis
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.
- Trial name
- NCT04525352
- Testing
- RP-L401
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Chronic Myeloid Leukemia (CML), Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)
A Study of Ponatinib in Japanese Participants With Chronic Myeloid Leukemia (CML) and Ph+ Acute Lymphoblastic Leukemia (ALL)
The purpose of this study is to assess the safety and efficacy of ponatinib in Japanese patients with chronic myeloid leukemia (CML) who have experienced failure of dasatinib or nilotinib or with Ph+ acute lymphoblastic leukemia…
- Trial name
- NCT01667133
- Testing
- ponatinib - Phase 1, ponatinib - Phase 2
- Where
- 11 sites in Japan
- Volunteers
- 35 people
- Sponsor
- Ariad Pharmaceuticals
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.
Chronic Phase Chronic Myeloid Leukemia
A Study Comparing Ponatinib and Nilotinib in Participants With Chronic Myeloid Leukemia
The purpose of this study is to compare the efficacy and safety of 2 starting doses of ponatinib compared to nilotinib in participants with imatinib-resistant chronic myeloid leukemia (CML) in chronic phase (CP).
- Trial name
- OPTIC-2L (NCT02627677)
- Testing
- Ponatinib 30 mg QD, Ponatinib 15 mg QD, Nilotinib 400 mg BID
- Where
- Belgium
- Volunteers
- 44 people
- Sponsor
- Ariad Pharmaceuticals
Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis
Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia
Phase 3, randomized, controlled study to evaluate the safety and efficacy of oral pacritinib compared to Best Available Therapy (BAT) in patients with thrombocytopenia and primary or secondary myelofibrosis.
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Trial name
- PAC326 (NCT02055781)
- Testing
- Pacritinib, Best Available Therapy
- Where
- 122 sites in United States, Australia and 10 more
- Volunteers
- 311 people
- Sponsor
- CTI BioPharma
Paroxysmal Nocturnal Hemoglobinuria, Marchiafava-Micheli Syndrome
Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH
It is a multicenter, open-label, randomized, parallel-group study of the efficacy and safety of Elizaria® (eculizumab, GENERIUM JSC, Russia) versus Soliris® (Alexion Pharma GmbH, Switzerland) in patients with paroxysmal nocturnal…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, Marchiafava-Micheli Syndrome, Paroxysmal Hemoglobinuria
- Trial name
- NCT04463056
- Testing
- Elizaria®, Soliris®
- Where
- 4 sites in Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Polycythemia Vera
Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
This study compared the efficacy and safety of ruxolitinib to Best Available Therapy (BAT) in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and did not have a palpable spleen.
- Trial name
- RESPONSE-2 (NCT02038036)
- Testing
- Best Available Therapy, Ruxolitinib
- Where
- 48 sites in Australia, Belgium and 10 more
- Volunteers
- 149 people
- Sponsor
- Novartis Pharmaceuticals
Polycythemia Vera
AOP2014 vs. BAT in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study.
Polycythemia Vera (PV) is a disease of bone marrow stem cells that manifests in a drastic increase of red blood cells and frequently also of white blood cells.
- Trial name
- CONTI-PV (NCT02218047)
- Testing
- Pegylated-Proline-interferon alpha-2b, Best available therapy (BAT)
- Where
- 47 sites in Austria, Bulgaria and 10 more
- Volunteers
- 170 people
- Sponsor
- AOP Orphan Pharmaceuticals AG
Leukemia, Myelogenous, Chronic, Breakpoint Cluster Region-Abelson Proto-oncogene (BCR-ABL) Positive
A Multicenter Phase 3, Open-Label Study of Bosutinib Versus Imatinib in Adult Patients With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia
Phase 3, 2-arm, randomized, open label trial. Patients will be randomized to receive bosutinib or imatinib for the duration of the study.
- Trial name
- NCT02130557
- Testing
- Bosutinib, Imatinib
- Where
- 189 sites in United States, Australia and 24 more
- Volunteers
- 536 people
- Sponsor
- Pfizer
MPN (Myeloproliferative Neoplasms)
Prospective Observational Study Of Patients With Polycythemia Vera In US Clinical Practices (REVEAL)
This is a Phase IV, multicenter, non-interventional, non-randomized, prospective, observational study in an adult population (patients >18 years old) of men and women who have been diagnosed with clinically overt PV and are being…
- Trial name
- NCT02252159
- Where
- 266 sites in United States, Puerto Rico
- Volunteers
- 2,544 people
- Sponsor
- Incyte Corporation
Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia
Open Label, Phase II Study to Evaluate Efficacy and Safety of Oral Nilotinib in Philadelphia Positive (Ph+) Chronic Myelogenous Leukemia (CML) Pediatric Patients.
To evaluate the safety, efficacy and pharmacokinetics of nilotinib over time in the Ph+ chronic myelogenous leukemia (CML) in pediatric patients (from 1 to <18 years).
- Trial name
- DIALOG (NCT01844765)
- Testing
- nilotinib
- Where
- 36 sites in United States, France and 11 more
- Volunteers
- 59 people
- Sponsor
- Novartis Pharmaceuticals
Myelogenous Leukemia, Chronic
A Study of Imatinib Versus Nilotinib in Adult Patients With Newly Diagnosed Philadelphia Chromosome Positive (Ph+) Chronic Myelogenous Leukemia in Chronic Phase (CML-CP)
In this study, the efficacy and safety of two nilotinib doses, 300 mg twice daily and 400 mg twice daily, were compared with imatinib 400 mg once daily in newly diagnosed patients with Philadelphia chromosome-positive (Ph+)…
- Trial name
- ENESTnd (NCT00471497)
- Testing
- nilotinib, imatinib
- Where
- 213 sites in United States, Argentina and 33 more
- Volunteers
- 846 people
- Sponsor
- Novartis Pharmaceuticals
Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease
Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)
An Early Access Program for patients with steroid refractory acute GvHD after hematopoietic stem cell transplantation. This Program is available for female and male who are recipients of allogenic HSCT and who have been newly…
- Conditions
- Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease, Graft-versus-host-disease, Graft Vs Host Disease
- Trial name
- NCT03172455
- Testing
- Glassia
- Sponsor
- Impatients N.V. trading as myTomorrows
Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis
Pacritinib Versus Best Available Therapy to Treat Myelofibrosis
Phase 3, randomized, controlled study to evaluate the safety and efficacy of oral pacritinib compared to Best Available Therapy (BAT) in patients with primary or secondary myelofibrosis.
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Trial name
- NCT01773187
- Testing
- Pacritinib, Best Available Therapy
- Where
- 81 sites in United States, Australia and 10 more
- Volunteers
- 327 people
- Sponsor
- CTI BioPharma
Myelofibrosis
Efficacy and Safety of Simtuzumab in Adults With Primary, Post Polycythemia Vera or Post Essential Thrombocythemia Myelofibrosis
This study is to evaluate the efficacy and safety of simtuzumab (GS-6624) on bone marrow fibrosis either alone or in combination with ruxolitinib in participants with primary myelofibrosis (PMF) and post polycythemia vera or post…
- Trial name
- NCT01369498
- Testing
- Simtuzumab, Ruxolitinib
- Where
- 7 sites in United States
- Volunteers
- 54 people
- Sponsor
- Gilead Sciences
Polycythemia Vera, Essential Thrombocythemia
Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia
This open-label study is to determine the safety and efficacy of momelotinib in participants with either polycythemia vera (PV) or essential thrombocythemia (ET) who have not yet received treatment with a Janus kinase (JAK)…
- Trial name
- NCT01998828
- Testing
- Momelotinib
- Where
- 15 sites in United States, Australia and 3 more
- Volunteers
- 39 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Primary Myelofibrosis, Thrombocytosis
A Phase Ib/II Dose-finding Study to Assess the Safety and Efficacy of LDE225 + INC424 in Patients With MF
The purpose of this phase Ib/II clinical trial was to: a) evaluate the safety of the co-administration of LDE225 and INC424 in myelofibrosis patients and establish a maximum tolerated dose and/or Recommended Phase II dose of the…
- Conditions
- Primary Myelofibrosis, Thrombocytosis, Essential Thrombocythemia, Polycythemia Vera
- Trial name
- NCT01787552
- Testing
- LDE225, INC424
- Where
- 18 sites in Australia, Belgium and 9 more
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndromes
Study of Azacitidine in Adult Taiwanese Subjects With Higher-Risk Myelodysplastic Syndromes (MDS)
The primary purpose of this study is to determine the effectiveness and safety of azacitidine in the treatment of Taiwanese subjects with higher-risk Myelodysplastic Syndrome (MDS).
- Trial name
- NCT01201811
- Testing
- Azacitidine
- Where
- 11 sites in Taiwan
- Volunteers
- 44 people
- Sponsor
- Celgene
Myelodysplastic Syndromes
An Efficacy Study for Epoetin Alfa in Anemic Patients With Myelodysplastic Syndromes
The purpose of this study is to demonstrate that epoetin alfa works better than placebo in improving anemia in patients with lower-risk myelodysplastic syndromes (MDS). The safety of epoetin alfa will also be evaluated.
- Trial name
- NCT01381809
- Testing
- Group 1: Epoetin alfa
- Where
- 28 sites in Bulgaria, France and 3 more
- Volunteers
- 130 people
- Sponsor
- Janssen-Cilag International NV
Leukemia, Leukemia, Myeloid
Radotinib Versus Imatinib in Newly Diagnosed Philadelphia Chromosome and Chronic Myeloid Leukemia Chronic Phase Patients
In this study, the efficacy and safety of two radotinib doses, 300 mg twice daily and 400 mg twice daily, will be compared with imatinib 400 mg once daily in newly diagnosed patients with Philadelphia chromosome-positive (Ph+)…
- Conditions
- Leukemia, Leukemia, Myeloid, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Philadelphia Chromosome
- Trial name
- NCT01511289
- Testing
- Imatinib, Radotinib
- Where
- 22 sites in Indonesia, Philippines and 2 more
- Volunteers
- 242 people
- Sponsor
- Il-Yang Pharm. Co., Ltd.