Clinical trials: Bone Marrow Disease
Polycythemia Vera (SNOMED CT: 109992005)
Phase 2 Study of Oral Epetraborole in Adults With Polycythemia Vera
This is a Phase 2 trial to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of oral epetraborole tablets for the treatment of adults with phlebotomy-dependent polycythemia vera (PV).
- Trial name
- NCT07891533
- Testing
- Epetraborole
- Where
- Australia
- Volunteers
- 75 people
- Sponsor
- AN2 Therapeutics, Inc
SSM, Mastocytosis, Indolent
(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
This is a multi-part, randomized, double-blind, placebo-controlled Phase 2 clinical study comparing the safety and efficacy of bezuclastinib (CGT9486) plus best supportive care (BSC) with placebo plus BSC in patients with…
- Conditions
- SSM, Mastocytosis, Indolent, Mastocytosis, Systemic, Mastocytosis
- Trial name
- NCT05186753
- Testing
- Bezuclastinib Tablets (Formulation A), Bezuclastinib Tablets (Formulation B)
- Where
- 57 sites in United States, Australia and 13 more
- Volunteers
- 237 people
- Sponsor
- Cogent Biosciences, Inc.
Bone Marrow Disease
A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)
The goal of this clinical research study is to determine whether it is safe and practical to give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow failure syndrome.
- Trial name
- NCT03773393
- Testing
- CK0801
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Cellenkos, Inc.
Myelofibrosis
Leading in MPNs Beyond Ruxolitinib in Combo With T-Regs
To assess the safety and tolerability of CK0804 as add-on therapy in participants with myelofibrosis, with suboptimal response to ruxolitinib
- Trial name
- TREG108 (NCT05423691)
- Testing
- CK0804
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- Cellenkos, Inc.
Anemia
A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India
The purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in India
- Trial name
- NCT05891249
- Testing
- Luspatercept
- Where
- 9 sites in India
- Volunteers
- 85 people
- Sponsor
- Bristol-Myers Squibb
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
Osteoporosis, Osteoarthritis
OSTEOMICS: Identifying Regulators of Bone Homeostasis
Diseases of bone associated with ageing, including osteoporosis (OP) and osteoarthritis (OA), reduce bone mass, bone strength and joint integrity.
- Conditions
- Osteoporosis, Osteoarthritis, Bone Diseases, Bone Fracture
- Trial name
- OSTEOMICS (NCT05732870)
- Testing
- Relevant orthopaedic surgery
- Where
- 8 sites in United Kingdom
- Volunteers
- 2,000 people
- Sponsor
- Relation Therapeutics
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
This study will evaluate the safety, tolerability, and efficacy of Orca-T in participants undergoing reduced intensity or non-myeloablative allogeneic hematopoietic cell transplantation (alloHCT) for hematologic malignancies.
- Conditions
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Mixed Phenotype Acute Leukemia
- Trial name
- NCT07216443
- Testing
- Orca-T
- Where
- 6 sites in United States
- Volunteers
- 80 people
- Sponsor
- Orca Biosystems, 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
Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis
A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis
This study (study ID PAC203 North America; PAC303 ex-North America) is evaluating 200 mg BID of pacritinib compared to physician's choice (P/C) therapy in patients with MF and severe thrombocytopenia (platelet count <50,000/μL).
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Trial name
- PACIFICA (NCT03165734)
- Testing
- Pacritinib, Physician's Choice medications
- Where
- 207 sites in United States, Australia and 22 more
- Volunteers
- 407 people
- Sponsor
- Swedish Orphan Biovitrum
Fanconi Anemia Complementation Group A, Fanconi Anemia
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial.
- Trial name
- NCT04437771
- Testing
- Safety and efficacy assessments
- Where
- Spain
- Volunteers
- 9 people
- Sponsor
- Rocket Pharmaceuticals Inc.
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
Leukemia, Leukemia, Myeloid
Study of PDR001 and/or MBG453 in Combination With Decitabine in Patients With AML or High Risk MDS
To characterize the safety and tolerability of 1) MBG453 as a single agent or in combination with PDR001 or 2) PDR001 and/or MBG453 in combination with decitabine or azacitidine in AML and intermediate or high- risk MDS patients…
- Conditions
- Leukemia, Leukemia, Myeloid, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
- Trial name
- NCT03066648
- Testing
- Decitabine, PDR001, MBG453
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 241 people
- Sponsor
- Novartis Pharmaceuticals
Acute Myeloid Leukemia, B-cell Acute Lymphoblastic Leukemia
Study of VIP943 in Subjects With Advanced CD123+ Hematologic Malignancies
Dose Escalation - Determine the maximum tolerated dose (MTD), if possible, or minimum optimal biologic dose (OBD), and evaluate the safety and tolerability of VIP943 in subjects with advanced CD123+ hematologic malignancies
- Conditions
- Acute Myeloid Leukemia, B-cell Acute Lymphoblastic Leukemia, High-risk Myelodysplastic Syndrome
- Trial name
- NCT06034275
- Testing
- VIP943 (QW), VIP943 (BIW)
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Vincerx Pharma, Inc.
Myelodysplastic Syndromes, Anemia
A Study of HS-20106 to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of HS-20106 on anemia in patients with very low, low or intermediate risk MDS.
- Conditions
- Myelodysplastic Syndromes, Anemia, MDS, Bone Marrow Disease
- Trial name
- NCT06594965
- Testing
- HS-20106
- Where
- China
- Volunteers
- 176 people
- Sponsor
- Hansoh BioMedical R&D Company
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
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
Thrombocytopenia, Hematologic Diseases
Thrombosomes® in Bleeding Thrombocytopenic Patients
The study evaluates the safety and potential early signals of efficacy of allogeneic Thrombosomes in bleeding thrombocytopenic patients
- Conditions
- Thrombocytopenia, Hematologic Diseases, Bone Marrow Aplasia
- Trial name
- NCT03394755
- Testing
- Thrombosomes
- Where
- 7 sites in United States, Norway
- Volunteers
- 24 people
- Sponsor
- Cellphire Therapeutics, Inc.
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