Clinical trials: Bone Marrow Failure, open to volunteers
- Condition Bone Marrow Failure
- Status Open to volunteers
- Funder type Industry (pharma, biotech, devices)
Aplastic Anemia
Revolade Tablets Specified Drug-use Survey
This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).
- Trial name
- NCT06287268
- Testing
- eltrombopag
- Where
- 17 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
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
Fanconi Anemia
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
- Trial name
- NCT07527975
- Testing
- RP-L102
- Where
- 3 sites in United States, Spain and 1 more
- Volunteers
- 14 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Ischemic Heart Failure
CardiAMP Heart Failure II Trial for Patients With Ischemic Heart Failure With Reduced Ejection Fraction
Concentrated autologous bone marrow mononuclear cells (ABM MNC) contain potentially therapeutic cell factors and past studies support therapeutic benefit to patients with cardiac diseases of acute myocardial infarction, ischemia…
- Trial name
- NCT06258447
- Testing
- Autologous bone marrow mononuclear cells processed / delivered using the CardiAMP cell therapy system
- Where
- 5 sites in United States
- Volunteers
- 250 people
- Sponsor
- BioCardia, Inc.
Aplastic Anemia
CK0801 for Treatment of Aplastic Anemia
This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care…
- Trial name
- NCT07499102
- Testing
- CK0801
- Volunteers
- 12 people
- Sponsor
- Cellenkos, Inc.
Epstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications
A Phase 3 Study of Tabelecleucel for Participants With Epstein-Barr Virus-Associated Post-Transplant Lymphoproliferative Disease After Failure With Rituximab or Rituximab and Chemotherapy
The purpose of this study is to determine the clinical benefit and characterize the safety profile of tabelecleucel for the treatment of Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) in the…
- Conditions
- Epstein-Barr Virus+ Associated Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications, Lymphoproliferative Disorders, Allogeneic Hematopoietic Cell Transplant
- Trial name
- ALLELE (NCT03394365)
- Testing
- tabelecleucel
- Where
- 71 sites in United States, Australia and 7 more
- Volunteers
- 115 people
- Sponsor
- Pierre Fabre Medicament
Neutropenia
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are…
- Trial name
- NCT06056297
- Testing
- Mavorixafor
- Where
- 114 sites in United States, Argentina and 23 more
- Volunteers
- 176 people
- Sponsor
- X4 Pharmaceuticals
Chronic Myeloid Leukemia, Chronic Phase, CML, Chronic Phase
A Phase 3 Study for the Efficacy and Safety of Radotinib in CP-CML Patients With Failure or Intolerance to Previous TKIs
In a multinational, multicenter, single-arm, open-label and Phase III Radotinib clinical study, chronic phase Ph+ chronic myeloid leukemia patients with failure or intolerance to previous TKIs therapy including Imatinib will be…
- Conditions
- Chronic Myeloid Leukemia, Chronic Phase, CML, Chronic Phase, CML, Refractory, CML - Philadelphia Chromosome
- Trial name
- NCT03459534
- Testing
- Radotinib HCl
- Where
- 18 sites in Russia, South Korea and 2 more
- Volunteers
- 173 people
- Sponsor
- Il-Yang Pharm. Co., Ltd.
Aplastic Anemia
Study of CM313(SC) Injection in Subjects With Relapsed/Refractory Aplastic Anemia
This is a randomized, open-label, phase IB/II clinical study to evaluate the safety, tolerability and preliminary efficacy of CM313(SC) injection in patients with relapsed/refractory aplastic anemia
- Trial name
- NCT06791824
- Testing
- CM313(SC) injection
- Where
- China
- Volunteers
- 125 people
- Sponsor
- Keymed Biosciences Co.Ltd
Telomere Biology Disorders With Bone Marrow Failure
Rollover Study From EXG-US-01
This is a long-term rollover follow-up study for Phase I/II study (Protocol EXG-US-01).
- Trial name
- NCT05868499
- Testing
- EXG34217
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Elixirgen Therapeutics, 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
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.