Clinical trials: Bone Marrow Failure Syndromes
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.
Aplastic Anemia
ATGAM General Investigation
The objective of this study is to confirm the safety of ATGAM in patients with moderate to severe aplastic anemia under the actual use in Japan.
- Trial name
- NCT06039020
- Testing
- Anti-human thymocyte immunoglobulin, equine
- Where
- Japan
- Volunteers
- 1 people
- Sponsor
- Pfizer
Severe Aplastic Anemia (SAA)
Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy
Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to…
- Trial name
- NCT05323617
- Testing
- Romiplostim, Antithymocyte Globulin, Cyclosporine A
- Sponsor
- Amgen
Bone Marrow Failure Disorders
Clinical Study of UCB Combined With UC-MSCs in the Treatment of Bone Marrow Failure Disorders
The multicenter collaborative clinical study conducted a systematic clinical observation in the treatment of bone marrow failure diseases via UCB\&UC-MSCs , in order to observe its clinical efficacy and safety.
- Trial name
- NCT05794425
- Testing
- Cyclosporine A, Umbilical cord blood & Umbilical cord derived mesenchymal stem cells
- Where
- 8 sites in China
- Volunteers
- 100 people
- Sponsor
- Shandong Qilu Stem Cells Engineering Co., Ltd.
Myelodysplastic Syndrome, MDS
Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMA
The study's primary objective [in a population of patients with MDS after failure of treatment with azacitidine (AZA) or decitabine (DAC)], is to compare the overall survival (OS) of patients in the rigosertib group vs the…
- Conditions
- Myelodysplastic Syndrome, MDS, Refractory Anemia With Excess Blasts, RAEB
- Trial name
- INSPIRE (NCT02562443)
- Testing
- rigosertib, Any approved or standard-of-care therapy, best supportive care (BSC)
- Where
- 158 sites in United States, Australia and 20 more
- Volunteers
- 372 people
- Sponsor
- Traws Pharma, Inc.
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
Multiple Myeloma
A Safety and Efficacy of Pomalyst® Capsules Under the Actual Use in All Patients Who Are Treated With Pomalyst at a Dose of 1 mg, 2 mg, 3 mg, or 4 mg
1. Planned enrollment period One year (The planned number of patients to be enrolled is set to 400 patients.) Since all patients who are prescribed with Pomalyst are registered in RevMate®, enrollment using the Registration Form…
- Trial name
- NCT02921828
- Where
- 2 sites in United States, Japan
- Volunteers
- 1,149 people
- Sponsor
- Celgene
Pure Red-Cell Aplasia, Chronic Kidney Failure
Surveillance Study to Estimate the Incidence of Pure Red Blood Cell Aplasia Among Patients With Chronic Kidney Failure
The purpose of this study is to estimate the incidence rate of pure red cell aplasia (PRCA; aplastic anemia) mediated by erythropoietin (EPO) antibodies in patients who are receiving subcutaneous (s.c.) epoetin alfa (polysorbate…
- Trial name
- PRIMS (NCT00391287)
- Where
- 387 sites in Australia, Belgium and 13 more
- Volunteers
- 15,334 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
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
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.
Myelodysplastic Syndrome
A Study to Evaluating the Pharmacokinetics, Safety, and Efficacy of ATG 016 Monotherapy in IPSS-R Intermediate Risk and Above Myelodysplastic Syndrome (MDS)
This is a Phase Ⅰ/II, Open-label Study to Investigate the Pharmacokinetics, Safety, and Efficacyof ATG 016 Monotherapy in IPSS-R Intermediate Risk and above Myelodysplastic Syndrome (MDS) Patients after Failure of Hypomethylating…
- Trial name
- HATCH (NCT04691141)
- Testing
- ATG-016
- Where
- 6 sites in China
- Volunteers
- 15 people
- Sponsor
- Shanghai Antengene Corporation Limited
Aplastic Anemia
A Study of the Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia
The aim of this study was to investigate the safety, tolerability, and effectiveness of eltrombopag in combination with immunosuppressive therapy (IST) in real world practice in pediatric patients with severe aplastic anemia…
- Trial name
- NCT07415083
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia
Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects
This was an open label, non-randomized, phase II study of eltrombopag in combination with rabbit ATG/CsA in subjects with moderate or more severe AA who did not received prior ATG/ALG-based immunosuppressive therapy.
- Trial name
- NCT02404025
- Testing
- Eltrombopag, Rabbit ATG, CsA
- Where
- 11 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
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.
Aplastic Anemia, Hypoplastic Myelodysplastic Syndrome
Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome
An open label single arm study to assess efficacy and safety of BL-8040 on top of standard immunotherapy regimen of hATG, cyclosporine and steroids in patients with Hypoplastic MDS and AA over the course of a six month (180 day)…
- Trial name
- NCT02462252
- Testing
- BL-8040, horse anti-thymocyte globulin (hATG), Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- BioLineRx, Ltd.