Clinical trials: Bone Marrow Failure
Thrombocytopenia
Managed Access Programs for ETB115, Eltrombopag
The purpose of this registration is to list Managed Access Programs (MAPs) related to ETB115, Eltrombopag
- Trial name
- NCT04837703
- Testing
- Eltrombopag
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia
Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia
The objective of this study is to evaluate the efficacy of romiplostim administered once weekly to Aplastic Anemia (AA) patients with thrombocytopenia refractory to or ineligible for immunosuppressive therapy in Japan and Korea.
- Trial name
- NCT02773290
- Testing
- Romiplostim
- Where
- 3 sites in Japan, South Korea
- Volunteers
- 46 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Primary Immunodeficiencies, Inborn Errors of Metabolism
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
The aim of the trial is to describe the safety and efficacy of intravenous (i.v.) Treosulfan compared to the conventional (myeloablative) dose of i.v.
- Conditions
- Primary Immunodeficiencies, Inborn Errors of Metabolism, Haemoglobinopathies, Bone Marrow Failure Syndromes
- Trial name
- NCT02349906
- Testing
- Treosulfan, Busilvex
- Where
- 21 sites in Czechia, Germany and 2 more
- Volunteers
- 106 people
- Sponsor
- medac GmbH
Heart Failure, Systolic
CardiAMP™ Cell Therapy for Heart Failure Trial
This is a prospective, multi-center, randomized, controlled, patient- and evaluator-blinded study comparing treatment with the CardiAMP cell therapy to a sham control treatment.
- Trial name
- NCT02438306
- Testing
- Autologous cell therapy
- Where
- 24 sites in United States, Canada
- Volunteers
- 125 people
- Sponsor
- BioCardia, Inc.
Lymphoma Nonhodgkin
Revlimid® Capsules General Drug Use-results Surveillance (Relapsed or Refractory FL and MZL)
To ascertain the safety of Revlimid® Capsules 2.5 mg or 5 mg under the actual use conditions in patients who received R2 combination therapy with the drug and rituximab for the first time for relapsed or refractory follicular…
- Trial name
- NCT04618081
- Where
- Japan
- Volunteers
- 151 people
- Sponsor
- Celgene
Advanced Solid Tumors
A Phase 1/2 Trial SRA737 in Combination With Gemcitabine and Cisplatin or Gemcitabine Alone in Advanced Cancer Subjects
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose (RP2D) and schedule of SRA737 in combination with low dose gemcitabine; and to…
- Trial name
- NCT02797977
- Testing
- SRA737, gemcitabine, cisplatin, SRA737, gemcitabine
- Where
- 20 sites in Spain, United Kingdom
- Volunteers
- 153 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Heart Failure
Safety and Efficacy of Autologous Cardiopoietic Cells for Treatment of Ischemic Heart Failure.
Evaluation the safety and efficacy of C3BS-CQR-1 by comparing the overall response to standard of care and C3BS-CQR-1 relative to standard of care and a sham procedure.
- Trial name
- CHART-1 (NCT01768702)
- Testing
- Injection of C3BS-CQR-1
- Where
- 35 sites in Belgium, Bulgaria and 7 more
- Volunteers
- 315 people
- Sponsor
- Celyad Oncology SA
Heart Failure, Systolic
Clinical Trial of Human Allogenic Culture-expanded Bone Marrow-derived Mesenchymal Stem Cells
This clinical study will utilize allogenic bone marrow-derived culture-expanded MSC that are expanded from mesenchymal stem cells and delivered using the investigational Helix transendocardial delivery catheter as a therapy for…
- Trial name
- CardiALLO (NCT05925608)
- Testing
- CardiALLO™ Human Allogenic Culture-expanded Bone marrow-derived Mesenchymal Stem Cells (hMSCs), Diagnostic Catheterization
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- BioCardia, Inc.
Fanconi Anemia
A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia
This is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and…
- Trial name
- NCT04522375
- Testing
- FP-045
- Sponsor
- Foresee Pharmaceuticals Co., Ltd.
Dilated Cardiomyopathy
Use of Ixmyelocel-T (Formerly Cardiac Repair Cell [CRC] Treatment) in Patients With Heart Failure Due to Dilated Cardiomyopathy (IMPACT-DCM)
This study is designed to assess the safety and tolerability of Cardiac Repair Cells (CRCs) compared to standard-of-care in patients with dilated cardiomyopathy (DCM).
- Trial name
- NCT00765518
- Testing
- Ixmyelocel-T, Standard of Care
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Vericel Corporation
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.
Aplastic Anemia (AA)
Effectiveness of Early Intervention in Transfusion Independent Aplastic Anemia: a Retrospective Medical Claims Database Study
This was a retrospective non-interventional cohort study with secondary use of data from the Medical Data Vision (MDV) hospital-based database to evaluate the effectiveness of early drug intervention in preventing transfusion…
- Trial name
- NCT06669221
- Where
- Japan
- Volunteers
- 1,603 people
- Sponsor
- Novartis Pharmaceuticals
Severe Aplastic Anemia
A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia （SAA） Patient
This is a Multicentre, Open-label, single-arm, Phase II Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Patients With Severe Aplastic Anemia. 55 adult patients with SAA will be enrolled in the study.
- Trial name
- NCT03557099
- Testing
- Hetrombopag Olamine
- Where
- China
- Volunteers
- 55 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
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
- 4 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Aplastic Anemia
A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia
The purpose of the study is to assess the efficacy and safety of PF-06462700 administered intravenously at 40 mg/kg/day for 4 days in Japanese participants with moderate and above aplastic anemia for making an approval…
- Trial name
- NCT04350606
- Testing
- PF-06462700
- Where
- 3 sites in Japan
- Volunteers
- 3 people
- Sponsor
- Pfizer
Aplastic Anemia
A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia
This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric…
- Trial name
- NCT03025698
- Testing
- Eltrombopag, hATG, CsA
- Where
- 20 sites in United States, Hong Kong and 4 more
- Volunteers
- 51 people
- Sponsor
- Novartis Pharmaceuticals
Transfusional Iron Overload
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity
The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional…
- Trial name
- NCT01044186
- Testing
- ICL670
- Where
- 18 sites in United States, Greece and 1 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Renal Failure
Safety and Efficacy of Autologous Bone Marrow Stem Cells for Treating Chronic Renal Failure
Chronic renal failure (CRF, or "chronic kidney failure", CKF, or "chronic kidney disease", CKD) is a slowly progressive loss of renal function over a period of months or years and defined as an abnormally low glomerular…
- Trial name
- NCT01152411
- Testing
- Autologous bone marrow stem cells
- Where
- India
- Volunteers
- 5 people
- Sponsor
- International Stemcell Services Limited
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