Clinical trials: Bone Marrow Failure Syndrome
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.
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.
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match
The purpose of this study is to evaluate the multi-lineage hematopoietic chimerism for unrelated umbilical cord blood (UCB) grafts pooled from two to three cord blood units.
- Conditions
- Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia
- Trial name
- NCT01500161
- Testing
- Busulfan, Clofarabine, Fludarabine
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Texas Oncology Cancer Center
Severe Aplastic Anemia
Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
This is a multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive. 180 treated naive patients with SAA will be enrolled in the study.
- Trial name
- NCT03825744
- Testing
- Hetrombopag Olamine+Standard Therapy
- Where
- China
- Volunteers
- 240 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Fanconi Anemia
Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Fanconi Anemia. The biospecimens will be used to create a biorepository that can be used to identify disease associated…
- Trial name
- NCT05910853
- Testing
- Specimen Donation
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sanguine Biosciences
Multiple Myeloma, Myelodysplastic Syndromes
A Study of Special Use Results Surveillance of Revlimid 5mg Capsules
To understand the safety and efficacy of Revlimid® 5 mg Capsules (hereinafter referred to as Revlimid) in all patients who are treated with it under the actual condition of use pursuant to the conditions of approval. 1.
- Trial name
- NCT02921802
- Where
- Japan
- Volunteers
- 4,626 people
- Sponsor
- Celgene
Aplastic Anemia
Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Chinese subjects with refractory or relapsed severe aplastic anemia (SAA).
- Trial name
- NCT03988608
- Testing
- Eltrombopag
- Where
- 5 sites in China
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Transfusion-dependent Iron Overload
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.
- Trial name
- NCT00171821
- Testing
- Deferasirox
- Where
- 97 sites in Australia, Austria and 21 more
- Volunteers
- 1,784 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Solid Tumors or Non-Hodgkin's Lymphoma (NHL)
A Phase 1/2 Trial of SRA737 in Subjects With Advanced Cancer
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose and schedule of SRA737; and to evaluate the efficacy of SRA737 in…
- Trial name
- NCT02797964
- Testing
- SRA737
- Where
- 15 sites in United Kingdom
- Volunteers
- 107 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Aplastic Anemia
Long Term Follow-up Observational Study After Clinical Trials of AMG531 (Romiplostim) in Patients With Untreated Aplastic Anemia
To evaluate the long-term efficacy and safety after the end of romiplostim treatment by observation [up to 5 years] of patients who were registered for Study 531-003/531-004 in immunosuppressive therapy-naïve patients with…
- Trial name
- NCT04870346
- Where
- Japan
- Volunteers
- 36 people
- Sponsor
- Kyowa Kirin Co., Ltd.
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
Telomere Shortening, Bone Marrow Failure
Study to Evaluate of EXG34217 in Patients With Telomere Biology Disorders With Bone Marrow Failure
This is a Phase I/II, open label, single center study to assess the safety and tolerability of EXG34217 in bone marrow failure patients with telomere biology disorders.
- Trial name
- NCT04211714
- Testing
- EXG34217
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Elixirgen Therapeutics, Inc.
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
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