Clinical trials: Bone Marrow Failure Syndrome
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
Sickle Cell Disease
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 to halt disease progression.
- Trial name
- NCT00777231
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Talaris Therapeutics Inc.
Aplastic Anemia
A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the…
- Trial name
- NCT02094417
- Testing
- AMG531
- Where
- South Korea
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Korea Co., Ltd.
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.
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
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
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.
Myelodysplastic Syndromes
Trial of Volasertib With or Without Azacitidine in Patients With Myelodysplastic Syndromes
The objectives of this trial are to evaluate the safety, tolerability, maximum tolerated dose (MTD), pharmacokinetics and preliminary efficacy of volasertib in two dosing schedules of intravenous volasertib as monotherapy or in…
- Trial name
- NCT02721875
- Testing
- Volasertib, Azacitidine
- Where
- Japan
- Volunteers
- 1 people
- Sponsor
- Boehringer Ingelheim
Aplastic Anemia
Expanded Access Use of Itacitinib to Treat a Single Patient With Aplastic Anemia
Expanded Access Use of Itacitinib to Treat a Single Patient With Aplastic Anemia
- Trial name
- NCT03906318
- Testing
- Iticitinib
- Sponsor
- Incyte Corporation
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
Aplastic Anemia
Study of AMG531(Romiplostim) in Patients With Aplastic Anemia
To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients…
- Trial name
- NCT03957694
- Testing
- Romiplostim
- Where
- Japan
- Volunteers
- 17 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Severe Aplastic Anemia (SAA)
REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
This study is researching an experimental drug called REGN7257 (called "study drug"). The study is focused on patients who have severe aplastic anemia (SAA), a disease of the bone marrow resulting in an impairment of the…
- Trial name
- NCT04409080
- Testing
- REGN7257
- Where
- 10 sites in United States, France and 2 more
- Volunteers
- 17 people
- Sponsor
- Regeneron Pharmaceuticals
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
Severe Aplastic Anemia
Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response
- Trial name
- SOAR (NCT02998645)
- Testing
- eltrombopag, Cyclosporine
- Where
- 20 sites in Brazil, Hong Kong and 7 more
- Volunteers
- 54 people
- Sponsor
- Novartis Pharmaceuticals
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
Anemia, Aplastic
Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia
Our hypothesis is that ex vivo activated immune cells would produce multiple known and unknown potent hematopoietic cytokines, working in concert, these cytokines help stem cell growth and differentiation.
- Trial name
- NCT00399971
- Testing
- Ex Vivo Immunotherapy
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Zhongxing Yangfan Biotech Co. Ltd.
Beta-thalassemia, Myelodysplastic Syndromes
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan
- Trial name
- NCT00061763
- Testing
- Deferasirox
- Where
- 6 sites in United States
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals
Thrombocytopenia, Hematologic Malignancy
Thrombosomes® in Bleeding Thrombocytopenic Patients Study
This prospective, multicenter, randomized, open-label, Phase 2, parallel, dose ranging, multidose trial will enroll patients into 3 Thrombosomes dose groups and 1 control liquid stored platelets (LSP) group in order to evaluate…
- Conditions
- Thrombocytopenia, Hematologic Malignancy, Bone Marrow Aplasia, Myeloproliferative Disorders
- Trial name
- NCT04631211
- Testing
- Thrombosomes, Liquid Stored Platelets (LSP)
- Where
- 6 sites in United States, Israel and 1 more
- Volunteers
- 21 people
- Sponsor
- Cellphire Therapeutics, Inc.
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
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.