Clinical trials: Aplastic Anemias
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
Aplastic Anemia
Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia
To evaluate the hematological responses based on the response assessment criteria defined in this study (the 531-004 response assessment criteria) when AMG531 is subcutaneously (SC)-administered with ciclosporin A (CsA) therapy…
- Trial name
- NCT04095936
- Testing
- Romiplostim
- Where
- Japan
- Volunteers
- 24 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Iron Overload
Safety and Efficacy of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia.
- Trial name
- NCT01546415
- Testing
- Desferasirox
- Where
- 7 sites in China
- Volunteers
- 64 people
- Sponsor
- Novartis Pharmaceuticals
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
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.
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.
Pure Red-cell Aplasia
A Study of Patients With Pure Red Cell Aplasia Associated With Recombinant Human Erythropoietin Treatment
The purpose of this study is to investigate the relationship of anti-erythropoietin antibodies to the clinical course and outcome of pure red cell aplasia (PRCA) in participants currently or previously treated with recombinant…
- Trial name
- NCT00211042
- Testing
- No intervention
- Where
- 21 sites in Brazil, Canada and 6 more
- Volunteers
- 52 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
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
Sever Aplastic Anaemia
A Bioequivalence Study of Hetrombopag in Healthy Subjects
This study consists of two parts. Part 1 is a pilot BE study, and Part 2 is a pivotal study to demonstrate the bioequivalence of test and reference formulation, both of which adopt a single-center, randomized, open-label…
- Trial name
- NCT05088655
- Testing
- Hetrombopag Olamine Tablet
- Where
- China
- Volunteers
- 58 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
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
Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria (PNH), Level of CD59 on Red and White Blood Cells in Bone Marrow Failure Syndromes
Examine red and white blood cells of PNH patients with bone marrow failure syndromes.
- Trial name
- EXPLORE (NCT01192425)
- Volunteers
- 5,580 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Cytopaenia
A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/microliter who were refractory…
- Trial name
- NCT02148133
- Testing
- Eltrombopag 12.5 mg, Eltrombopag 25 mg
- Where
- 12 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
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
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
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.
Treatment-naïve Severe Aplastic Anemia
A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic Anemia
This is a randomized, double-blind, multicenter trial designed to evaluate treatment with romiplostim N01+ IST compared with placebo + IST in the participants with treatment-naïve severe aplastic anemia.
- Trial name
- NCT07345000
- Testing
- Romiplostim N01, Cyclosporine A (CsA), pALG/ rATG
- Volunteers
- 210 people
- Sponsor
- Qilu Pharmaceutical Co., Ltd.
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.
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.
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.
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