Clinical trials: Bone Marrow Disease
- Condition Bone Marrow Disease
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Chronic Myelogenous Leukemia
Phase II Trial of Decitabine in Patients With Chronic Myelogenous Leukemia Accelerated Phase Who Are Refractory to Imatinib Mesylate (Gleevec)
To determine the safety and efficacy of decitabine in patients with Philadelphia chromosome-positive chronic myelogenous leukemia accelerated phase that were previously treated with imatinib mesylate (STI 571) and became…
- Trial name
- NCT00041990
- Testing
- decitabine (5-aza-2'deoxycytidine)
- Where
- 8 sites in United States, Canada
- Volunteers
- 40 people
- Sponsor
- Astex Pharmaceuticals, Inc.
Myelodysplastic Syndrome, Leukemia, Myelomonocytic, Juvenile
Study With Azacitidine in Pediatric Subjects With Newly Diagnosed Advanced Myelodysplastic Syndrome (MDS) and Juvenile Myelomonocytic Leukemia (JMML)
Indication Treatment of pediatric subjects with newly diagnosed advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) prior to hematopoietic stem cell transplantation (HSCT).
- Trial name
- NCT02447666
- Testing
- Azacitidine
- Where
- 39 sites in Austria, Belgium and 11 more
- Volunteers
- 28 people
- Sponsor
- Celgene
Acute Leukemia, Myelodysplastic Syndrome
Study of QUAIL-100 in Pediatric and Young Adult Participants With High-Risk Hematologic Malignancies Who Have Received a Hematopoietic Stem Cell Transplantation
Study of QUAIL-100 in Patients With High Risk Acute Leukemia and Myelodysplastic Syndrome Who Have Received Hematopoietic Stem Cell Transplant
- Trial name
- NCT07573111
- Testing
- QUAIL-100
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Laguna Biotherapeutics, Inc.
Myelodysplastic Syndromes, Beta-thalassemia
Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL.
- Trial name
- NCT04973280
- Where
- 4 sites in Austria, Belgium and 2 more
- Volunteers
- 187 people, healthy volunteers welcome
- Sponsor
- Celgene
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Chronic Myeloproliferative Disorders, Infection
Antifungal Therapy for Fever and Neutropenia in Patients Receiving Treatment for Hematologic Cancer
RATIONALE: Antifungal therapy with liposomal nystatin may reduce fever and neutropenia in patients undergoing treatment for hematologic cancer.
- Conditions
- Chronic Myeloproliferative Disorders, Infection, Leukemia, Lymphoma
- Trial name
- NCT00002742
- Testing
- amphotericin B deoxycholate, nystatin
- Where
- 59 sites in United States
- Sponsor
- Aronex Pharmaceuticals
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.
Chronic Iron Overload
Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation
Extend evaluation of deferasirox film-coated tablet (FCT) formulation
- Trial name
- NCT02720536
- Testing
- Deferasirox
- Where
- 14 sites in Austria, Greece and 1 more
- Volunteers
- 53 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Myelogenous Leukemia
Randomized Phase Lll Study of Imatinib Dose Optimization vs Nilotinib in CML Patients With Suboptimal Response to Imatinib
There is no available data on the clinical benefit of dose escalation for patients with suboptimal response to imatinib, and patients may still improve their response with continuation of therapy at the standard dose as shown in…
- Trial name
- LASOR (NCT00802841)
- Testing
- nilotinib, imatinib
- Where
- 56 sites in Argentina, Brazil and 10 more
- Volunteers
- 191 people
- Sponsor
- Novartis Pharmaceuticals
Acute Myeloid Leukemia, Myelodysplastic Syndromes
Study of Haplo-HSCT + Rivogenlecleucel vs Haplo-HSCT + Post Transplant Cyclophosphamide in Patients With AML or MDS
This study compares the safety and effectiveness of giving rivogenlecleucel (BPX-501 T cells) to patients with AML or MDS post haploidentical hematopoietic stem cell transplant compared to post-transplant cyclophosphamide.
- Trial name
- THRIVE (NCT03699475)
- Testing
- rivogenlecleucel, rimiducid, Cyclophosphamide
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
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.
Essential Thrombocythemia (ET)
Pediatric Disease Registry in Essential Thrombocythaemia (ET)
The aim of this study is to evaluate the progression of ET in children (aged 6-17years inclusive) over a period of 5 years maximum.
- Trial name
- NCT01198717
- Where
- 32 sites in Czechia, France and 6 more
- Volunteers
- 60 people
- Sponsor
- Shire
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Thrombocythemia, Essential
Safety and Pregnancy Outcomes in Thrombocythemia Patients Exposed to XAGRID® (Anagrelide Hydrochloride) Compared to Other Treatments
This is an observational safety study being conducted in Europe comparing patients taking Xagrid to patients taking other cytoreductive treatments.
- Trial name
- NCT00567502
- Where
- 164 sites in Cyprus, Denmark and 12 more
- Volunteers
- 3,647 people
- Sponsor
- Shire
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
Paroxysmal Nocturnal Hemoglobinuria, PNH
Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study
How does long term treatment with Soliris affect HAHA in PNH patients?
- Trial name
- NCT01412047
- Where
- 14 sites in United States, Australia and 8 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Leukemia, Lymphoblastic, Acute, T-cell, Myelogenous Leukemia
A Notch Signalling Pathway Inhibitor for Patients With T-cell Acute Lymphoblastic Leukemia/Lymphoma (ALL)(0752-013)
A Notch signalling pathway inhibitor study in pediatric and adult patients with relapsed (worsening) or refractory (not responding to treatment) T-cell acute lymphoblastic leukemia/lymphoma (T-ALL).
- Conditions
- Leukemia, Lymphoblastic, Acute, T-cell, Myelogenous Leukemia, Chronic Lymphocytic Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00100152
- Testing
- MK0752, (Notch Inhibitor)
- Volunteers
- 50 people
- Sponsor
- Merck Sharp & Dohme LLC
Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory
First-in-human Study of SAR443579 Infusion in Male and Female Children and Adult Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
This is an open-label, multicenter, Phase 1/Phase 2, dose escalation and dose expansion study to evaluate the safety, pharmacokinetics, pharmacodynamics and anti-leukemic activity of SAR443579 in various hematological…
- Conditions
- Acute Lymphocytic Leukaemia, Acute Myeloid Leukaemia Refractory, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasia
- Trial name
- NCT05086315
- Testing
- SAR443579
- Where
- 23 sites in United States, Australia and 3 more
- Volunteers
- 101 people
- Sponsor
- Sanofi
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
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals