Clinical trials: Disease of the Marrow
- Condition Disease of the Marrow
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
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
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
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.
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.
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
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.
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acute Myeloid Leukemia, Accelerated Phase Chronic Myeloid Leukemia
Safety and Efficacy of Ponatinib for Treatment of Pediatric Recurrent or Refractory Leukemias, Lymphomas or Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of ponatinib in children aged 1 to < 18 years with advanced leukemias, lymphomas, and solid tumors.
- Conditions
- Acute Myeloid Leukemia, Accelerated Phase Chronic Myeloid Leukemia, Blast Phase Chronic Myeloid Leukemia, Chronic Phase Chronic Myeloid Leukemia
- Trial name
- NCT03934372
- Testing
- Ponatinib
- Where
- 23 sites in Belgium, France and 5 more
- Volunteers
- 70 people
- Sponsor
- Incyte Biosciences International Sàrl
Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal…
- Trial name
- NCT04901936
- Testing
- Pegcetacoplan
- Where
- 12 sites in United States, Czechia and 7 more
- Volunteers
- 12 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Myeloid Leukemia, Philadelphia Positive
Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
The aim of this study is to support development of asciminib in the pediatric population (1 to <18 years) previously treated with one or more TKIs.
- Trial name
- NCT04925479
- Testing
- Asciminib Pediatric formulation group, Asciminib Adult formulation group
- Where
- 39 sites in United States, China and 12 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Fever, Sweats, and Hot Flashes, Infection
Recombinant Human Mannose-Binding Lectin (MBL) in Treating Young Patients With MBL Deficiency and Fever and Neutropenia
RATIONALE: Recombinant human mannose-binding lectin (MBL) may be effective in preventing infection in young patients with fever and neutropenia receiving chemotherapy for blood disease or cancer.
- Conditions
- Fever, Sweats, and Hot Flashes, Infection, Leukemia, Lymphoma
- Trial name
- NCT00886496
- Testing
- recombinant human mannose-binding lectin
- Where
- 3 sites in United States
- Sponsor
- Enzon Pharmaceuticals, Inc.
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
Paroxysmal Nocturnal Hemoglobinuria
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
- Trial name
- NCT05389449
- Testing
- Danicopan
- Where
- 45 sites in United States, Brazil and 13 more
- Volunteers
- 80 people
- Sponsor
- Alexion Pharmaceuticals, 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.
Essential Thrombocythemia (ET)
Exploratory Multi-centre Trial In Patients With ET Treated With XAGRID®
This study is hypothesis-generating to explore the impact of JAK2 (V617F) mutation status on the treatment response to anagrelide hydrochloride
- Trial name
- EMIX (NCT01352585)
- Testing
- Anagrelide hydrochloride
- Where
- 10 sites in Italy
- Volunteers
- 47 people
- Sponsor
- Shire
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.
Advanced Hematologic Malignancy, Relapsed Acute Myeloid Leukemia
FHD-286 as Monotherapy or Combination Therapy in Subjects With Advanced Hematologic Malignancies
This Phase 1, multicenter, open-label, dose escalation study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-286 administered orally as…
- Conditions
- Advanced Hematologic Malignancy, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Relapsed Myelodysplastic Syndromes
- Trial name
- NCT04891757
- Testing
- FHD-286, Low Dose Cytarabine, Decitabine
- Where
- 5 sites in United States
- Volunteers
- 144 people
- Sponsor
- Foghorn Therapeutics Inc.
Polycythemia Vera
A Study to Better Understand the Diagnosis and Treatment of Polycythaemia Vera
PV is a rare, long-term blood cancer that causes the body to make too many blood cells. This can make the blood thicker than normal and the risk of blood clots higher.
- Trial name
- NCT07868081
- Testing
- No Intervention
- Volunteers
- 90 people
- Sponsor
- Takeda
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
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