Clinical trials: Hematologic Disease and Disorders
Hemophilia A
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants.
- Trial name
- PROTECT-ALT (NCT06684314)
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Taiwan
- Volunteers
- 79 people
- Sponsor
- Sanofi
B-precursor Acute Lymphoblastic Leukemia, ALL
GNR-084 Safety and Pharmacological Characteristics in Refractory or Relapse B-cell Precursor ALL
It is an open-label dose-escalating study in sequential cohorts to assess safety and pharmacokinetics of GNR-084.
- Conditions
- B-precursor Acute Lymphoblastic Leukemia, ALL, GNR-084
- Trial name
- NCT04601584
- Testing
- Cohort 1, GNR-084, Cohort 2, GNR-084, Cohort 3, GNR-084
- Where
- 3 sites in Russia
- Volunteers
- 14 people
- Sponsor
- AO GENERIUM
Immune Thrombocytopenic Purpura (ITP)
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a low number of platelets, making it easier to bruise or bleed.
- Trial name
- NCT06722235
- Testing
- Mezagitamab
- Where
- 124 sites in United States, Australia and 16 more
- Volunteers
- 171 people
- Sponsor
- Takeda
Metabolic Disorders, Hematologic, Immune, or Bone Marrow Disorders
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells.
- Conditions
- Metabolic Disorders, Hematologic, Immune, or Bone Marrow Disorders, Hemoglobinopathies, Non-malignant Disorders
- Trial name
- NCT00920972
- Testing
- Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4, GVHD Regimen A: UCB Recipients
- Where
- 28 sites in United States, Canada
- Volunteers
- 220 people
- Sponsor
- Washington University School of Medicine
Advanced Systemic Mastocytosis (AdvSM), SM With an Associated Hematologic Neoplasm (SM-AHN)
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm…
- Conditions
- Advanced Systemic Mastocytosis (AdvSM), SM With an Associated Hematologic Neoplasm (SM-AHN), Mast Cell Leukemia (MCL), Aggressive Systemic Mastocytosis (ASM)
- Trial name
- NCT04996875
- Testing
- bezuclastinib
- Where
- 42 sites in United States, Australia and 12 more
- Volunteers
- 140 people
- Sponsor
- Cogent Biosciences, Inc.
Immune Thrombocytopenic Purpura (ITP)
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a lower number of platelets, making it easier to bruise or bleed.
- Trial name
- NCT06948318
- Testing
- Mezagitamab
- Where
- 118 sites in United States, Australia and 13 more
- Volunteers
- 150 people
- Sponsor
- Takeda
Hematologic Diseases, Hematopoietic Stem Cell Transplantation
Immersive Virtual Reality Applied to Therapeutic Physical Exercise in Cellular Therapy
Cell therapy (CT), including hematopoietic stem cell transplantation (HSCT) and advanced CAR T-cell therapies, is used for the treatment of oncohematological and other diseases.
- Conditions
- Hematologic Diseases, Hematopoietic Stem Cell Transplantation, Cellular Therapy, Quality of Life
- Trial name
- RVI-ET (NCT07679958)
- Testing
- Intervention with IVR, Control
- Where
- Spain
- Volunteers
- 144 people
- Sponsor
- Fundacin Biomedica Galicia Sur
Multiple Myeloma in Relapse, Myeloma Multiple
A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma
The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractory multiple myeloma.
- Conditions
- Multiple Myeloma in Relapse, Myeloma Multiple, Multiple Myeloma Progression, Neoplasms by Histologic Type
- Trial name
- inMMyCAR (NCT07075185)
- Testing
- KLN-1010
- Where
- 11 sites in United States, Australia
- Volunteers
- 70 people
- Sponsor
- Kelonia Therapeutics, Inc.
Hematologic Disease and Disorders
Reconstruction Kinetics of γδ T Cells After Unrelated Cord Blood Transplantation
The primary objective is to investigate the correlation between specific patterns of γδ T cell reconstitution after umbilical cord blood transplantation and key clinical outcomes.
- Trial name
- NCT07782684
- Testing
- Umbilical Cord Blood Transplantation as Treatment of Adult Patients with Hematologic Disorders
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Anhui Provincial Hospital
Bone Marrow Aspiration, Pain
Visual and Auditory Distraction for the Relief of Pain and Anxiety
Bone marrow (BM) examinations are performed in diagnostic workup of malignancies, in particular hematological diseases, and are frequently associated with significant pain, distress, and anxiety.
- Conditions
- Bone Marrow Aspiration, Pain, Anxiety, Hematologic Diseases
- Trial name
- NCT07023692
- Testing
- Distraction with VR (Virtual Reality) headsets
- Where
- Sweden
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Region Skane
Hematologic Disorder, Bleeding Disorder
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly.
- Conditions
- Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia
- Trial name
- NCT04398628
- Where
- 76 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- American Thrombosis and Hemostasis Network
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation With Reduced Dose Post Transplantation Cyclophosphamide GvHD Prophylaxis
The goal of this clinical trial is to determine the effectiveness of Reduced Dose Post-Transplant Cyclophosphamide (PTCy) in patients with hematologic malignancies after receiving an HLA-Mismatched Unrelated Donor (MMUD) .
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Leukemia, Myelodysplastic Syndromes
- Trial name
- OPTIMIZE (NCT06001385)
- Testing
- Busulfan, Fludarabine, PBSC Hematopoietic Stem Cell Transplantation (HSCT)
- Where
- 32 sites in United States
- Volunteers
- 313 people
- Sponsor
- Center for International Blood and Marrow Transplant Research
Myelodysplastic Syndromes, MDS
Pacritinib With Aza for Upfront Myelodysplastic Syndrome
This study will be conducted as a phase 1/2 study of safety and preliminary efficacy of pacritinib in combination with azacitidine for IPSS-M moderate low to very high risk MDS.
- Conditions
- Myelodysplastic Syndromes, MDS, Myelodysplastic Syndrome, Unclassifiable, Hematologic Diseases
- Trial name
- MDS (NCT07387354)
- Testing
- Pacritinib, Azacitidine, Bone Marrow Biopsy and Aspirate
- Sponsor
- Thomas Jefferson University
Haematological Malignancy, Malignant Neoplasm
DETERMINE Trial Treatment Arm 07: Dabrafenib in Combination With Trametinib in Adult, Paediatric and Teenage/Young Adult Patients With BRAF V600 Mutation-Positive Cancers.
This clinical trial is looking at two drugs called dabrafenib and trametinib. Dabrafenib and trametinib are approved as standard of care treatment for adult patients with melanoma (a type of skin cancer) or lung cancer and in…
- Conditions
- Haematological Malignancy, Malignant Neoplasm, Lymphoproliferative Disorders, Neoplasms by Histologic Type
- Trial name
- DETERMINE (NCT07440290)
- Testing
- Dabrafenib, Trametinib
- Where
- 27 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Cancer Research UK
Hematopoietic and Lymphoid Cell Neoplasm, High Risk Acute Myeloid Leukemia
Busulfan, Fludarabine Phosphate, and Post-Transplant Cyclophosphamide in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant
This phase II trial studies the side effect of busulfan, fludarabine phosphate, and post-transplant cyclophosphamide in treating patients with blood cancer undergoing donor stem cell transplant.
- Conditions
- Hematopoietic and Lymphoid Cell Neoplasm, High Risk Acute Myeloid Leukemia, High Risk Myelodysplastic Syndrome, Lymphoproliferative Disorder
- Trial name
- NCT02861417
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Busulfan, Cyclophosphamide
- Where
- United States
- Volunteers
- 204 people
- Sponsor
- M.D. Anderson Cancer Center
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
CD-30 Positive Anaplastic Large T-cell Cutaneous Lymphoma, Lymphoma, Primary Cutaneous Anaplastic Large Cell
SGN-35 in CD30-positive Lymphoproliferative Disorders (ALCL), Mycosis Fungoides (MF), and Extensive Lymphomatoid Papulosis (LyP)
The goal of this clinical research study is to learn if SGN-35 (brentuximab vedotin) can help to control ALCL, LyP or MF in patients with at least 1 of the 3 skin lymphomas. The safety of the study drug will also be studied.
- Conditions
- CD-30 Positive Anaplastic Large T-cell Cutaneous Lymphoma, Lymphoma, Primary Cutaneous Anaplastic Large Cell, Lymphomatoid Papulosis, Mycosis Fungoides
- Trial name
- NCT01352520
- Testing
- SGN-35
- Where
- United States
- Volunteers
- 79 people
- Sponsor
- M.D. Anderson Cancer Center
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII.
- Trial name
- GENEr8-INH (NCT04684940)
- Testing
- Valoctocogene roxaparvovec
- Where
- 9 sites in United States, Brazil and 5 more
- Volunteers
- 10 people
- Sponsor
- BioMarin Pharmaceutical
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
Trial of Orca-T Following Reduced Intensity or Nonmyeloablative Conditioning in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
This study will evaluate the safety, tolerability, and efficacy of Orca-T in participants undergoing reduced intensity or non-myeloablative allogeneic hematopoietic cell transplantation (alloHCT) for hematologic malignancies.
- Conditions
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Mixed Phenotype Acute Leukemia
- Trial name
- NCT07216443
- Testing
- Orca-T
- Where
- 6 sites in United States
- Volunteers
- 80 people
- Sponsor
- Orca Biosystems, Inc.
Hematologic Malignancies, Inherited Disorders of Metabolism
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.
- Conditions
- Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders
- Trial name
- NCT01351545
- Testing
- A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
- Where
- 142 sites in United States
- Volunteers
- 99,999 people
- Sponsor
- Center for International Blood and Marrow Transplant Research