Clinical trials: Preleukemia
Paroxysmal Nocturnal Hemoglobinuria
Specified Drug-use Surveillance of Fabhalta Capsules
This is a multicenter, single-arm, non-interventional study (NIS) with a central registration system and an all-case surveillance system. The observation period is 48 weeks after the start of treatment with Fabhalta.
- Trial name
- NCT06606314
- Where
- 91 sites in Japan
- Volunteers
- 132 people
- Sponsor
- Novartis 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
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
A study designed to evaluate the safety of crovalimab with eculizumab in participants with PNH currently treated with complement inhibitors. This study will enroll approximately 190 participants.
- Trial name
- COMMODORE 1 (NCT04432584)
- Testing
- Crovalimab, Eculizumab
- Where
- 80 sites in United States, Belgium and 22 more
- Volunteers
- 190 people
- Sponsor
- Hoffmann-La Roche
Life Threatening Diseases
Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
Exploratory study to examine the effect(s) of Imatinib mesylate treatment on life threatening rare diseases with known associations to one or more Imatinib mesylate -sensitive tyrosine kinases, and to identify the contribution of…
- Trial name
- NCT00154388
- Testing
- Imatinib mesylate
- Volunteers
- 185 people
- Sponsor
- Novartis 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.
Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML)
Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies
Pilot Study Evaluating the Safety and Efficacy of a Co-Transplantation of NiCord®, a UCB-derived ex Vivo Expanded Population of Stem and Progenitor Cells with a Second, Unmanipulated CBU in Patients with Hematological Malignancies
- Conditions
- Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Non-Hodgkin's Lymphoma
- Trial name
- NCT01221857
- Testing
- NiCord®
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Gamida Cell ltd
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.
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
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.
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.
Paroxysmal Nocturnal Hemoglobinuria
E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
The objective of this study was to assess the long-term safety and efficacy of eculizumab in hemolytic PNH patients who completed the 4-week screening and 12-week treatment period of the C07-001 study.
- Trial name
- NCT01194804
- Testing
- Eculizumab
- Volunteers
- 27 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Myelodysplastic Syndromes
AVIDA The Vidaza® (Azacitidine) Patient Registry
This registry is a prospective, multi-center, observational study compiling data on the natural history and management of patients receiving Vidaza, and will provide unique insights into the management of myelodysplastic…
- Trial name
- AVIDA (NCT00481273)
- Testing
- Azacitidine
- Where
- United States
- Volunteers
- 479 people
- Sponsor
- Celgene
Leukemia, Lymphoma
Safety Study of Human Myeloid Progenitor Cells (CLT-008) After Cord Blood Transplant for Hematologic Malignancy
Ex vivo expanded human myeloid progenitor cells (hMPCs; CLT-008) have the potential to accelerate neutrophil recovery in patients receiving myeloablative conditioning as part of an umbilical cord blood transplant for hematologic…
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma, Plasma Cell Neoplasm
- Trial name
- NCT00891137
- Testing
- human myeloid progenitor cells
- Where
- 8 sites in United States
- Volunteers
- 30 people
- Sponsor
- Cellerant Therapeutics
Paroxysmal Nocturnal Hemoglobinuria, PNH
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- Trial name
- NCT06449001
- Testing
- Danicopan
- Where
- 4 sites in Canada, France and 1 more
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Leukaemia
First in Human Testing of Dose-escalation of SAR440234 in Patients With Acute Myeloid Leukemia, Acute Lymphoid Leukemia and Myelodysplastic Syndrome
Primary Objective: Dose escalation: To determine the maximum tolerated dose (MTD) of SAR440234 administered as a single agent in participants with relapsed or refractory acute myeloid leukemia (R/R AML), high risk myelodysplastic…
- Trial name
- NCT03594955
- Testing
- SAR440234
- Where
- 4 sites in United States, France
- Volunteers
- 7 people
- Sponsor
- Sanofi
Advanced MDS, Acute Myeloid Leukemia
A Study of ARRY-520 in Patients With Advanced Myeloid Leukemia
This is a 2-phase study during which patients with select myeloid leukemias or advanced myelodysplastic syndrome (MDS), who have failed, refused or are not eligible for standard treatment, will receive investigational study drug…
- Trial name
- NCT00637052
- Testing
- ARRY-520, KSP(Eg5) inhibitor; intravenous
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Pfizer
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
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 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.
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.