Clinical trials: Newborn; Anemia
Leukemia, Myelodysplastic Syndromes
3-AP and Cytarabine in Treating Patients With Hematologic Cancer
RATIONALE: Drugs used in chemotherapy such as cytarabine use different ways to stop cancer cells from dividing so they stop growing or die.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00064090
- Testing
- cytarabine, triapine
- Where
- United States
- Sponsor
- Vion Pharmaceuticals
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Sickle Cell Disease
Evaluation of Spectra Optia Red Blood Cell Exchange in Sickle Cell Patients
The purpose of this study is to evaluate the performance of the Spectra Optia system red blood cell exchange (RBCx) protocols (exchange and depletion/exchange) in study participants with sickle cell disease.
- Trial name
- ESSENTIAL (NCT01736657)
- Testing
- Red blood cell exchange in sickle cell
- Where
- 5 sites in United States
- Volunteers
- 73 people
- Sponsor
- Terumo BCT
Sickle Cell Disease
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult…
- Trial name
- SPARKLE (NCT06439082)
- Testing
- Crizanlizumab
- Where
- 36 sites in United States, Brazil and 3 more
- Volunteers
- 354 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Sickle Cell Anemia
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir
This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Pociredir in participants with sickle cell disease.
- Trial name
- NCT05169580
- Testing
- Pociredir oral capsule(s)
- Where
- 18 sites in United States, Nigeria and 1 more
- Volunteers
- 45 people
- Sponsor
- Fulcrum Therapeutics
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
Anemia, Sickle Cell
Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease
The purpose of the study is to determine the maximum tolerated dose, safety and effect on induction of fetal hemoglobin of pomalidomide in patients with Sickle Cell Disease.
- Trial name
- SCD-001 (NCT01522547)
- Testing
- pomalidomide
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Celgene
Graft Versus Host Disease, Leukemia
OTI-010 for Graft-Versus-Host Disease Prophylaxis in Treating Patients Who Are Undergoing Donor Peripheral Stem Cell Transplantation for Hematologic Malignancies
RATIONALE: OTI-010 may be effective for graft-versus-host disease prophylaxis (prevention) in patients who are undergoing donor peripheral stem cell transplantation for hematologic malignancies (cancer of the blood or bone…
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00081055
- Testing
- autologous expanded mesenchymal stem cells OTI-010, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Mesoblast International Sàrl
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire
Myelodysplastic Syndrome, Beta Thalassemia
A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia
The purpose of this observational study is to assess the real-world safety of luspatercept in Korean participants with myelodysplastic syndrome (MDS) or beta thalassemia.
- Trial name
- NCT06073860
- Testing
- Luspatercept
- Where
- 2 sites in South Korea
- Volunteers
- 104 people
- Sponsor
- Bristol-Myers Squibb
Iron Overload, Beta-Thalassemia
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
- Trial name
- NCT02274233
- Testing
- SP-420
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 24 people
- Sponsor
- Sideris Pharmaceuticals
Anemia
Measurement of Total Hemoglobin by Pulse Oximetry in Neonatal Population
Assess ability of Masimo SpHb sensor to demonstrate robust performance on various sensor application sites on neonates/infants
- Trial name
- NCT03120702
- Testing
- Investigational Rainbow SpHb sensor
- Where
- United States
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- Masimo Corporation
Beta-Thalassemia
A Study of EPEG in Beta Thalassemia Patients
An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry
- Trial name
- NCT02950857
- Testing
- EPEG
- Where
- Thailand
- Volunteers
- 6 people
- Sponsor
- Prolong Pharmaceuticals
Sickle Cell Disease, Sickle Cell Anemia
Actigraphy Improvement With Voxelotor (ActIVe) Study
This is a study to evaluate the effect of voxelotor on daily physical activity and sleep quality, as measured by a wrist-worn device in participants with sickle cell disease (SCD) and chronic moderate anemia.
- Trial name
- ActIVe (NCT04400487)
- Testing
- Voxelotor
- Where
- 10 sites in United States
- Volunteers
- 25 people
- Sponsor
- Pfizer
Sickle Cell Disease
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
The purpose of the study is to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease
- Trial name
- HESTIA3 (NCT03615924)
- Testing
- Ticagrelor
- Where
- 55 sites in United States, Belgium and 14 more
- Volunteers
- 193 people
- Sponsor
- AstraZeneca
Cancer, Breakthrough Pain
Efficacy/Safety Study of ACTIQ® for Opioid-Tolerant Children and Adolescents With Breakthrough Pain (BTP)
The primary objective of the study is to evaluate the efficacy of ACTIQ treatment for the management of breakthrough pain (BTP) compared to placebo treatment in children with cancer and non-cancer pain who are receiving…
- Trial name
- NCT00236041
- Testing
- ACTIQ®
- Where
- 31 sites in United States, Puerto Rico
- Volunteers
- 38 people
- Sponsor
- Cephalon
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Sickle Cell Disease
An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia
This is an open-label extension study of IMR-687 in adult patients who completed Imara's blinded Phase 2a study (IMR-SCD-102). The open-label extension study will evaluate long-term safety and tolerability.
- Trial name
- NCT04053803
- Testing
- IMR-687
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 30 people
- Sponsor
- Imara, Inc.
Autoimmune Hemolytic Anemia
A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia
The purpose of this study is to evaluate the safety and efficacy of parsaclisib administered orally to participants with autoimmune hemolytic anemia (AIHA) who have decreased hemoglobin and evidence of ongoing hemolysis that…
- Trial name
- NCT03538041
- Testing
- Parsaclisib
- Where
- 12 sites in United States, Austria and 2 more
- Volunteers
- 25 people
- Sponsor
- Incyte Corporation