Clinical trials: Red Blood Cells
Thrombocytopenia, Metachromatic Leukodystrophy
Stem Cell Transplantation (SCT) for Genetic Diseases
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II.
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major
- Trial name
- NCT00004378
- Testing
- Stem Cell Transplantation
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Multiple Myeloma, Hematologic Diseases
Multiple Myeloma Prognostic Indices
The aim of this study was to evaluate the impact of albumin / fibrinogen ratio, blood viscosity and RDW on the prognosis of a newly diagnosed MM patients
- Trial name
- NCT06095752
- Testing
- blood viscosity , albumin / fibrinogen ratio and red cell distribution
- Where
- Egypt
- Volunteers
- 48 people
- Sponsor
- Assiut University
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period
Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving…
- Trial name
- ACTIVATE-Kids (NCT05175105)
- Testing
- Mitapivat
- Where
- 19 sites in United States, Canada and 5 more
- Volunteers
- 30 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Nervous System Disease, Genetic Syndrome
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
Objectives: The objective of study was to evaluate the safety and the efficacy of EryDex (Dexamethasone sodium phosphate encapsulated in autologous erythrocytes, using the EryDex System - EDS) at two dose levels (low dose and…
- Trial name
- ATTeST (NCT02770807)
- Testing
- EryDex Low dose DSP, EryDex High dose DSP
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 176 people
- Sponsor
- Quince Therapeutics S.p.A.
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Infant, Premature, Diseases
Enteral Supplementation With Docosahexaenoic Acid and Arachidonic Acid (DHA-AA) in Preterm Infants
Docosahexaenoic acid (DHA) and arachidonic acid (AA) have a critical effect on the health and neuronal development of the fetus and newborn.
- Trial name
- NCT06366893
- Testing
- DHA/AA emulsion supplement for preterm infant
- Where
- Spain
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Sickle Cell Disease, Thalassemia
Multi-Center Study of Iron Overload: Pilot Study
The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions…
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- MCSIO (NCT01114776)
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Nervous System Disorder, Genetic Syndrome
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX…
- Trial name
- IEDAT-01 (NCT01255358)
- Testing
- Dexamethasone
- Where
- 2 sites in Italy
- Volunteers
- 22 people
- Sponsor
- Quince Therapeutics S.p.A.
Red Blood Cell Transfusion
The Effect of Retrograde Autologous Priming on Transfusion Requirements After Cardiac Surgery
The TheRAPy vanguard trial is a multicentre, multiple period randomized, cluster crossover vanguard trial testing the feasibility of a full-scale trial to evaluate whether a centre-based policy of routine use of RAP versus a…
- Trial name
- TheRAPy (NCT06230198)
- Testing
- retrograde autologous priming (RAP), Crystalloid priming
- Where
- 4 sites in Canada
- Volunteers
- 4,500 people, healthy volunteers welcome
- Sponsor
- Population Health Research Institute
Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency Epidemiological Study (PIECE)
Pyruvate kinase deficiency (PKD) is the most common red cell glycolytic enzyme defect causing hereditary non-spherocytic hemolytic anemia, caused by mutations in the PKLR gene.
- Trial name
- PIECE (NCT03866590)
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- CENTOGENE GmbH Rostock