Pediatric Hematology/Blood Related Clinical Trials
Category:Pediatric
Status:Active
Displaying all 20 trials
Human-cl-rhFVIII in Previously Untreated Patients
Investigate the inhibitor development rate of Human cl rhFVIII in previously untreated patients with severe Hemophilia A.
Diagnosis: Pediatric Hematology/Blood Related
Phase: 3
Protocol Number:
Evaluation of the Duration of Therapy for Thrombosis in Children
The Kids-DOTT trial is a randomized controlled clinical trial whose primary objective is to evaluate non-inferiority of shortened-duration (6 weeks) versus conventional-duration (3 months) anticoag...
Diagnosis: Pediatric Hematology/Blood Related
Phase: 3
Protocol Number:
This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis n...
Diagnosis: Pediatric Hematology/Blood Related
Phase: 3
Protocol Number:
A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors
In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk vascular...
Diagnosis: Pediatric Hematology/Blood Related
Phase: 2
Protocol Number:
The Wiskott-Aldrich Syndrome (WAS) is an inherited disorder that results in defects of the blood and bone marrow. It affects boys because the genetic mistake is carried on the X chromosome. Normal ...
Diagnosis: Pediatric Hematology/Blood Related, Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase: 1 / 2
Protocol Number:
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents a...
Diagnosis: Pediatric Hematology/Blood Related, Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase: 1 / 2
Protocol Number:
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disorder which results from defects that prevent white blood cells from effectively killing bacteria, fungi and other microorgan...
Diagnosis: Pediatric Hematopoietic Stem Cell Transplant (HSCT), Pediatric Hematology/Blood Related
Phase: 1 / 2
Protocol Number:
Pyruvate Kinase Deficiency Natural History Study
The purpose of this study is to describe the range and incidence of symptoms, treatments, and complications related to pyruvate kinase deficiency (PKD). Eligible patients are those of all ages with...
Diagnosis: Pediatric Hematology/Blood Related
Phase: N/A
Protocol Number:
This trial is conducted globally. The aim of the trial is to evaluate the safety and efficacy, including pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) ...
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions. The leucine...
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
Web-based Application for the Population Pharmacokinetic Service - Phase 1
The aims of this trials are: 1. to collect published and unpublished individual classic pharmacokinetic data (individual patient data from independent investigators and pharmaceutical companies) 2....
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
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.
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
This is the first evaluation of edoxaban in pediatric subjects. In this Phase 1 study, a single dose of edoxaban will be given to pediatric subjects who require anticoagulant therapy to see what th...
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
A Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency
Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 in patients with PK deficiency.
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
Apixaban for the Acute Treatment of Venous Thromboembolism in Children
To assess the safety and extrapolated efficacy of apixaban in pediatric subjects requiring anticoagulation for the treatment of a VTE.
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
This is a 2-part study of pembrolizumab (MK-3475) in pediatric participants who have either advanced melanoma or a programmed cell death ligand 1 (PD-L1)-positive advanced, relapsed or refractory s...
Diagnosis: Pediatric Solid Tumors, Pediatric Hematology/Blood Related
Phase:
Protocol Number: 16-235
Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia
The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic s...
Diagnosis: Pediatric Hematology/Blood Related, Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase:
Protocol Number:
Crizotinib (Xalkori) Expanded Access Protocol For The Treatment Of Adult Or Pediatric Patients
This is an open label expanded access protocol for the treatment of up to approximately 40 adult or pediatric (defined as age <18 years) patients with tumors harboring either a chromosomal trans...
Diagnosis: Pediatric Hematology/Blood Related, Pediatric Solid Tumors, Solid Tumor/Phase I
Phase:
Protocol Number: 16-555
Dose-Finding Study of SC411 in Children With Sickle Cell Disease
This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to eva...
Diagnosis: Pediatric Hematology/Blood Related
Phase:
Protocol Number:
