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Pediatric Hematology/Blood Related Clinical Trials

Category:Pediatric
Status:Active

Displaying all 22 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:

Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease

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:

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:

Evaluation of PET/MRI in Children With Cancer

This research study is a Pilot study (a small preliminary study to assess the feasibility of a larger, more in depth study involving a new test or procedure) and is being done to evaluate the feasi...

Diagnosis: Pediatric Hematology/Blood Related

Phase: N/A

Protocol Number: 12-307

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

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:

A Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

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:

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.

Diagnosis: Pediatric Hematology/Blood Related

Phase:

Protocol Number:

Phase 1 Pediatric PK/PD Study

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:

Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

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:

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:

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:

A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia

This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in combination with immunosuppressive...

Diagnosis: Pediatric Hematology/Blood Related

Phase:

Protocol Number:

Gene Transfer for Sickle Cell Disease

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to tre...

Diagnosis: Pediatric Hematology/Blood Related

Phase:

Protocol Number:

Pilot Study of Metformin for Patients With Fanconi Anemia

This is a single institution, open-label, single arm pilot study of Metformin in patients with Fanconi Anemia (FA) and cytopenias with the primary endpoint of hematologic response. This study will ...

Diagnosis: Pediatric Hematology/Blood Related

Phase:

Protocol Number:

A Gene Transfer Study for Hemophilia A

This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophi...

Diagnosis: Pediatric Hematology/Blood Related

Phase:

Protocol Number:

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back or does not respond to treatment. Trametinib may stop the growth ...

Diagnosis: Pediatric Leukemia, Pediatric Hematology/Blood Related

Phase:

Protocol Number: 18-712

Study of ARQ 092 in Patients With Overgrowth Diseases and/or Vascular Anomalies

This is an open label, Phase 1/2 study of oral ARQ 092 administered to patients at least 2 years of age with overgrowth diseases and/or vascular anomalies with genetic alterations of the PI3K/AKT p...

Diagnosis: Pediatric Hematology/Blood Related

Phase:

Protocol Number:

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