Header

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:

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

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:

Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector

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:

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:

A Study of Pembrolizumab (MK-3475) in Pediatric Participants With Advanced Melanoma or Advanced, Relapsed, or Refractory PD-L1-Positive Solid Tumors or Lymphoma (MK-3475-051/KEYNOTE-051)

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:

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:

Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement

This pilot phase II trial studies the side effects of azacitidine and combination chemotherapy in infants with acute lymphoblastic leukemia and KMT2A gene rearrangement. Drugs used in chemotherapy,...

Diagnosis: Pediatric Leukemia, Pediatric Hematology/Blood Related

Phase:

Protocol Number: 17-714

'