Genetic Syndromes Clinical Trials

Full title:

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

Study goal:

To find out how much patients with hemophilia B, their caregivers, and health care workers know about gene therapy for this disease, and how they feel about gene therapy. This will help us better educate patients and caregivers about  gene therapy for hemophilia B.

Diagnosis:

Hemophilia B

Age:

12 years and older

Study goal:

The main goal of this study is to learn more about the reasons childhood tumors form and how to treat them better.

Diagnosis:

Leukemia, lymphoma, genetics and genomics, solid tumor

Study goal:

To learn more about participants beliefs, attitudes, and questions about gene therapy to help make web-based resources to share information. This could help patients make treatment decisions.

Diagnosis:

Neurological disorders, sickle cell disease, hemophilia, inherited genetic diseases

Age:

18 to 35 years old with rare genetic diseases

Full title:

PG4KDS: Clinical Implementation of Pharmacogenetics

Study goal:

1) Test each patient for hundreds of gene variations that might be important for drug use. All of the gene test results will be in the research laboratory, but as time goes on, the study will evaluate scientific evidence and selectively move test results for a few genes into the medical record if the evidence strongly shows that the result can help in better prescribing of drugs for patients; 2) Estimate how often pharmacogenetic test results are moved from research tests into a patient’s medical record; 3) Use methods to choose which of these tests should be put in the medical record; 4) Use computer-based tools in the electronic medical record to help doctors use gene test results when prescribing drugs; 5) Share feelings and concerns of patients and their families about gene test information being put in their medical record.

Full title:

Familial Investigations of Childhood Cancer Predisposition

Study goal:

The main purpose of this trial is to learn about the genetic causes of cancer. 

Diagnosis:

Solid tumors, brain tumors, leukemia

Age:

Younger than 50 years old

Full title:

TBANK: Protocol for Collecting, Banking, and Distributing Human Tissue Samples: St. Jude Children’s Research Hospital Biorepository

Study goal:

To provide a high-quality repository of tumor and normal samples to facilitate translational research performed by St Jude faculty and their collaborators

Diagnosis:

All diseases

Full title:

Friedreich’s Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI)

Study goal:

To collect data about how Friedreich’s ataxia progresses over time and affects the daily life of patients.

Diagnosis:

Friedreich’s ataxia

Age:

Any