Studying Patient Views on Gene Therapy for Blood Disorders

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults with transfusion dependent beta-thalassemia or sickle cell disease, or parents of children with these diseases, who could receive gene therapy.

Age

18 years or older. Parents of children under 18 can also join.

Sex

All sexes can join.

Disease stage

Transfusion dependent beta-thalassemia or sickle cell disease, for people who could receive gene therapy for their disease.

Main reasons for not taking part

• People who do not speak English cannot join.

The registry criteria, word for word

What the trial wants to find out

Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness, and to assess the likely impact of gene therapy on patients' quality of life.

The group in the trial

• Group 1

  Patients or parents of pediatric patients diagnosed with beta-thalassemia or sickle cell disease and eligible to receive treatment for their disease with gene therapy.