Sickle Cell Disease Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

This study has several different groups. You may qualify for one of the following:

• Group 1 – Gene therapy received (for a rare genetic disease): You are a parent or caregiver of a child who has received gene therapy, OR you are a patient aged 8 or older who has received gene therapy, OR you are a parent or caregiver of a child who passed away between 6 and 24 months ago after receiving gene therapy (and at least 3 months have passed since the death).
• Group 2 – Gene therapy offered but not received (rare genetic disease): You are a parent, caregiver, or patient aged 8 or older with a rare genetic disease who was offered gene therapy but either chose not to have it or was not eligible for it.
• Group 3 – Healthcare providers: You are a healthcare worker who has cared for at least 2 patients who received gene therapy.
• Group 4 – Gene therapy received (for a bone marrow failure condition): Same as Group 1, but specifically for a bone marrow failure condition.
• Group 5 – Gene therapy offered but not received (bone marrow failure condition): You are a parent, caregiver, or patient aged 8 or older with a bone marrow failure condition who was offered gene therapy but either chose not to have it or was not eligible for it.
• Group 6 – Gene therapy never offered (bone marrow failure condition): You are a parent, caregiver, or patient aged 8 or older with a bone marrow failure condition who has never been offered gene therapy.
• Group 7 – Healthcare providers (bone marrow failure condition): You are a healthcare worker who has cared for at least 2 patients who received gene therapy for a bone marrow failure condition.
• All participants must be willing to take part in a one-on-one video interview (or in-person, for healthcare providers) using a phone or computer with internet access.
• All participants must be willing to give verbal consent to take part in the study.
• All participants must be willing to sign a form allowing the study team to contact their (or their child's) doctor to confirm their diagnosis and treatment details.
• The diagnosis of a rare genetic disease must be confirmed by a healthcare provider.

Who may not be able to join:

• People who are not able to hold a conversation fluently in English.
• People who are unwilling or unable to give verbal consent to participate.
• People who do not have access to a phone or computer that supports video calls.
• People whose health condition — in the view of the study doctors — makes participation unsafe or not practical, such as significant cognitive impairment or a serious current illness (confirm with trial site).

Important: Always verify eligibility with the trial site directly before applying.