Status:
WITHDRAWN
Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents
Lead Sponsor:
National Human Genome Research Institute (NHGRI)
Conditions:
ADA-SCID
Eligibility:
All Genders
5-50 years
Brief Summary
Background: * Severe combined immunodeficiency (SCID) is a rare inherited disorder in which certain white blood cells have impaired function and are unable to properly fight infections. SCID typicall...
Detailed Description
The objectives of this study are to measure quality of life, neuropsychological sequelae and parental stress before and after gene therapy for ADA-SCID. The population to be studied will include up to...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Patients:
- Patients must be enrolled on protocol 01-HG-0189 in order to be eligible for enrollment on this protocol.
- Written informed consent from adult patients, or from the parents or guardians of minor patients must be obtained. Assent must be obtained from minor children when applicable.
- Patients must be English-speaking. Not all of the study instruments have been validated in other languages, and personnel are not available with training to administer the instruments in other languages.
- Parents or Guardians
- Parents must have a child enrolled on protocol 01-HG-0189 in order to be eligible for enrollment on this protocol. Guardians must have a ward enrolled on protocol 01-HG-0189 in order to be eligible for enrollment on this protocol.
- Written informed consent must be obtained from parents or guardians.
- EXCLUSION CRITERIA:
- Inability to complete the study instruments. This includes inability to speak English. Not all of the study instruments have been validated in other languages, and personnel are not available to administer the test instruments in other languages.
- Judgment of the clinical investigators that participation would be detrimental to the patient, parent or guardian.
- Judgment of the clinical investigators that participation would be detrimental to the study.
Exclusion
Key Trial Info
Start Date :
August 5 2010
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
September 25 2014
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT01182857
Start Date
August 5 2010
End Date
September 25 2014
Last Update
December 17 2019
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