Last updated: 01/15/2019 18:10:09
This product has been transferred to Orchard Therapeutics Limited. GSK Clinical Study Register is no longer maintained for this study. The most up to date information is available on clinicaltrials.gov

Retroviral insertion site methodology study

GSK study ID
205813
Clinicaltrials.gov ID
NCT03311074
EudraCT ID
2017-001731-39
EU CT Number
Not applicable
Trial status
No longer a GSK study
No longer a GSK study
Overview
Eligibility
Locations
Study documents
Results summary
Plain language summaries
Additional information

Trial overview

Official title: Methodology study to investigate the utility of retroviral insertion site analysis in samples from subjects treated with Strimvelis™ gene therapy
Trial description: Adenosine deaminase (ADA) is an enzyme involved in the development and functioning of the immune system. Deficiency of ADA results in severe combined immunodeficiency (SCID), a fatal inherited immune disorder. GSK2696273 is a gene therapy that aims to insert ADA function into blood cells and halt or reverse the conditions caused by decreased ADA enzyme levels, such as impaired immune function. It is important to consider long term follow-up of patients who have received GSK2696273, including evaluation of the risk of insertion near certain genes that may lead to unexpected activation of those genes (oncogenesis). The objective of this study is to evaluate the use of a new technique to identify where GSK2696273 has become inserted in the genetic sequence, and potential implications for patient care. This new technique is known as sonication linker mediated polymerase chain reaction (SLiM-PCR) for retroviral insertion site (RIS) analysis. The study will recruit at least 15 pediatric or adult patients with ADA-SCID who have been treated with GSK2696273, either in previous clinical trials or as a registered product. Recruitment for the study may remain open for up to 2 years even if 15 subjects are recruited sooner. Study participation will last for up to 5 years. A total of 5 blood samples will be collected from each subject at approximately annual intervals. STRIMVELIS is a trade mark of the GSK group of companies.
Primary purpose:
Treatment
Trial design:
Single Group Assignment
Masking:
None (Open Label)
Allocation:
Not applicable
Primary outcomes:

Mean abundance measurement

Timeframe: Up to 5 years

Coefficient of variation measurement

Timeframe: Up to 5 years

Secondary outcomes:

Measurement of clone abundance of more than 5 percent

Timeframe: Up to 5 years

Shannon diversity index measurement

Timeframe: Up to 5 years

Interventions:
  • Biological/vaccine: GSK2696273
    • Enrollment:
    15
    Primary completion date:
    Not applicable
    Observational study model:
    Not applicable
    Time perspective:
    Not applicable
    Clinical publications:
    Not applicable
    Medical condition
    Immune System Diseases
    Product
    GSK2696273
    Collaborators
    Pharmaceutical Product Development (PPD)
    Study date(s)
    October 2018 to July 2024
    Type
    Interventional
    Phase
    4

    Participation criteria

    Sex
    Female & Male
    Age
    Not applicable
    Accepts healthy volunteers
    No
    • Male or female, pediatric or adult, subjects with ADA-SCID, who have been previously treated with GSK2696273.
    • Capable of giving signed informed consent or signed informed consent provided by the subject’s parent or legal guardian.
    • Presence of concomitant condition(s) that in the Investigator’s opinion makes participation in the study unsuitable or may prevent compliance with the protocol requirements.
    • Unlikely to comply with the requirements of the protocol (i.e. attendance for blood sampling on an approximately annual basis).
    Inclusion and exclusion criteria
    Inclusion criteria:
    • Male or female, pediatric or adult, subjects with ADA-SCID, who have been previously treated with GSK2696273.
    • Capable of giving signed informed consent or signed informed consent provided by the subject’s parent or legal guardian.
    Exclusion criteria:
    • Presence of concomitant condition(s) that in the Investigator’s opinion makes participation in the study unsuitable or may prevent compliance with the protocol requirements.
    • Unlikely to comply with the requirements of the protocol (i.e. attendance for blood sampling on an approximately annual basis).
    • Transportation of viable samples to the European Union (EU) central laboratory from the subject’s home country is not possible.

    Trial location(s)

    Location
    Status
    Contact us
    Contact us
    Location
    GSK Investigational Site
    Bruxelles, Belgium, 1200
    Status
    Will Be Recruiting
    Contact us
    Location
    GSK Investigational Site
    Hannover, Niedersachsen, Germany, 30625
    Status
    Will Be Recruiting
    Contact us
    Location
    GSK Investigational Site
    Jerusalem, Israel, 91120
    Status
    Will Be Recruiting
    Contact us
    Location
    GSK Investigational Site
    Milano, Lombardia, Italy, 20132
    Status
    Will Be Recruiting
    Contact us

    Study documents

    No study documents available.

    Results overview

    Study Results yet to be posted

    Recruitment status
    No longer a GSK study
    Actual primary completion date
    Not applicable
    Actual study completion date
    Not applicable

    Plain language summaries

    Plain language summaries of clinical trial results for Phase 2-4 clinical trials that were initiated on or after January 2022 will be posted by GSK within one year following study completion.

    Additional information about the trial

    Additional information
    Not applicable
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