Retroviral insertion site methodology study

Phase 1

• Conditions: Adenosine deaminase (ADA) deficiency severe combined immunodeficiency; MedDRA version: 20.1Level: LLTClassification code 10066367Term: Adenosine deaminase deficiencySystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Immune System Diseases [C20]

• Registration Number: EUCTR2017-001731-39-IT
• Lead Sponsor: Orchard Therapeutics (Europe) Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-07-02
• Last Update Posted: 7 September 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1.Male or female, paediatric or adult, patients with ADA-SCID, who have been previously treated with Strimvelis or GSK2696273
Informed Consent
2.Capable of giving signed informed consent as described in Appendix 2 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Or signed informed consent provided by the participant’s parent or legal guardian.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1.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.
2.Unlikely to comply with the requirements of the protocol (i.e. attendance for blood sampling on an approximately annual basis)
3.Transportation of viable samples to the EU central laboratory from the participant’s home country is not possible

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the accuracy and precision of SLiM-PCR methodology for RIS analysis using control insertion site DNA spiked into whole blood samples taken from patients treated with Strimvelis;Secondary Objective: Abundance & Diversity of insertion sites in patients treated with Strimvelis;Primary end point(s): For subject samples spiked with known amounts of control insertion sites mean abundance and %CV will be calculated:<br>•between subjects at every time point<br>•within subjects over time points<br>•between the same sample within a time point within a subject.;Timepoint(s) of evaluation of this end point: Samples will be taken approximately annually for 5 years. The study will include newly treated subjects for whom these samples will be taken approximately annually from 1-5 years relative to the treatment date

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Clone abundance at each timepoint for each subject will be calculated and displayed.<br>•Clones that demonstrate abundance >5% will be listed<br>•Descriptive statistics (mean, % CV) on clones present >5% will be<br>captured<br>•Shannon Diversity Index<br>•Descriptive statistics (% CV) will be captured;Timepoint(s) of evaluation of this end point: Samples will be taken approximately annually for 5 years. The study will include newly treated subjects for whom these samples will be taken approximately annually from 1-5 years relative to the treatment date