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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 7 September 2020
Main ID:  EUCTR2017-001731-39-IT
Date of registration: 02/07/2020
Prospective Registration: No
Primary sponsor: Orchard Therapeutics (Europe) Ltd
Public title: Retroviral insertion site methodology study
Scientific title: Methodology study to investigate the utility of retroviral insertion site analysis in samples from subjects treated with Strimvelis™ gene therapy - Retroviral insertion site methodology study
Date of first enrolment: 12/10/2017
Target sample size: 15
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-001731-39
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: Metodology study If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1  
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes
Countries of recruitment
Italy Switzerland Turkey
Contacts
Name: Clinical   
Address:  108 Cannon street EC4N 6EU Londra United Kingdom
Telephone: +4402037270797
Email: clinical@orchard-tx.com
Affiliation:  ORCHARD THERAPEUTICS LTD
Name: Clinical   
Address:  108 Cannon street EC4N 6EU Londra United Kingdom
Telephone: +4402037270797
Email: clinical@orchard-tx.com
Affiliation:  ORCHARD THERAPEUTICS LTD
Key inclusion & exclusion criteria
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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Adenosine deaminase (ADA) deficiency severe combined immunodeficiency
MedDRA version: 20.1 Level: LLT Classification code 10066367 Term: Adenosine deaminase deficiency System Organ Class: 100000004850
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Intervention(s)

Trade Name: Strimvelis
Product Name: Strimvelis
Product Code: [NA]
Pharmaceutical Form: Powder for solution for infusion
Current Sponsor code: .
Concentration unit: Other
Concentration type: equal
Concentration number: 1-

Primary Outcome(s)
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
Primary end point(s): For subject samples spiked with known amounts of control insertion sites mean abundance and %CV will be calculated:
•between subjects at every time point
•within subjects over time points
•between the same sample within a time point within a subject.
Secondary Objective: Abundance & Diversity of insertion sites in patients treated with Strimvelis
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(s)
Secondary end point(s): Clone abundance at each timepoint for each subject will be calculated and displayed.
•Clones that demonstrate abundance >5% will be listed
•Descriptive statistics (mean, % CV) on clones present >5% will be
captured
•Shannon Diversity Index
•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
Secondary ID(s)
STRIM002-(205813)
Source(s) of Monetary Support
ORCHARD THERAPEUTICS LTD
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 12/10/2017
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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