Adenosine deaminase (ADA) deficiency severe combined immunodeficiency MedDRA version: 20.1 Level: LLT Classification code 10066367 Term: Adenosine deaminase deficiency System Organ Class: 100000004850
Conditions
Interventions
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
Con
Sponsors
Orchard Therapeutics (Europe) Ltd
Eligibility
Sex/Gender
All
Inclusion 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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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: •between subjects at every time point •within subjects over time points •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
| Measure | Time frame |
|---|---|
| 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 | — |
Countries
Italy, Switzerland, Turkey
Contacts
Public ContactClinical
ORCHARD THERAPEUTICS LTD
Outcome results
None listed