Severe Combined Immunodeficiency Due to ADA DeficiencyNCT04959890
Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy
An observational study of Severe Combined Immunodeficiency Due to ADA Deficiency, run by Fondazione Telethon at 1 site in Italy.
Trial summary
This study is a post approval commitment to evaluate the accuracy and precision of retroviral insertion site (RIS) analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects treated with Strimvelis. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Severe Combined Immunodeficiency Due to ADA Deficiency
- What is being tested?
- Strimvelis
- How many people
- 15 participants
- Where
- 1 siteItaly
- Study dates
- 23 Apr 2021to 31 Mar 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This study will use biological samples previously taken from subjects treated with Strimvelis gRV-GT.
Who may be able to join
- Subjects must have previously received treatment with gRV-GT, either during clinical development (clinical trials and early access programs) or in the post-marketing setting as the approved product (Strimvelis) or under hospital exemption, and for whom at least one biological sample is available that meets the following eligibility criteria::
- Peripheral blood, bone marrow, or DNA extracted from either source.
- Taken at least 6 months after gRV-GT.
- Stored at -20oC or below since the time of sampling.
- Likely to provide at least 1.5 μg of DNA (following extraction by the central laboratory).
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Methodology Study to Investigate the Utility of Retroviral Insertion Site Analysis in Samples From Subjects Treated With Strimvelis Gene Therapy.
This study is a post approval commitment to evaluate the accuracy and precision of retroviral insertion site (RIS) analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects treated with Strimvelis.
Read the detailed description
This non-interventional, retrospective, methodology study will use peripheral blood and bone marrow samples previously taken from subjects treated with Strimvelis. The study does not require subjects to undergo any further treatment, intervention or blood withdrawal.
The objective of this methodology study is to evaluate the accuracy and precision of shearing extension primer tag selection ligation-mediated polymerase chain reaction (S-EPTS/LM-PCR) for RIS analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects previously treated with Strimvelis for severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.
- Being tested
- Strimvelis
- Keywords
- ada-scid, retroviral vector, gene therapy
- Medical subject headings
- Severe combined immunodeficiency due to adenosine deaminase deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
StrimvelisGenetic
This non-interventional, retrospective, methodology study will use peripheral blood and bone marrow samples previously taken from subjects treated with gRV-GT. The study does not require subjects to undergo any further treatment, intervention or blood withdrawal. Eligible samples will be shipped to a central laboratory and will undergo DNA extraction prior to S-EPTS/LM-PCR analysis. Each analysis run will include a control DNA sample and up to four subject samples. Each sample will be analysed in triplicate.
| Study group | Role | What they receive |
|---|---|---|
| Subjects Previously Treated with Strimvelis (or GSK2696273) Gene Therapy | It is expected that this study will include approximately 70 eligible samples from approximately 15 subjects previously treated with gamma retroviral gene therapy (gRV-GT). Receives: Genetic: Strimvelis |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the precision of S-EPTS/LM-PCR methodology for RIS analysis using control insertion site DNA
Precision will be determined by the variability (%CV) of the abundance data.
Measured: Retrospective sample analysis.
To assess the accuracy of S-EPTS/LM-PCR methodology for RIS using control insertion site DNA
Accuracy will be determined based on the difference between the mean retrieved abundance and the expected abundance of control DNA.
Measured: Retrospective sample analysis.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2021
- Study start23 Apr 2021
- Primary completion31 Mar 2024 (estimated)
- Study completion31 Mar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Combined Immunodeficiency Due to ADA DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione Telethon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Fondazione Telethon, Study director, Fondazione Telethon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04959890
- Sponsor's protocol ID
- STRIM-005
- Status last verified
- Jan 2024
- First submitted
- 21 Jun 2021
- Last updated
- 29 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Methodology Study to Investigate the Utility of Retroviral Insertion Site Analysis in Samples From Subjects Treated With Strimvelis Gene Therapy.
- ClinicalTrials.gov ID
- NCT04959890
- Acronym / sponsor ID
- STRIM-005
- Keywords
- ada-scid, retroviral vector, gene therapy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Fondazione TelethonUniversity, hospital or other
- Study start
- 23 Apr 2021 (actual)
- Primary completion
- 31 Mar 2024 (estimated)
- Study completion
- 31 Mar 2024 (estimated)
- First posted
- 13 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is a post approval commitment to evaluate the accuracy and precision of retroviral insertion site (RIS) analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects treated with Strimvelis.
Detailed description
This non-interventional, retrospective, methodology study will use peripheral blood and bone marrow samples previously taken from subjects treated with Strimvelis. The study does not require subjects to undergo any further treatment, intervention or blood withdrawal.
The objective of this methodology study is to evaluate the accuracy and precision of shearing extension primer tag selection ligation-mediated polymerase chain reaction (S-EPTS/LM-PCR) for RIS analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects previously treated with Strimvelis for severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects Previously Treated with Strimvelis (or GSK2696273) Gene Therapy | – | Genetic: StrimvelisIt is expected that this study will include approximately 70 eligible samples from approximately 15 subjects previously treated with gamma retroviral gene therapy (gRV-GT). |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This study will use biological samples previously taken from subjects treated with Strimvelis gRV-GT.
Key inclusion criteria (5 listed)
- Subjects must have previously received treatment with gRV-GT, either during clinical development (clinical trials and early access programs) or in the post-marketing setting as the approved product (Strimvelis) or under hospital exemption, and for whom at least one biological sample is available that meets the following eligibility criteria::
- Peripheral blood, bone marrow, or DNA extracted from either source.
- Taken at least 6 months after gRV-GT.
- Stored at -20oC or below since the time of sampling.
- Likely to provide at least 1.5 μg of DNA (following extraction by the central laboratory).
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| StrimvelisThis non-interventional, retrospective, methodology study will use peripheral blood and bone marrow samples previously taken from subjects treated with gRV-GT. The study does not require subjects to undergo any further treatment, intervention or blood withdrawal. Eligible samples will be shipped to a central laboratory and will undergo DNA extraction prior to S-EPTS/LM-PCR analysis. Each analysis run will include a control DNA sample and up to four subject samples. Each sample will be analysed in triplicate. | Genetic | Subjects Previously Treated with Strimvelis (or GSK2696273) Gene Therapy |
Primary outcome
To assess the precision of S-EPTS/LM-PCR methodology for RIS analysis using control insertion site DNA
Time frame: Retrospective sample analysis.
Precision will be determined by the variability (%CV) of the abundance data.
To assess the accuracy of S-EPTS/LM-PCR methodology for RIS using control insertion site DNA
Time frame: Retrospective sample analysis.
Accuracy will be determined based on the difference between the mean retrieved abundance and the expected abundance of control DNA.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jan 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fondazione Telethon study director
Fondazione Telethon
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Severe Combined Immunodeficiency Due to ADA Deficiency, run by Fondazione Telethon at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 29 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the precision of S-EPTS/LM-PCR methodology for RIS analysis using control insertion site DNA (time frame: Retrospective sample analysis.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jan 2024.