Observational long-term follow- up study for patients previously treated with ex vivo gene therapy

Long term follow-up for patients with inborn errors of immunity treated with autologous ex vivo gene modified CD34+ advanced therapies at Great Ormond Street Hospital

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

ISRCTN

Registry ID

ISRCTN91900773

Enrollment

Registered

2025-06-11

Start date

2026-07-01

Completion date

Unknown

Last updated

2026-05-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Medical condition: X-linked SCID (SCID-X1)

Interventions

Patients will continue to receive routine medical care and follow-up appointments at GOSH, just as they normally would. This study will involve reviewing medical records and collecting information fro

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Participants must have previously received a CD34+ ATIMP through a clinical trial or compassionate use program at GOSH. 2. The patient displays persistent detectable gene marking. 3. The patient is not followed-up on another LTFU study for the same condition. 4. The patient or their guardian can provide informed consent. 5. Inclusion can be prospective or retrospective.

Exclusion criteria

Exclusion criteria: Refusal to sign informed consent

Design outcomes

Primary

Measure	Time frame
Characterize the long-term safety of the gene therapy treatment and detect potential gene therapy-related adverse events in participants who received an autologous ex vivo gene modified CD34+ advanced therapy at GOSH. Monitoring will be performed during yearly visit from Year 3 or 4 to Year 15 post-infusion and will include documentation of the: 1.Adverse events (AEs), serious adverse events (SAEs), and/or adverse reactions are measured through clinical assessments and review of medical records. 2.Overall survival (OS) and event-free survival (EFS) are assessed through clinical review of survival status and defined clinical events (rescue HSCT, second gene therapy, mutagenic therapy exposure). 3.Vector copy number (VCN) in peripheral blood cell lineages, measured using quantitative PCR (qPCR).	—

Measure

Time frame

Characterize the long-term safety of the gene therapy treatment and detect potential gene therapy-related adverse events in participants who received an autologous ex vivo gene modified CD34+ advanced therapy at GOSH. Monitoring will be performed during yearly visit from Year 3 or 4 to Year 15 post-infusion and will include documentation of the: 1.Adverse events (AEs), serious adverse events (SAEs), and/or adverse reactions are measured through clinical assessments and review of medical records. 2.Overall survival (OS) and event-free survival (EFS) are assessed through clinical review of survival status and defined clinical events (rescue HSCT, second gene therapy, mutagenic therapy exposure). 3.Vector copy number (VCN) in peripheral blood cell lineages, measured using quantitative PCR (qPCR).

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Secondary

Measure	Time frame
Evaluate long-term, sustained disease correction and clinical efficacy of the gene therapy treatment through collection of ‘standard of care’ assessment data. Monitoring will be performed during yearly visit from Year 3 or 4 to Year 15 post-infusion and will include documentation of the: 1. Immune reconstitution, measured via laboratory assessments of blood cell counts, lymphocyte subsets (absolute numbers and percentages), and immunoglobulin levels. 2. Discontinuation of immunoglobulin replacement therapy, assessed through review of treatment records and time to discontinuation.	—

Measure

Time frame

Evaluate long-term, sustained disease correction and clinical efficacy of the gene therapy treatment through collection of ‘standard of care’ assessment data. Monitoring will be performed during yearly visit from Year 3 or 4 to Year 15 post-infusion and will include documentation of the: 1. Immune reconstitution, measured via laboratory assessments of blood cell counts, lymphocyte subsets (absolute numbers and percentages), and immunoglobulin levels. 2. Discontinuation of immunoglobulin replacement therapy, assessed through review of treatment records and time to discontinuation.

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Countries

United Kingdom

Contacts

Public ContactFahmida Hoque

fahmida.hoque@gosh.nhs.uk+44 20 74059200

Outcome results

None listed

Source: ISRCTN (via WHO ICTRP) · Data processed: May 22, 2026