CompletedPhase 1/2

Gene Therapy for Wiskott-Aldrich Syndrome

Italy8 participantsStarted 2010-04-20

Plain-language summary

This is phase I/II protocol to evaluate the safety and efficacy of WAS gene transfer into hematopoietic stem/progenitor cells for the treatment of Wiskott Aldrich Syndrome.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:
✓. No HLA-identical sibling donor
✓. Negative search for a matched unrelated donor (10/10) or an adequate unrelated cord blood donor (5-6/6) within 4-6 months
✓. Parental/guardian/patient signed informed consent.

Exclusion criteria

✕. Patients positive for HIV-infection.
✕. Patients affected by neoplasia.
✕. Patients with cytogenetic alterations typical of MDS/AML.
✕. Patients with end-organ functions or any other severe disease which, in the judgement of the investigator, would make the patient inappropriate for entry into this study.
✕. Patients who underwent an allogeneic haematopoietic stem cell transplantation in the previous 6 months.
✕. Patients who underwent an allogeneic haematopoietic stem cell transplantation with evidence of residual cells of donor origin.

What they're measuring

Safety of Reduced Conditioning Regimen

Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)

Safety of Lentivirus Gene Transfer Into HSC

Timeframe: after 48 hours after Telethon003 infusion

Sustained Engraftment of Genetically Corrected Haematopoietic Stem Cells in Peripheral Blood and/or in Bone Marrow

Timeframe: at 1 year after Telethon003 infusion

Presence of Detectable Vector-derived WASP

Timeframe: Median duration: 11.1 years (range: 8.01 -13.3 years)

Improved T-cell Functions

Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)

Antigen-specific Responses to Vaccination

Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)

Improved Platelet Count and MPV Normalization

Timeframe: up to 3 years after Telethon003 infusion

Trial details

NCT IDNCT01515462

SponsorFondazione Telethon

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2023-10-04

Results submitted2024-12-16

View on ClinicalTrials.gov More Wiskott-Aldrich Syndrome (WAS) trials

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:
✓. No HLA-identical sibling donor
✓. Negative search for a matched unrelated donor (10/10) or an adequate unrelated cord blood donor (5-6/6) within 4-6 months
✓. Parental/guardian/patient signed informed consent.

Exclusion criteria

✕. Patients positive for HIV-infection.
✕. Patients affected by neoplasia.
✕. Patients with cytogenetic alterations typical of MDS/AML.
✕. Patients with end-organ functions or any other severe disease which, in the judgement of the investigator, would make the patient inappropriate for entry into this study.
✕. Patients who underwent an allogeneic haematopoietic stem cell transplantation in the previous 6 months.
✕. Patients who underwent an allogeneic haematopoietic stem cell transplantation with evidence of residual cells of donor origin.

What they're measuring

Safety of Reduced Conditioning Regimen

Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)

Safety of Lentivirus Gene Transfer Into HSC

Timeframe: after 48 hours after Telethon003 infusion

Sustained Engraftment of Genetically Corrected Haematopoietic Stem Cells in Peripheral Blood and/or in Bone Marrow

Timeframe: at 1 year after Telethon003 infusion

Presence of Detectable Vector-derived WASP

Timeframe: Median duration: 11.1 years (range: 8.01 -13.3 years)

Improved T-cell Functions

Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)

Antigen-specific Responses to Vaccination

Timeframe: Follow up phase - Median duration: 11.1 years (range: 8.01 -13.3 years)

Improved Platelet Count and MPV Normalization

Timeframe: up to 3 years after Telethon003 infusion