Ignite Creation Date:
2024-05-06 @ 11:48 AM
Last Modification Date:
2024-10-26 @ 12:50 PM
Study NCT ID:
NCT03601286
Status:
RECRUITING
Last Update Posted:
2023-10-12
First Post:
2018-02-22
Brief Title:
Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
Sponsor:
Great Ormond Street Hospital for Children NHS Foundation Trust
Organization:
Great Ormond Street Hospital for Children NHS Foundation Trust
Study Overview
Official Title:
Phase III Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan
Status:
RECRUITING
Status Verified Date:
2023-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Severe combined immunodeficiency disorder SCID is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function resulting in lack of both cellular and humoral immunity SCID arises from a variety of molecular defects which affect lymphocyte development and function The most common form of SCID is an X-linked form SCID-X1 which accounts for 30-50 of all cases SCID-X1 is caused by defects in the common cytokine receptor gamma chain which was originally identified as a component of the high affinity interleukin-2 receptor IL2RG
Allogeneic haematopoietic stem cell transplantation HSCT which replaces the patients bone marrow with that of a healthy donor is the only treatment that definitively restores the normal function of the bone marrow HSCT is the first choice of treatment for patients with signs of bone marrow failure and a fully-matched related donor However patients without a fully-matched related donor have much worse overall outcomes from HSCT
This study will investigate whether patients with SCID-X1 without a fully matched related donor may benefit from gene therapy To do this the investigators propose to perform a phase III clinical trial to evaluate the safety and efficacy effect of gene therapy for SCID-X1 patients using a lentivirus delivery system containing the IL2RG gene Up to 5 eligible SCID-X1 patients will undergo mobilisation and harvest of their haematopoietic stem precursor cells HPSCs In the laboratory the disabled lentivirus will be used to insert a normal human IL2RG gene into the patients harvested HPSCs Patients will receive chemotherapy conditioning prior to cell infusion in order to enhance grafting The genetically corrected stem cells will then be re-infused into the patient Patients will be followed up for 2 years This trial will determine whether gene therapy for SCID-X1 using a lentiviral vector is safe feasible and effective
Allogeneic haematopoietic stem cell transplantation HSCT which replaces the patients bone marrow with that of a healthy donor is the only treatment that definitively restores the normal function of the bone marrow HSCT is the first choice of treatment for patients with signs of bone marrow failure and a fully-matched related donor However patients without a fully-matched related donor have much worse overall outcomes from HSCT
This study will investigate whether patients with SCID-X1 without a fully matched related donor may benefit from gene therapy To do this the investigators propose to perform a phase III clinical trial to evaluate the safety and efficacy effect of gene therapy for SCID-X1 patients using a lentivirus delivery system containing the IL2RG gene Up to 5 eligible SCID-X1 patients will undergo mobilisation and harvest of their haematopoietic stem precursor cells HPSCs In the laboratory the disabled lentivirus will be used to insert a normal human IL2RG gene into the patients harvested HPSCs Patients will receive chemotherapy conditioning prior to cell infusion in order to enhance grafting The genetically corrected stem cells will then be re-infused into the patient Patients will be followed up for 2 years This trial will determine whether gene therapy for SCID-X1 using a lentiviral vector is safe feasible and effective
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None