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Gene Therapy Trial Report

Summary

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I


NCTID NCT05682144 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Disease Ontology Term 🔄 DOID:12802
Compound Name 🔄 ISP-001
Sponsor Immusoft of CA, Inc.
Funder Type Industry
Recruitment Status
Enrollment Count 2 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 IDUA
Therapy Type 🔄 Gene transfer
Therapy Route 🔄 Ex-vivo
Mechanism of Action 🔄 Functional gene replacement
Route of Administration 🔄 Intravenous
Drug Product Type 🔄 Autologous cells
Target Tissue/Cell 🔄 CD19 positive B cells
Delivery System 🔄 Electroporation
Vector Type 🔄 Sleeping Beauty
Dose 1 🔄 2.5E7 cells/kg
Dose 2 🔄 10E8 cells/kg

Study Record Dates


Current Stage Phase1
Submit Date 2022-12-12
Completion Date 2039-06-01
Last Update 2025-04-30

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome. * Age ≥ 18 years at time of study registration. * Creatinine clearance, calculated or measured directly, that is \>60ml/min/1.73m2. * Ejection fraction ≥ 40% by echocardiogram. * Must commit to traveling to the study site for the necessary follow-up evaluations. * Must agree to stay \<45-minute drive from the study site for a minimum of 5 days after cell infusion. Exclusion Criteria: * Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to determine presence of germline mutations. * History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders. * Evidence of active graft-vs-host disease. * Underwent a previous hematopoietic stem cell transplant (HSCT). * Requirement for systemic immune suppression. * Requirement for continuous supplemental oxygen. * Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment. * In the investigator's judgement, the subject is unlikely to complete all protocol-required study visits or procedures, including follow up visits, or comply with the study requirements for participation. Other protocol defined inclusion/exclusion criteria may apply.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
FDA Designations 🔄 Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates First patient dosed December 2023, drug is redosable

Resources/Links