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Gene Therapy Trial Report

Summary

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease


NCTID NCT04519749 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Fabry Disease
Disease Ontology Term DOID:14499
Compound Name 4D-310
Compound Description adeno-associated virus serotype 2 capsid variant (4D-C102) carrying a transgene encoding a codon-optimized human alpha-galactosidase A gene
Sponsor 4D Molecular Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 18 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant GLA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Cardiomyocyte
Delivery System Viral transduction
Vector Type AAV C102
Editor Type none
Dose 1 1E13 vg/kg

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-08-14
Completion Date 2030-06
Last Update 2024-04-08

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Male or female ≥ 18 years of age 2. Pathogenic GLA mutation consistent with Fabry Disease 3. Confirmed diagnosis of classic or late-onset Fabry disease 4. Individuals on ERT must be on a stable dose for at least 6 months (and a minimum of 12 months total exposure) prior to study enrollment 5. Agree to use highly effective contraception Exclusion Criteria: 1. Presence of high titer neutralizing antibody to 4D-310 capsid, or presence of high antibody titer to AGA 2. eGFR \<45 mL/min/1.73 m2 3. Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis 4. HIV, active or chronic hepatitis B or C, 5. Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control 6. History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism) 7. Contraindication to systemic corticosteroid therapy or immunosuppressive therapy 8. Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months. 9. Moderately severe to severe cardiovascular disease or uncontrolled hypertension 10. Left ventricular ejection fraction of \<45% on echocardiogram (ECHO) 11. Currently receiving investigational drug, device or therapy or having ever received gene therapy 12. History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation 13. History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent) 14. Pregnant or breast-feeding
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates No further significant investment is expected on this program, pending additional financing or partnerships

Resources/Links