4D-310 in Adults With Fabry Disease and Cardiac Involvement

NCT05629559 | Recruiting Phase 1 FDA Drug

• 1 other identifier: 4D-310-C002
• Study Type: interventional
• Target: 18
• Locations: 2 countries
• Sites: 4

Brief Summary

This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement

Conditions

Fabry Disease

Keywords

Lysosomal Storage Diseases; Nervous System Brain Diseases; Metabolic; Inborn Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases; X-Linked; Inborn; Metabolic Diseases; Lipid Metabolism Disorders; Sphingolipidoses; Metabolism; Inborn Errors; Lipidoses; Lipid Metabolism

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of adverse events

  Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose

  1 year

Study Arms (4)

4D-310 Dose Level -1

EXPERIMENTAL

Single IV administration of 4D-310 Dose Level -1

Biological: 4D-310

4D-310 Dose Level 2

EXPERIMENTAL

Single IV administration of 4D-310 Dose Level 2

Biological: 4D-310

4D-310 Dose Level 3

EXPERIMENTAL

Single IV administration of 4D-310 at Dose Level 3

Biological: 4D-310

4D-310 Dose Level 1 (No longer enrolling)

EXPERIMENTAL

No longer enrolling - Single IV administration of 4D-310 at Dose Level 1

Biological: 4D-310

Interventions

4D-310 BIOLOGICAL

Single IV administration of 4D-310

4D-310 Dose Level -1; 4D-310 Dose Level 1 (No longer enrolling); 4D-310 Dose Level 2; 4D-310 Dose Level 3

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female ≥ 18 to ≤65 years of age
• Pathogenic GLA mutation consistent with Fabry Disease
• Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement
• Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat
• Individuals receiving ERT, must be on a stable dose for at least 6 months
• Agree to use highly effective contraception

You may not qualify if:

• Presence of pre-existing antibodies to 4D-310 capsid or to AGA
• eGFR \<65 mL/min/1.73 m2
• Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis
• HIV, active or chronic hepatitis B or C,
• Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control
• History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism)
• Contraindication to systemic corticosteroid therapy or immunosuppressive therapy
• Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.
• Moderately severe to severe cardiovascular disease or uncontrolled hypertension
• Left ventricular ejection fraction of \<45% on echocardiogram (ECHO)
• Currently receiving investigational drug, device or therapy or having ever received gene therapy
• History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation
• History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent)
• Pregnant or breast-feeding

Sponsors & Collaborators

• 4D Molecular Therapeutics: lead

Study Sites (4)

Royal Melbourne Hospital

Melbourne, 3050, Australia

ACTIVE NOT RECRUITING

Royal Perth Hospital

Perth, 6000, Australia

ACTIVE NOT RECRUITING

Westmead Hospital

Westmead, 2145, Australia

NOT YET RECRUITING

Taipei Veterans General Hospital

Taipei, 11217, Taiwan

RECRUITING

MeSH Terms

Conditions

Fabry Disease; Lysosomal Storage Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Metabolic Diseases; Lipid Metabolism Disorders; Sphingolipidoses; Lipidoses

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Genetic Diseases, X-Linked; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipid Metabolism, Inborn Errors; Nutritional and Metabolic Diseases

Study Officials

• Alan H Cohen, MD

  4D Molecular Therapeutics

  STUDY DIRECTOR

Central Study Contacts

4DMT Patient Advocacy

CONTACT

(888) 748-8881; clinicaltrials@4DMT.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 4, 2022
• First Posted: November 29, 2022
• Study Start: October 16, 2022
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2030
• Last Updated: February 21, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

TW (1); AU (3)