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Trial record 2 of 2 for:    4D Molecular Therapeutics | Fabry Disease
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4D-310 in Adults With Fabry Disease and Cardiac Involvement

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05629559
Recruitment Status : Active, not recruiting
First Posted : November 29, 2022
Last Update Posted : January 31, 2023
Sponsor:
Information provided by (Responsible Party):
4D Molecular Therapeutics

Study Description
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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

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: 4D-310 Phase 1 Phase 2

Detailed Description:
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
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement
Actual Study Start Date : October 16, 2022
Estimated Primary Completion Date : May 31, 2024
Estimated Study Completion Date : May 31, 2028

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 4D-310 Dose Level 1 - AAV Neutralizing Antibody (NAb) Group A
4D-310 Dose Level 1 - AAV NAb Titer Group A patients
 Biological: 4D-310
Single IV administration of 4D-310 Dose Level 1
Experimental: 4D-310 Dose Level 1 - AAV NAb Titer Group B
4D-310 Dose Level 1 - AAV NAb titer Group B patients
 Biological: 4D-310
Single IV administration of 4D-310 Dose Level 1
Experimental: 4D-310 Dose Level 2 - AAV NAb Titer Group A and/or B
4D-310 at Dose Level 2 in AAV NAb titer Group A and/or B patients
 Biological: 4D-310
Single IV administration of 4D-310 Dose Level 2
Experimental: 4D-310 Dose Expansion
Dose expansion cohort of 4D-310 at the selected dose and selected AAV Nab titer group(s) patients
 Biological: 4D-310
Single IV administration of 4D-310 at the selected dose


Outcome Measures
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Primary Outcome Measures :
  1. Incidence and severity of adverse events [ Time Frame: 1 year ]
    Incidence and severity of adverse events following a single IV dose of 4D-310


Secondary Outcome Measures :
  1. Change from baseline in serum AGA activity [ Time Frame: 1 year ]
    Change from baseline in serum AGA activity

  2. Change from baseline serum globotriaosylsphingosine (lysoGb3) [ Time Frame: 1 year ]
    Change from baseline serum globotriaosylsphingosine (lysoGb3)


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Adult males and females
  2. Pathogenic GLA mutation consistent with Fabry Disease
  3. Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement
  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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05629559


Locations
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Australia
Royal Melbourne Hospital
Melbourne, Australia, 3050
Royal Perth Hospital
Perth, Australia, 6000
Taiwan
Taipei Veterans General Hospital
Taipei, Taiwan, 11217
Sponsors and Collaborators
4D Molecular Therapeutics
Investigators
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Study Director: Mitra Tavakkoli, MD, PharmD 4D Molecular Therapeutics
More Information
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Responsible Party: 4D Molecular Therapeutics
ClinicalTrials.gov Identifier: NCT05629559    
Other Study ID Numbers: 4D-310-C002
First Posted: November 29, 2022    Key Record Dates
Last Update Posted: January 31, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by 4D Molecular Therapeutics:
Lysosomal Storage Diseases
Nervous System Brain Diseases
Inborn Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases
 Metabolic Diseases
Lipid Metabolism Disorders
Metabolic
X-Linked
Inborn
Sphingolipidoses
Metabolism
Inborn Errors
Lipodoses
Lipid Metabolism
Additional relevant MeSH terms:
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Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
 Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors