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Gene Therapy
Gene Therapy for Fabry Disease
Phase 1 & 2
Waitlist Available
Research Sponsored by 4D Molecular Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Confirmed diagnosis of classic or late-onset Fabry disease
Pathogenic GLA mutation consistent with Fabry Disease
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights
Study Summary
This trial is testing a new drug for Fabry Disease in adult males. The goal is to see if it is safe and effective.
Who is the study for?
Adults with Fabry Disease can join this trial. They must have a confirmed diagnosis, be on stable enzyme replacement therapy if applicable, and not have severe diseases like liver or lung disease, recent strokes, or uncontrolled diabetes. Participants need to agree to use effective contraception and cannot be pregnant or breastfeeding.Check my eligibility
What is being tested?
The trial is testing the safety and effects of a single IV dose of gene therapy called 4D-310 in adults with Fabry Disease. It's an open-label study where everyone knows they're getting the treatment, which will vary in dosage as researchers find the best level.See study design
What are the potential side effects?
Possible side effects may include reactions related to immune response due to gene therapy such as fever, fatigue, muscle pain; potential liver issues since patients with existing liver conditions are excluded; also infusion-related responses similar to those seen with enzyme replacement therapies.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with classic or late-onset Fabry disease.
Select...
I have a genetic mutation linked to Fabry Disease.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence and severity of adverse events
Secondary outcome measures
Change from baseline in serum AGA activity
Change from baseline serum globotriaosylsphingosine (lysoGb3)
Trial Design
4Treatment groups
Experimental Treatment
Group I: 4D-310 Dose Level 2 - AAV NAb Titer Group A and/or BExperimental Treatment1 Intervention
Single IV administration of 4D-310 at Dose Level 2 in AAV NAb titer Group A and/or B patients
Group II: 4D-310 Dose Level 1 - AAV Neutralizing Antibody (NAb) Group AExperimental Treatment1 Intervention
Single IV administration of 4D-310 Dose Level 1 - AAV NAb Titer Group A patients
Group III: 4D-310 Dose Level 1 - AAV NAb Titer Group BExperimental Treatment1 Intervention
Single IV administration of 4D-310 Dose Level 1 - AAV NAb titer Group B patients
Group IV: 4D-310 Dose ExpansionExperimental Treatment1 Intervention
Dose expansion cohort of single IV administration of 4D-310 at the selected dose and selected AAV Nab titer group(s) patients
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Who is running the clinical trial?
4D Molecular TherapeuticsLead Sponsor
7 Previous Clinical Trials
355 Total Patients Enrolled
1 Trials studying Fabry Disease
18 Patients Enrolled for Fabry Disease
Alan H Cohen, MDStudy Director4D Molecular Therapeutics
2 Previous Clinical Trials
120 Total Patients Enrolled
1 Trials studying Fabry Disease
18 Patients Enrolled for Fabry Disease
Raphael Schiffmann, MD, MHSc, FAANStudy Director4D Molecular Therapeutics
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I do not have HIV or active/chronic hepatitis B or C.I have severe liver, lung disease, or poorly controlled diabetes.I am 18 years old or older.I haven't had cancer in the past 2 years, except for non-melanoma skin cancer or treated prostate cancer.I am currently in a trial for a new treatment or have had gene therapy.I have serious heart disease or high blood pressure that isn't under control.I have not had a stroke, mini-stroke, or major blood clot in the past year.I had a bad reaction to enzyme replacement therapy that made me stop it.I have been taking oral steroids for 3 months or more in the past year.I have had a kidney transplant or am currently on dialysis.I have been on the same dose of enzyme replacement therapy for at least 6 months.You have a high level of antibodies against the 4D-310 capsid or AGA.I cannot take systemic steroids or immunosuppressive drugs due to health reasons.Your kidney function is less than 45 mL/min/1.73 m2.I have been diagnosed with classic or late-onset Fabry disease.Your heart's pumping ability is less than 45% on an echocardiogram.I have a genetic mutation linked to Fabry Disease.
Research Study Groups:
This trial has the following groups:- Group 1: 4D-310 Dose Level 1 - AAV Neutralizing Antibody (NAb) Group A
- Group 2: 4D-310 Dose Level 2 - AAV NAb Titer Group A and/or B
- Group 3: 4D-310 Dose Expansion
- Group 4: 4D-310 Dose Level 1 - AAV NAb Titer Group B
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any current opportunities for individuals to participate in this trial?
"This research project, which has been posted since September 1st 2020 and updated on March 24th 2022, is currently recruiting participants as stated by clinicaltrials.gov."
Answered by AI
How many healthcare centers are participating in this investigation?
"This trial is being conducted in various locations, such as the Lysosomal & Rare Disorders Research & Treatment Center, Inc in Fairfax, Emory University in Atlanta, and University of Alabama at Birmingham. Additionally there are three other sites hosting this clinical study."
Answered by AI
What is the aggregate size of the cohort being observed in this clinical trial?
"To facilitate this medical research, 18 participants who meet the criteria need to be recruited. The sponsor 4D Molecular Therapeutics will run the study in multiple sites including Lysosomal & Rare Disorders Research & Treatment Center, Inc and Emory University located respectively in Fairfax (Virginia) and Atlanta (Georgia)."
Answered by AI
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