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Gene Therapy

Gene Therapy (AT845) for Pompe Disease (FORTIS Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Audentes Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Subject has upright FVC ≥ 30% of predicted normal value.
Subject has been on a stable standard dose (at least 20 mg/kg every 2 weeks) of ERT with rhGAA for at least the previous 6 months.
Must not have
Subject has received immune-modulating agents within 90 days before dosing (use of inhaled corticosteroids is allowed); use of other concomitant medications to manage chronic conditions must have been stable for at least 30 days before dosing. Concomitant medications that may predispose the subject to peripheral neuropathy will be evaluated.
Subject has a contraindication to study drug or to corticosteroids, or has demonstrated hypersensitivity to any of the components of the study drug.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up change from baseline and up to month 60
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new drug for people with Late Onset Pompe Disease, a rare muscle disorder. The goal is to see if the drug is safe and effective.

Who is the study for?
Adults over 18 with Late Onset Pompe Disease (LOPD) who have been on enzyme replacement therapy for at least 2 years are eligible. They must not have certain allergies, be part of another study, or have conditions that could affect safety or results. Participants must agree to not donate blood or reproductive cells for a period after receiving the trial treatment.
What is being tested?
The clinical trial is testing AT845, a gene transfer therapy, in adults with LOPD to assess its safety and effectiveness. This phase 1/2 trial involves multiple centers where participants receive increasing doses of AT845 while their health outcomes are monitored.
What are the potential side effects?
While specific side effects aren't listed here, gene therapies like AT845 can potentially cause immune reactions, infusion-related responses, and may interact with existing medications or pre-existing health conditions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My lung function is at least 30% of what is expected for someone healthy.
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I have been on a consistent dose of ERT with rhGAA for 6 months.
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I have been diagnosed with Pompe disease through genetic testing.
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I am 18 years old or older.
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I have been on enzyme replacement therapy with rhGAA for at least 2 years.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I haven't taken immune-modulating drugs in the last 90 days, except for inhaled corticosteroids.
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I am allergic or cannot take the study drug or corticosteroids due to health reasons.
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I do not have a serious heart condition or an ejection fraction below 40%.
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I have taken immunosuppressive drugs for myopathy or neuropathy in the last 3 months.
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I am not currently in a clinical trial or have had gene or cell therapy.
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I have a high risk of severe allergic reactions to certain enzyme treatments.
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My blood test shows I have AAV8 antibodies.
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I have been diagnosed with nerve damage in my hands or feet.
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I have a bleeding condition due to blood-thinning medications.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~change from baseline and up to month 60
This trial's timeline: 3 weeks for screening, Varies for treatment, and change from baseline and up to month 60 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
GAA enzymatic activity
GAA protein expression
Safety and Tolerability over time
Secondary study objectives
6-Minute Walk Test (for ambulatory patients)
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) Questionnaire
Forced Vital Capacity (FVC)
+8 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups
Experimental Treatment
Group I: Third Dose CohortExperimental Treatment1 Intervention
1x10\^14 vg/kg of AT845 administered via intravenous infusion
Group II: Second Dose CohortExperimental Treatment1 Intervention
6x10\^13 vg/kg of AT845 administered via intravenous infusion
Group III: Initial Dose CohortExperimental Treatment1 Intervention
3x10\^13 vg/kg of AT845 administered via intravenous infusion

Find a Location

Who is running the clinical trial?

Audentes TherapeuticsLead Sponsor
7 Previous Clinical Trials
262 Total Patients Enrolled
Astellas Gene TherapiesLead Sponsor
6 Previous Clinical Trials
386 Total Patients Enrolled
Angela Smith, MDStudy ChairAstellas Gene Therapies
4 Previous Clinical Trials
166 Total Patients Enrolled

Media Library

AT845 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04174105 — Phase 1 & 2
Pompe Disease Research Study Groups: Second Dose Cohort, Third Dose Cohort, Initial Dose Cohort
Pompe Disease Clinical Trial 2023: AT845 Highlights & Side Effects. Trial Name: NCT04174105 — Phase 1 & 2
AT845 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04174105 — Phase 1 & 2
~10 spots leftby Nov 2029
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