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Gene Therapy (AT845) for Pompe Disease (FORTIS Trial)
FORTIS Trial 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.
FORTIS Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowFORTIS Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.FORTIS Trial Design
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Who is running the clinical trial?
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- You have high levels of GAA antibodies in your blood.I am allergic or cannot take the study drug or corticosteroids due to health reasons.I haven't taken immune-modulating drugs in the last 90 days, except for inhaled corticosteroids.You have evidence of HIV-1 or HIV-2 in your blood.My lung function is at least 30% of what is expected for someone healthy.I do not have a serious heart condition or an ejection fraction below 40%.You have certain medical conditions like uncontrolled diabetes, alcohol use disorder, or active malignancy requiring chemotherapy and/or radiation that are commonly linked to peripheral neuropathy. Other medical conditions that may lead to peripheral neuropathy will be reviewed on a case-by-case basis.I have been on a consistent dose of ERT with rhGAA for 6 months.I have been diagnosed with Pompe disease through genetic testing.I agree not to donate blood, sperm, or eggs for at least 90 days after treatment or until I have 3 negative tests.You have had a bad reaction to certain asthma medications, which means you can't participate in the lung function tests.I have taken immunosuppressive drugs for myopathy or neuropathy in the last 3 months.I am not currently in a clinical trial or have had gene or cell therapy.I have a high risk of severe allergic reactions to certain enzyme treatments.I do not have significant liver disease or active hepatitis B or C.I have no other health conditions that could affect my safety or the study results.My blood test shows I have AAV8 antibodies.You have had a bad reaction to MRI contrast agents before.You currently have a viral infection that is making you feel sick.You have any abnormal lab test results that could affect your ability to take part in the study, except for specific tests related to the condition being studied.I have been diagnosed with nerve damage in my hands or feet.I am 18 years old or older.I have been on enzyme replacement therapy with rhGAA for at least 2 years.I have a bleeding condition due to blood-thinning medications.
- Group 1: Second Dose Cohort
- Group 2: Third Dose Cohort
- Group 3: Initial Dose Cohort
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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
Are participants of 45 years and older being considered for this research?
"Those that hope to join this medical trial must be aged between 18 and 80. There are separate clinical trials available for children below the age of 18, as well as those over 65."
In how many locations can this clinical trial be accessed?
"Across the United States, 7 medical centres are engaging in this clinical trial. These include University of California Irvine, Department of Neurology in Dallas, Stanford University in Atlanta and University of Utah Division of Medical Genetics in Orange - as well as other sites nationwide."
Is the recruitment phase of this research project still open?
"This clinical trial is no longer in need of participants, as indicated on the website. The initial posting was made October 28th 2020 and it has been edited most recently on July 6th 2022. Fortunately, there are 17 other studies with open recruitment at this moment."
What is the prognosis for success of this research experiment?
"The primary evaluation metric of this trial, taking place over a 48 week period, is the safety and tolerability of the treatment. Secondary objectives include assessing vector copy number/mRNA transcripts in muscle biopsies, maximum expiratory pressure using pulmonary function testing, as well as thigh fat fraction with magnetic resonance imaging (MRI)."
Would I be eligible to participate in this research?
"This clinical trial is recruiting 12 volunteers between eighteen and eighty years old that have acid maltase deficiency. Key criteria for candidacy include: Written informed consent given by the subject or their legally authorised representative (LAR), aged 18+ with a verified diagnosis of Pompe disease, 2 year history of enzyme replacement therapy (ERT) using rhGAA on an appropriate dose, FVC ≥ 30% of predicted normal value, contraception to be used from date of signing consent through 6 months post AT845 administration period."
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