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Tests treatment safety and results for Pompe Disease (Late-onset)
Official title A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
ClinicalTrials.gov ID: NCT07282847
What this study is testing
What is AB-1009 (GAA Gene)?
AB-1009 (GAA Gene) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for pompe disease (late-onset).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a single-arm, open-label, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of AB-1009 in adult participants with late-onset Pompe disease (LOPD).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- Participant must be ≥18 to ≤65 years of age at the time of signing the informed consent form.
- Confirmed GAA enzyme deficiency from any tissue source and/or confirmed biallelic GAA gene mutations.
- Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (Lumizyme®), avalglucosidase alfa-ngpt (Nexviazyme®)), or cipaglucosidase...
- FVC in the upright position ≥30% and ≤80% of predicted;
- Capable of walking at least 100 meters in the 6MWT (use of a cane, quad cane, or standard walker is permitted);
You likely can't join if
- Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) \<40% or New York Heart Association (NYHA) functional class 3 or above;
- Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
- Intolerance to ERT or investigator-assessed intolerance to ERT, prior experience of serious ERT-related infusion-associated reactions (IARs);
- Have known intrinsic liver diseases, including hepatitis, HIV-related liver disease, prior diagnosis of portal hypertension, splenomegaly, hepatic...
- Prior or ongoing medical condition(s), including any active infection, malignancy within 5 years of screening (except basal or squamous cell skin...
- Have received gene therapy prior to screening;
See the full eligibility criteria
- Participant must be ≥18 to ≤65 years of age at the time of signing the informed consent form.
- Confirmed GAA enzyme deficiency from any tissue source and/or confirmed biallelic GAA gene mutations.
- Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (Lumizyme®), avalglucosidase alfa-ngpt (Nexviazyme®)), or cipaglucosidase alfa (Pombiliti®) for at least 6 months (at least 10 infusions) before...
- FVC in the upright position ≥30% and ≤80% of predicted;
- Capable of walking at least 100 meters in the 6MWT (use of a cane, quad cane, or standard walker is permitted);
- Male or female. Contraceptive/barrier use by men and women requirements as per protocol.
- Capable of giving informed consent.
- Able to understand and comply with all study procedures.
- Severe cardiomyopathy, defined as left ventricular ejection fraction (LVEF) \<40% or New York Heart Association (NYHA) functional class 3 or above;
- Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
- Intolerance to ERT or investigator-assessed intolerance to ERT, prior experience of serious ERT-related infusion-associated reactions (IARs);
- Have known intrinsic liver diseases, including hepatitis, HIV-related liver disease, prior diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, severe fatty liver, cirrhosis or liver fibrosis ≥stage...
- Prior or ongoing medical condition(s), including any active infection, malignancy within 5 years of screening (except basal or squamous cell skin cancer), physical finding(s), assessment findings, or laboratory...
- Have received gene therapy prior to screening;
- Have received any systemic immunosuppressants (except inhalation or topical use) other than glucocorticoids 30 days prior to screening through completion of screening through completion of screening, and/or known...
- Use of investigational drugs or drugs that could affect this study as evaluated by the investigator within 30 days prior to screening through completion of Week 52 or within 5 half-lives of the investigational drug...
- Have received any vaccine within 30 days prior to dosing;
- Other conditions that make the participant not eligible for the study according to the investigator.
- Contraindication to MRI, hypersensitivity to contrast dyes, shellfish, or iodine, or implanted spinal rods, cardiac pacemaker, or other implantation that would distort cMRI images.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Irvine, California, United States
- Palo Alto, California, United States
- New York, New York, United States
- Durham, North Carolina, United States
- Philadelphia, Pennsylvania, United States
- Pittsburgh, Pennsylvania, United States
- Dallas, Texas, United States
- Richmond, Virginia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Irvine, California, United States; Palo Alto, California, United States; New York, New York, United States; Durham, North Carolina, United States; Philadelphia, Pennsylvania, United States and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.