ClinicalTrials.gov record NCT07282847
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)
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)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a single-arm, open-label, dose-escalation study testing one intravenous infusion of AB-1009 in adults with late-onset Pompe disease. Up to 12 participants will be assigned to one of two dose cohorts and followed for a primary observation period of 52 weeks, then for long-term follow-up up to 4 years. The study collects safety and multiple measures of enzyme activity, muscle, breathing, and physical function.
What it is studying. The study tests whether a single IV infusion of AB-1009 is safe and tolerated and how it affects GAA enzyme activity, muscle glycogen and function, breathing, viral shedding, and patient-reported physical function in adults with LOPD.
What the researchers hope to learn. To evaluate the safety and tolerability of a single IV infusion of AB-1009 in adults with late-onset Pompe disease. To assess biological and clinical measures including serum GAA activity, urinary Glc4, muscle biopsy GAA activity and glycogen content, viral shedding, respiratory measures (FVC, MIP, MEP), 6-minute walk distance, and patient- and clinician-reported global and physical-function outcomes.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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).
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Genetic
- AB-1009 (GAA Gene)
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion 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 signing the initial informed consent form. During the screening process, participants need to remain on their current ERT until close to dosing;
- 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.
Exclusion criteria
- 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 2, ultrasound-identified liver neoplasms, or laboratory tests suggesting elevated alpha-fetoprotein. Patients with liver function tests including ALT or AST >3× upper limit of normal (ULN) or any total bilirubin above ULN during screening will also be excluded;
- 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 abnormality that, in the investigator's opinion, would impact participant's safety and compliance with the study procedures.
- 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 intolerance to immunosuppressants such as glucocorticoids; other concomitant immunosuppression would require sponsor approval.
- 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 (whichever is longer);
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This is an open-label study, up to 12 participants will receive a single IV infusion of AB-1009. Participants will be assigned to either cohort 1 (1.0E13 vg/kg) or Cohort 2 (1.5E13 vg/kg) based on enrollment in the study. Study duration will include a screening period of up to 75 days, primary observation of 52 weeks, and a long-term follow-up period of 4 years.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After a screening period (up to 75 days), participants receive one single intravenous infusion of AB‑1009 and are closely monitored. Primary monitoring lasts 52 weeks and includes regular clinic visits, blood tests, respiratory tests (FVC, MIP/MEP), 6‑Minute Walk Tests, patient questionnaires, and sampling for viral shedding (blood, saliva, urine). Muscle biopsies are collected at baseline and Week 52 (an invasive procedure). Participants enter a long‑term follow‑up period of 4 years for ongoing safety monitoring.
How the study is structured. Participants receive one intravenous infusion of AB-1009 and are not randomized. The study is open-label (both participants and investigators know the treatment) and uses a sequential, dose-escalation plan with two dose cohorts (1.0E13 vg/kg and 1.5E13 vg/kg). Study time includes screening (up to 75 days), a 52-week primary observation period, and a long-term follow-up of up to 4 years. Up to 12 adults will be enrolled.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Pompe disease
Important dates
- Study start
- Apr 15, 2026
- Primary completion
- September 2028 (estimated)
- Study completion
- September 2032 (estimated)
- Target enrollment
- 12 participants (estimated)
- Last updated on registry
- Jul 20, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- AskBio Inc
- Collaborators
- Bayer
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- AskFirst Patient Engagement 📞 919-561-6210 ✉ AskFirst@AskBio.com
Participating sites (9)
- Barrow Neurological InstituteNot Yet Recruiting
Phoenix, Arizona, 85013 · United States
- Shafeeq Ladha, MD
- University of California, Irvine (UCI)Recruiting
Irvine, California, 92697 · United States
- UCI Alpha Clinic · 949-824-3990 · alphaclinic@hs.uci.edu
- Tahseen Mozaffar, MD
- Stanford Neuroscience Health CenterNot Yet Recruiting
Palo Alto, California, 94304 · United States
- John Day, MD, PhD
- NYU LangoneNot Yet Recruiting
New York, New York, 10016 · United States
- Nicolas Abreu, MD
- Duke UniversityRecruiting
Durham, North Carolina, 27705 · United States
- Ming Xu, RN, MSN · 919-681-1945 · mingfen.xu@duke.edu
- Natalie Katz, MD, PhD
- Hospital of the University of PennsylvaniaRecruiting
Philadelphia, Pennsylvania, 19104 · United States
- Amanda Hicks · 267-496-3772 · Amanda.Hicks@pennmedicine.upenn.edu
- Abigail Browngoehl · Abigail.Browngoehl@Pennmedicine.upenn.edu
- Paul McIntosh, MD
- University of Pittsburgh Medical Center (UPMC)Not Yet Recruiting
Pittsburgh, Pennsylvania, 15213 · United States
- Paula Clemens, MD
- University of Texas Southwest Medical CenterRecruiting
Dallas, Texas, 75235 · United States
- Markey McNutt, MD, PhD · 214-645-7411 · geneticsresearch@utsouthwestern.edu
- Juana Luevao · 214-645-7411 · geneticsresearch@utsouthwestern.edu
- Markey McNutt, MD, PhD
- Virginia Commonwealth University (VCU)Not Yet Recruiting
Richmond, Virginia, 23298 · United States
- Susan Sparks, MD, PhD
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- dose-escalation
- Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
- informed consent form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- FVC
- A breathing test that measures the total amount of air a person can blow out after a full breath in. It is often given as a percentage of what is expected for a person's age and size.
- 6MWT
- A test of how far a person can walk on a flat surface in six minutes. It is used to measure stamina and to track change over time.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- during screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- immunosuppressants
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- glucocorticoids
- Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
- sponsor
- The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- Contraindication
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- MRI
- A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- long-term follow-up
- Observation that continues for years after the study treatment, often required for gene and cell therapies.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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