Pompe Disease (Late-onset)Phase 1/2NCT07282847
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 Phase 1/2 study testing AB-1009 (GAA Gene) in people with Pompe Disease (Late-onset) and Pompe Disease Late-Onset, run by AskBio Inc at 9 sites in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
- What is being tested?
- AB-1009 (GAA Gene)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 9 sitesUnited States
- Study dates
- 15 Apr 2026to Sep 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 11Show fewer
- 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.
Trial locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
9 sites in 1 country
United States9 sites, 9 recruiting
- Recruiting
University of Texas Southwest Medical Center
Dallas, Texas, 75235
Markey McNutt, MD, PhD, 214-645-7411, geneticsresearch@utsouthwestern.edu
Juana Luevao, 214-645-7411, geneticsresearch@utsouthwestern.edu
- Recruiting
Duke University
Durham, North Carolina, 27705
Ming Xu, RN, MSN, 919-681-1945, mingfen.xu@duke.edu
Natalie Katz, MD, PhD
- Recruiting
University of California, Irvine (UCI)
Irvine, California, 92697
UCI Alpha Clinic, 949-824-3990, alphaclinic@hs.uci.edu
Tahseen Mozaffar, MD
- Not yet recruiting
NYU Langone
New York, New York, 10016
Nicolas Abreu, MD
- Not yet recruiting
Stanford Neuroscience Health Center
Palo Alto, California, 94304
John Day, MD, PhD
- Recruiting
Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Amanda Hicks, 267-496-3772, Amanda.Hicks@pennmedicine.upenn.edu
Abigail Browngoehl, Abigail.Browngoehl@Pennmedicine.upenn.edu
- Not yet recruiting
Barrow Neurological Institute
Phoenix, Arizona, 85013
Shafeeq Ladha, MD
- Recruiting
University of Pittsburgh Medical Center (UPMC)
Pittsburgh, Pennsylvania, 15213
Gabriela A Niizawa, 412-383-9775, niizawaga@upmc.edu
Paula Clemens, MD
- Not yet recruiting
Virginia Commonwealth University (VCU)
Richmond, Virginia, 23298
Susan Sparks, MD, PhD
About this study
Official title: 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)
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).
Read the detailed description
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.
- Conditions
- Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
- Being tested
- AB-1009 (GAA Gene)
- Keywords
- Pompe Disease, Glycogen Storage Disease, Lysosomal Storage Diseases, Acid Maltase Deficiency, Acid Maltase Deficiency Disease, Gene Therapy, AB-1009, Neuromuscular Disease, LOPD, GAA gene, Adeno-Associated Virus (AAV), Late-Onset Pompe Disease, Acid Alpha-Glucosidase (GAA)
- Medical subject headings
- Glycogen Storage Disease Type II, Glycogen Storage Disease, Lysosomal Storage Diseases, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AB-1009 (GAA Gene)Genetic
A single intravenous infusion of AB-1009
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 1.0E13 vg/kg Receives: Genetic: AB-1009 (GAA Gene) |
| Cohort 2 | Experimental | 1.5E13 vg/kg Receives: Genetic: AB-1009 (GAA Gene) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the primary observation period
Measured: Day 1 (Dosing) through Week 52 (the end of the primary observation period)
Secondary outcomes (13)
Area under the curve (AUC) of GAA activity in serum
Measured: Baseline through Week 52
Viral shedding (whole blood, saliva, urine)
Measured: Day 1 through Week 24 (or until 3 consecutive data points are at or below the limit of detection)
AUC of urinary Glc4
Measured: Baseline during the primary observation period (through Week 52)
Change from baseline in GAA activity in muscle biopsy tissue at Week 52
Measured: Baseline and Week 52
Change from baseline in muscle biopsy glycogen content at Week 52
Measured: Baseline and Week 52
Change from baseline in forced vital capacity (FVC) (% predicted) at Week 24 and Week 52
Measured: Baseline, Week 24, and Week 52
Change from baseline in maximum inspiratory pressure (MIP) at Week 24 and Week 52
Measured: Baseline, Week 24, and Week 52
Change from baseline in maximum expiratory pressure (MEP) at Week 24 and Week 52
Measured: Baseline, Week 24, and Week 52
Change from baseline in distance walked in the 6-Minute Walk Test (6MWT) at Week 24 and Week 52
Measured: Baseline, Week 24, and Week 52
Patient's Global Impression of Change (PGI-C) at Week 24 and Week 52
Score range: 1-7; lower scores indicate greater improvement and a better outcome.
Measured: Week 24 and Week 52
Clinical Global Impression of Change (CGI-C) at Week 24 and Week 52
Score range: 1-7; lower scores indicate greater improvement and a better outcome
Measured: Week 24 and Week 52
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) for physical function changes at Week 24 and Week 52
PROMIS Short Form v2.0- Physical Function 20a consists of 20 questions that are scored on a scale of 1 to 5. The total score (i.e. sum) ranges between 20 and 100, with a higher score indicating better physical functioning.
Measured: Baseline, Week 24, and Week 52
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) for fatigue changes at Week 24 and Week 52
PROMIS Short Form v1.0- Fatigue 8a consists of eight questions, scored on a scale of 1 to 5. The total score (i.e. sum) ranges between 8 and 40, with lower scores indicating less fatigue.
Measured: Baseline, Week 24, and Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2025
- Study start15 Apr 2026
- Primary completionSep 2028 (estimated)
- Study completionSep 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe Disease (Late-onset)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AskBio Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Bayer
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07282847
- Sponsor's protocol ID
- ASK-POM9-CS101
- Status last verified
- Jul 2026
- First submitted
- 26 Nov 2025
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07282847
- Acronym / sponsor ID
- ASK-POM9-CS101
- Conditions
- Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
- Keywords
- Pompe Disease, Glycogen Storage Disease, Lysosomal Storage Diseases, Acid Maltase Deficiency, Acid Maltase Deficiency Disease, Gene Therapy, AB-1009, Neuromuscular Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- AskBio IncIndustry (pharma, biotech, devices)
- Collaborators
- Bayer
- Study start
- 15 Apr 2026 (actual)
- Primary completion
- Sep 2028 (estimated)
- Study completion
- Sep 2032 (estimated)
- First posted
- 15 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Genetic: AB-1009 (GAA Gene)1.0E13 vg/kg |
| Cohort 2 | Experimental | Genetic: AB-1009 (GAA Gene)1.5E13 vg/kg |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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);
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AB-1009 (GAA Gene)A single intravenous infusion of AB-1009 | Genetic | Cohort 1, Cohort 2 |
Primary outcome
Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the primary observation period
Time frame: Day 1 (Dosing) through Week 52 (the end of the primary observation period)
13 secondary outcomes
Area under the curve (AUC) of GAA activity in serum
Time frame: Baseline through Week 52
Viral shedding (whole blood, saliva, urine)
Time frame: Day 1 through Week 24 (or until 3 consecutive data points are at or below the limit of detection)
AUC of urinary Glc4
Time frame: Baseline during the primary observation period (through Week 52)
Change from baseline in GAA activity in muscle biopsy tissue at Week 52
Time frame: Baseline and Week 52
Change from baseline in muscle biopsy glycogen content at Week 52
Time frame: Baseline and Week 52
Change from baseline in forced vital capacity (FVC) (% predicted) at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Change from baseline in maximum inspiratory pressure (MIP) at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Change from baseline in maximum expiratory pressure (MEP) at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Change from baseline in distance walked in the 6-Minute Walk Test (6MWT) at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Patient's Global Impression of Change (PGI-C) at Week 24 and Week 52
Time frame: Week 24 and Week 52
Clinical Global Impression of Change (CGI-C) at Week 24 and Week 52
Time frame: Week 24 and Week 52
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) for physical function changes at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) for fatigue changes at Week 24 and Week 52
Time frame: Baseline, Week 24, and Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Pompe Disease (Late-onset)
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingObservational study
Pompe Disease Registry Protocol
Glycogen Storage Disease Type II, Pompe Disease
Not yet recruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Pompe Disease
Active, not recruiting
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Pompe Disease (Late-onset)
Active, not recruitingPhase 3
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Pompe Disease (Late-onset)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AB-1009 (GAA Gene) in people with Pompe Disease (Late-onset) and Pompe Disease Late-Onset, run by AskBio Inc at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact AskFirst Patient Engagement (919-561-6210, AskFirst@AskBio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the primary observation period (time frame: Day 1 (Dosing) through Week 52 (the end of the primary observation period)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2026.