Pompe DiseasePhase 1/2NCT02675465

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

A Phase 1/2 study testing ATB200 and AT2221 in people with Pompe Disease, run by Amicus Therapeutics at 19 sites in 6 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease. Read more about this study

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Pompe Disease
What is being tested?: ATB200 and AT2221
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 29 participants
Where: 19 sitesin 6 countries
Study dates: Apr 2016to 22 Aug 2024
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

None listed.

Who cannot join

Received treatment with prohibited medications within 30 days of Baseline Visit
Subject, if female, was pregnant or breastfeeding at screening
Subject, whether male or female, planned to conceive a child during the study
Had a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements
Had a history of allergy or sensitivity to alglucosidase alfa, miglustat or other iminosugars (Cohorts 1, 2, and 4)
Required invasive ventilatory support, or used noninvasive ventilatory support ≥ 6 hours a day while awake (Cohorts 1, 3, and 4)
Had active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis; subjects with autoimmune disease must have been discussed with the Amicus Medical Monitor
Had active bronchial asthma; subjects with bronchial asthma must have been discussed with the Amicus Medical Monitor

Trial locations and contacts

19 sites in 6 countries.

Country

City, postcode or hospital

19 sites in 6 countries

United States12 sites

Emory University Division of Medical Genetics
Decatur, Georgia, 30033
Duke University Medical Center
Durham, North Carolina, 27710
Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)
Fairfax, Virginia, 22030
University of Florida
Gainesville, Florida, 32610
Infusion Associates
Grand Rapids, Michigan, 49525
Great Falls Clinic, LLP
Great Falls, Montana, 59405
Rutgers New Jersey Medical School
Newark, New Jersey, 08103
University of California Irvine
Orange, California, 92868
Perelman Center for Advanced Medicine
Philadelphia, Pennsylvania, 19104
Neuromuscular Research Centre
Phoenix, Arizona, 85028
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Abramson Cancer Center Chester County Hospital
West Chester, Pennsylvania, 19380

Other trials in United States

Germany2 sites

University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital
Bochum, 44791
Friedrich-Baur-Institure, Dep of Neurology - University Munich
Munich, 80336

Other trials in Germany

United Kingdom2 sites

University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center
Birmingham, B15 2TH
Salford Royal NHS Foundation Trust
Salford, M6 8HD

Other trials in United Kingdom

Australia1 site

Womens & Childrens Hospital, Adelaide
North Adelaide, South Australia, 05006

Other trials in Australia

Netherlands1 site

Erasmus Medical Center
Rotterdam

Other trials in Netherlands

New Zealand1 site

School of Medicine, University of Auckland
Auckland, 01051

Other trials in New Zealand

About this study

Official title: An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease

This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.

Read the detailed description

This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the effect of a highly targeted rhGAA (ATB200) co-administered with an enzyme stabilizer (AT2221).

The study aims to evaluate safety, tolerability, pharmacokinetics (PK), efficacy, pharmacodynamics (PD), and immunogenicity of ATB200 co-administered with AT2221.

Stage 1: evaluation of safety, tolerability, and PK following sequential single ascending doses of intravenously infused ATB200

Stage 2: evaluation of safety, tolerability, and PK following single- and multiple-ascending dose combinations of ATB200 and AT2221

Stage 3: evaluation of long term safety, tolerability, and efficacy following 24 month treatment of ATB200 co-administered with AT2221

Stage 4: open-label extension period with functional assessments every 6 months

Conditions: Pompe Disease
Being tested: ATB200, AT2221
Keywords: Pompe, rhGAA
Medical subject headings: Glycogen Storage Disease Type II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ATB200Drug
Also called Cipaglucosidase alfa
AT2221Drug
Also called Miglustat

Study groups in NCT02675465
Study group	Role	What they receive
ATB200	Experimental	Sequential single ascending doses of intravenously infused ATB200 for 3 dosing periods Receives: Drug: ATB200
ATB200 + AT2221	Experimental	ATB200 co-administered with AT2221 (Miglustat) Receives: Drug: ATB200; Drug: AT2221

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug
Number of subjects with TEAE, TESAE, and AE leading to discontinuation during the 2 year treatment period and extension (Stage 3 and 4 combined)
Measured: Stage 3 (2 year treatment) and Stage 4 (Extension) combined, (mean = 71 months on treatment)
Plasma Human Acid α-glucosidase (GAA) Activity Levels as Measured by Maximum Observed Plasma Concentration (Cmax).
Plasma GAA levels (Cmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat
Measured: 18 Weeks
Plasma GAA Activity Levels as Measured by Time to Reach the Maximum Observed Plasma Concentration (Tmax).
Plasma GAA levels (Tmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat
Measured: 18 Weeks
Plasma GAA Activity Levels as Measured by Area Under the Plasma Drug Concentration-time Curve (AUC).
Plasma GAA levels (AUC) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat
Measured: 18 Weeks

Secondary outcomes (6)

Change From Baseline in 6-minute Walk Distance (6MWD)
Motor function was measured in ambulatory subjects using 6MWD (meters).
Measured: Baseline, Month 60
Change From Baseline in Pulmonary Function Tests
Pulmonary function was measured by sitting and supine % predicted forced vital capacity (FVC)
Measured: Baseline, Month 60
Change From Baseline in Muscle Strength Tests
Muscle strength was measured by total manual muscle test (MMT) score. Total MMT score ranges from 0 to 80 based on all 16 muscle groups, which are right/left shoulder abduction, right/left shoulder adduction, right/left elbow flexion, right/left elbow extension, right/left hip flexion, right/left hip abduction, right/left knee flexion, and right/left knee extension. Higher scores indicate less disease impact on muscle functions.
Measured: Baseline, Month 60
Change From Baseline in Fatigue Severity Score (FSS)
The Fatigue Severity Score (FSS) consists of 9 questions, each scored on a scale from 1 ("completely disagree") to 7 ("completely agree"). The total score ranges from 9 to 63, with higher values representing higher level of fatigue due to the disease condition.
Measured: Baseline, Month 60
Change From Baseline in Overall Physical Wellbeing (Subject's Global Impression of Change [SGIC], Question1)
The Subject's Global Impression of Change overall physical wellbeing (question 1) is scored on a 7-point rating scale. Improved = response of 5 or higher, No change = response of 4, and Declined = response of 3 or lower.
Measured: Baseline, Month 60
Change From Baseline in Overall Physical Wellbeing (Physician's Global Impression of Change [PGIC])
The Physician's Global Impression of Change overall physical wellbeing is scored on a 7-point rating scale. Improved = response of 5 or higher, No change = response of 4, and Declined = response of 3 or lower.
Measured: Baseline, Month 60

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Apr 201622 Aug 2024

First posted5 Feb 2016 (estimated)
Study startApr 2016
Primary completion22 Aug 2024
Study completion22 Aug 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Pompe DiseaseStudies 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: Single group
Masking: None (open label)
Enrolment: 29 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02675465
Sponsor's protocol ID: ATB200-02
Status last verified: Oct 2025
First submitted: 26 Jan 2016
Last updated: 23 Oct 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Andersen H, Diaz-Manera J, Goker-Alpan O, Mozaffar T, Sitaraman Das S, Fox B, Amon F, O'Brien-Prince K, Goldman M, Holdbrook F, Jain V, Byrne BJ. Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials. Ther Adv Rare Dis. 2026 Jan 31;7:26330040261416943. doi: 10.1177/26330040261416943. eCollection 2026 Jan-Dec.
PubMed 41631150
Roberts ME, Proskorovsky I, Guyot P, Shukla P, Thibault N, Hamed A, Pulikottil-Jacob R, O'Callaghan L, Pollissard L. An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease. Adv Ther. 2025 Nov;42(11):5578-5599. doi: 10.1007/s12325-025-03301-9. Epub 2025 Sep 8.
PubMed 40920287
Byrne BJ, Schoser B, Kishnani PS, Bratkovic D, Clemens PR, Goker-Alpan O, Ming X, Roberts M, Vorgerd M, Sivakumar K, van der Ploeg AT, Goldman M, Wright J, Holdbrook F, Jain V, Benjamin ER, Johnson F, Das SS, Wasfi Y, Mozaffar T. Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02). J Neurol. 2024 Apr;271(4):1787-1801. doi: 10.1007/s00415-023-12096-0. Epub 2023 Dec 6.
PubMed 38057636

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
RecruitingPhase 1/2
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)
Pompe Disease (Late-onset), Pompe Disease Late-Onset
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
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RecruitingObservational study
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All Pompe Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 23 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease
ClinicalTrials.gov ID: NCT02675465
Acronym / sponsor ID: ATB200-02
Conditions: Pompe Disease
Keywords: Pompe, rhGAA
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 29 participants (actual)
Lead sponsor: Amicus TherapeuticsIndustry (pharma, biotech, devices)
Study start: Apr 2016 (actual)
Primary completion: 22 Aug 2024 (actual)
Study completion: 22 Aug 2024 (actual)
First posted: 5 Feb 2016 (estimated)
Results: Posted 23 Oct 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.

Detailed description

This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the effect of a highly targeted rhGAA (ATB200) co-administered with an enzyme stabilizer (AT2221).

The study aims to evaluate safety, tolerability, pharmacokinetics (PK), efficacy, pharmacodynamics (PD), and immunogenicity of ATB200 co-administered with AT2221.

Stage 1: evaluation of safety, tolerability, and PK following sequential single ascending doses of intravenously infused ATB200

Stage 2: evaluation of safety, tolerability, and PK following single- and multiple-ascending dose combinations of ATB200 and AT2221

Stage 3: evaluation of long term safety, tolerability, and efficacy following 24 month treatment of ATB200 co-administered with AT2221

Stage 4: open-label extension period with functional assessments every 6 months

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
ATB200	Experimental	Drug: ATB200Sequential single ascending doses of intravenously infused ATB200 for 3 dosing periods
ATB200 + AT2221	Experimental	Drug: ATB200, Drug: AT2221ATB200 co-administered with AT2221 (Miglustat)

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (0 listed)

Key exclusion criteria (8 listed)

Received treatment with prohibited medications within 30 days of Baseline Visit
Subject, if female, was pregnant or breastfeeding at screening
Subject, whether male or female, planned to conceive a child during the study
Had a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements
Had a history of allergy or sensitivity to alglucosidase alfa, miglustat or other iminosugars (Cohorts 1, 2, and 4)

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
ATB200Also: Cipaglucosidase alfa	Drug	ATB200, ATB200 + AT2221
AT2221Also: Miglustat	Drug	ATB200 + AT2221

Primary outcome

Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug
Time frame: Stage 3 (2 year treatment) and Stage 4 (Extension) combined, (mean = 71 months on treatment)
Number of subjects with TEAE, TESAE, and AE leading to discontinuation during the 2 year treatment period and extension (Stage 3 and 4 combined)
Plasma Human Acid α-glucosidase (GAA) Activity Levels as Measured by Maximum Observed Plasma Concentration (Cmax).
Time frame: 18 Weeks
Plasma GAA levels (Cmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat
Plasma GAA Activity Levels as Measured by Time to Reach the Maximum Observed Plasma Concentration (Tmax).
Time frame: 18 Weeks
Plasma GAA levels (Tmax) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat
Plasma GAA Activity Levels as Measured by Area Under the Plasma Drug Concentration-time Curve (AUC).
Time frame: 18 Weeks
Plasma GAA levels (AUC) measured in Cohorts 1 and 3 following 1st and 3rd doses of cipaglucosidase alfa + miglustat

6 secondary outcomes

Change From Baseline in 6-minute Walk Distance (6MWD)
Time frame: Baseline, Month 60
Change From Baseline in Pulmonary Function Tests
Time frame: Baseline, Month 60
Change From Baseline in Muscle Strength Tests
Time frame: Baseline, Month 60
Change From Baseline in Fatigue Severity Score (FSS)
Time frame: Baseline, Month 60
Change From Baseline in Overall Physical Wellbeing (Subject's Global Impression of Change [SGIC], Question1)
Time frame: Baseline, Month 60
Change From Baseline in Overall Physical Wellbeing (Physician's Global Impression of Change [PGIC])
Time frame: Baseline, Month 60

Results and safety

Results posted to the registry on 23 Oct 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Cohort 1 (ERT Experienced; Ambulatory): SAD 5 to 20 mg/kg ATB200	11	11
Cohort 1 (ERT Experienced; Ambulatory) MAD 20 mg/kg ATB200 + 130 to 260 mg AT2221	0	0
Cohort 1 (ERT Experienced; Ambulatory):	0	0
Cohort 2 (ERT Experienced; Non-ambulatory): 20 mg/kg ATB200 + 260 mg AT2221	0	0
Cohort 3 (ERT Naive; Ambulatory): 20 mg/kg ATB200 + 260 mg AT2221	0	0
Cohort 4 (ERT Experienced; Ambulatory): 20 mg/kg ATB200 + 260 mg AT2221	0	0

Primary outcome: Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug

Time frame: Stage 3 (2 year treatment) and Stage 4 (Extension) combined, (mean = 71 months on treatment). Count of participants. Unit: Participants

Measure	Cohort 1 n = 11	Cohort 2 n = 6	Cohort 3 n = 6	Cohort 4 n = 6
Number of subjects with TEAEs	11	6	6	6
Number of subjects with TESAEs	7	4	4	6
Number of subjects with AEs leading to discontinuation	1	2	0	0

Primary outcome: Plasma Human Acid α-glucosidase (GAA) Activity Levels as Measured by Maximum Observed Plasma Concentration (Cmax).

Time frame: 18 Weeks. Geometric mean (Geometric Coefficient of Variation). Unit: nmol/mL/hr

Measure	Cohort 1 n = 11	Cohort 3 n = 6
Cipaglucosidase alfa 20 mg/kg + miglustat 260 mg (1st dose)	108836 (20.5)	132400 (14.3)
Cipaglucosidase alfa 20 mg/kg + miglustat 260 mg (3rd dose)	119624 (25.7)	105842 (15.4)

Population analysed: Pharmacokinetic (PK) analysis only performed in Cohort 1 and Cohort 3. One subject each in Cohort 1 and Cohort 3 did not have PK sample available following first dose.

Primary outcome: Plasma GAA Activity Levels as Measured by Time to Reach the Maximum Observed Plasma Concentration (Tmax).

Time frame: 18 Weeks. Median (Full Range). Unit: h

Measure	Cohort 1 n = 11	Cohort 3 n = 6
Cipaglucosidase alfa 20 mg/kg + miglustat 260 mg (1st Dose)	3.49 (3.47 to 4.00)	3.97 (3.47 to 4.00)
Cipaglucosidase alfa 20 mg/kg + miglustat 260 mg (3rd Dose)	3.57 (3.35 to 4.00)	3.66 (3.50 to 3.98)

Population analysed: PK analysis only performed in Cohort 1 and Cohort 3. One subject each in Cohort 1 and Cohort 3 did not have PK sample available following first dose.

Primary outcome: Plasma GAA Activity Levels as Measured by Area Under the Plasma Drug Concentration-time Curve (AUC).

Time frame: 18 Weeks. Geometric mean (Geometric Coefficient of Variation). Unit: nmol*hr/mL

Measure	Cohort 1 n = 11	Cohort 3 n = 6
Cipaglucosidase alfa 20 mg/kg + miglustat 260 mg (1st Dose)	608180 (23.7)	762484 (22.0)
Cipaglucosidase alfa 20 mg/kg + miglustat 260 mg (3rd Dose)	670754 (29.4)	638984 (18.1)

Population analysed: PK analysis only performed in Cohort 1 and Cohort 3. One subject each in Cohort 1 and Cohort 3 did not have PK sample available following first dose.

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse event data collected over duration of the study. Duration of participation varied by patient (range: 1.4 to 93.1 months).. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Cohort 1: ATB200 5 mg	0 of 11 (0%)	7 of 11 (63.6%)	0 of 11
Cohort 1: ATB 200 10 mg	0 of 11 (0%)	5 of 11 (45.5%)	0 of 11
Cohort 1: ATB200 20 mg	0 of 11 (0%)	4 of 11 (36.4%)	0 of 11
Cohort 1: ATB200 20 mg + 130/260 mg Miglustat	0 of 11 (0%)	9 of 11 (81.8%)	0 of 11
Cohort 1 ATB200 20 mg/kg + Miglustat 260 mg	7 of 11 (63.6%)	11 of 11 (100%)	0 of 11
Cohort 2 ATB200 20 mg/kg + Miglustat 260 mg	4 of 6 (66.7%)	6 of 6 (100%)	1 of 6
Cohort 3 ATB200 20 mg/kg + Miglustat 260 mg	4 of 6 (66.7%)	6 of 6 (100%)	0 of 6
Cohort 4 ATB200 20 mg/kg + Miglustat 260 mg	6 of 6 (100%)	6 of 6 (100%)	0 of 6

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 41631150Andersen H, Diaz-Manera J, Goker-Alpan O, Mozaffar T, Sitaraman Das S, Fox B, Amon F, O'Brien-Prince K, Goldman M, Holdbrook F, Jain V, Byrne BJ. Safety of home administration of cipaglucosidase alfa plus miglustat in late-onset Pompe disease: results from multiple clinical trials. Ther Adv Rare Dis. 2026 Jan 31;7:26330040261416943. doi: 10.1177/26330040261416943. eCollection 2026 Jan-Dec.
Derived from this study: PubMed 40920287Roberts ME, Proskorovsky I, Guyot P, Shukla P, Thibault N, Hamed A, Pulikottil-Jacob R, O'Callaghan L, Pollissard L. An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease. Adv Ther. 2025 Nov;42(11):5578-5599. doi: 10.1007/s12325-025-03301-9. Epub 2025 Sep 8.
Derived from this study: PubMed 38057636Byrne BJ, Schoser B, Kishnani PS, Bratkovic D, Clemens PR, Goker-Alpan O, Ming X, Roberts M, Vorgerd M, Sivakumar K, van der Ploeg AT, Goldman M, Wright J, Holdbrook F, Jain V, Benjamin ER, Johnson F, Das SS, Wasfi Y, Mozaffar T. Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02). J Neurol. 2024 Apr;271(4):1787-1801. doi: 10.1007/s00415-023-12096-0. Epub 2023 Dec 6.

Related trials for Pompe Disease

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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)
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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. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

19 sites in 6 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing ATB200 and AT2221 in people with Pompe Disease, run by Amicus Therapeutics at 19 sites in 6 countries.

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 0 inclusion and 8 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: finished, as of the sponsor's last update on 23 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States, Germany, United Kingdom, Australia, Netherlands and 1 more country. 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?

Yes. Results are posted in the registry (first posted 23 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug (time frame: Stage 3 (2 year treatment) and Stage 4 (Extension) combined, (mean = 71 months on treatment)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02675465, which also lists 3 linked publications.

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 23 Oct 2025.