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A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04138277
Recruitment Status : Recruiting
First Posted : October 24, 2019
Last Update Posted : September 30, 2020
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Brief Summary:
This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.

Condition or disease Intervention/treatment Phase
Pompe Disease (Late-onset) Drug: AT2221 Biological: ATB200 Phase 3

Detailed Description:
This is an open label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 110 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease
Actual Study Start Date : December 18, 2019
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2023


Arm Intervention/treatment
Experimental: ATB200/AT2221
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
Drug: AT2221
Participants received ATB200 co-administered with AT2221 (Miglustat)
Other Name: Miglustat

Biological: ATB200
Enzyme Replacement Therapy via intravenous infusion




Primary Outcome Measures :
  1. Proportion of participants with Treatment Emergent Adverse Events (TEAE) [ Time Frame: baseline, up to approximately 4 years ]

Secondary Outcome Measures :
  1. 6-Minute Walk Test [ Time Frame: baseline, up to approximately 4 years ]
    Change in 6MWD from baseline to assess the efficacy of ATB200/AT2221 co-administration

  2. Pulmonary Function - Forced vital capacity (FVC) [ Time Frame: baseline, up to approximately 4 years ]
    Change from baseline in FVC (sitting) to assess the efficacy of ATB200/AT2221 co-administration

  3. Change from baseline in muscle strength measured by Quantitative Muscle Strength testing [ Time Frame: baseline, up to approximately 4 years ]
  4. Change from baseline in muscle strength measured by Manual Muscle Strength testing [ Time Frame: baseline, up to approximately 4 years ]
  5. The Rasch-built Pompe-specific activity (R-PAct) questionnaires [ Time Frame: baseline, up to approximately 4 years ]
    Change in R-Pact from baseline to assess the efficacy of ATB200/AT2221 co-administration

  6. EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires [ Time Frame: baseline, up to approximately 4 years ]
    Change from baseline in scores of EQ-5D-5L questionnaire to assess the efficacy of ATB200/AT2221 co-administration.

  7. Change from baseline in scores of PROMIS - physical function questionnaire [ Time Frame: baseline, up to approximately 4 years ]
  8. Change from baseline in scores of PROMIS - fatigue questionnaire [ Time Frame: baseline, up to approximately 4 years ]
  9. Change from baseline in scores of PROMIS - dyspnea questionnaire [ Time Frame: baseline, up to approximately 4 years ]
  10. Change from baseline in scores of PROMIS - upper extremity questionnaire [ Time Frame: baseline, up to approximately 4 years ]
  11. Motor Function - Gait, Stairs, Gower, Chair (GSGC) test [ Time Frame: baseline, up to approximately 4 years ]
    Change from baseline in GSGC score to assess the efficacy of ATB200/AT2221 co-administration.

  12. Physician's Global Impression of Change [ Time Frame: baseline, up to approximately 4 years ]
    Change in the Physician's Global Impression of Change (PGIC) evaluation to assess the efficacy of ATB200/AT2221 co-administration.

  13. Subject's Global Impression of Change [ Time Frame: baseline, up to approximately 4 years ]
    Change from baseline in scores of Subject's Global Impression of Change (SGIC) questionnaire to assess the efficacy of ATB200/AT2221 co-administration.

  14. Change from baseline Biomarker -CK [ Time Frame: baseline, up to approximately 4 years ]
  15. Change from baseline Biomarker -uHex4 [ Time Frame: baseline, up to approximately 4 years ]
  16. Immunogenicity: Incidence of neutralizing [ Time Frame: baseline, up to approximately 4 years ]
  17. Immunogenicity: anti-drug antibodies [ Time Frame: baseline, up to approximately 4 years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

1. Subject must have completed Study ATB200-03.

Exclusion Criteria

  1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
  2. Subject, if female, is pregnant or breastfeeding.
  3. Subject, whether male or female, is planning to conceive a child during the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04138277


Contacts
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Contact: For Site 609-662-2000 PompeSiteInfo@amicusrx.com
Contact: For Patient 609-662-2000 patientadvocacy@amicusrx.com

Locations
Show Show 41 study locations
Sponsors and Collaborators
Amicus Therapeutics
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Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT04138277    
Other Study ID Numbers: ATB200-07
First Posted: October 24, 2019    Key Record Dates
Last Update Posted: September 30, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amicus Therapeutics:
Pompe
rhGAA
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Miglustat
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Glycoside Hydrolase Inhibitors
Hypoglycemic Agents
Physiological Effects of Drugs