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ZIP Study - A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects Aged 12 to <18

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: NCT03911505
Recruitment Status : Recruiting
First Posted : April 11, 2019
Last Update Posted : May 1, 2020
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Tracking Information
First Submitted Date  ICMJE April 9, 2019
First Posted Date  ICMJE April 11, 2019
Last Update Posted Date May 1, 2020
Actual Study Start Date  ICMJE February 13, 2020
Estimated Primary Completion Date September 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 9, 2019)
  • Assessment of pharmacokinetic parameters [ Time Frame: 52 weeks ]
    ATB200 protein and AT2221 concentrations in plasma
  • Number of participants with adverse events [ Time Frame: 52 weeks ]
    incidence of treatment-emergent AEs, SAEs, infusion-associated reactions, and AEs leading to discontinuation of study drug
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 30, 2020)
  • Change from baseline 6-Minute Walk Test [6MWT] [ Time Frame: 52 weeks ]
  • Change from baseline Gait, Stair, Gower, and Chair maneuver [GSGC] test [ Time Frame: 52 weeks ]
  • Change from baseline Timed Up and Go [TUG] test) [ Time Frame: 52 weeks ]
  • Change from baseline manual muscle tests [MMT]) test) [ Time Frame: 52 weeks ]
  • Change from baseline forced vital capacity [FVC] [ Time Frame: 52 weeks ]
  • Change from baseline supine and sitting, slow vital capacity [SVC] [ Time Frame: 52 weeks ]
  • Change from baseline supine and sitting, maximal inspiratory pressure [MIP] [ Time Frame: 52 weeks ]
  • Change from baseline maximal expiratory pressure [MEP] [ Time Frame: 52 weeks ]
  • Change from baseline Patient-reported Outcomes Measurement Information System (PROMIS®) [ Time Frame: 52 weeks ]
  • Change from baseline Gross Motor Function Measure-88 Items (GMFM-88) [ Time Frame: 52 weeks ]
  • Change from baseline NeuroQOL Lower Extremity Function (Mobility) [ Time Frame: 52 weeks ]
  • Change from baseline Subject/Physician Global Impression of Change (SGIC/PGIC) [ Time Frame: 52 weeks ]
  • Change from baseline time to initiation of use of assistive device Scale (VAS) for pain assessment [ Time Frame: 52 weeks ]
  • Biomarkers/Pharmacodynamics of muscle injury and disease substrate [ Time Frame: 52 weeks ]
    Change from baseline in Creatine Kinase and Urinary Hexose Tetrasaccharide
  • Immunogenicity [ Time Frame: 52 weeks ]
    Change in anti-rhGAA antibodies from baseline over time
Original Secondary Outcome Measures  ICMJE
 (submitted: April 9, 2019)
  • Change from baseline 6-Minute Walk Test [6MWT] [ Time Frame: 52 weeks ]
  • Change from baseline Gait, Stair, Gower, and Chair maneuver [GSGC] test [ Time Frame: 52 weeks ]
  • Change from baseline Timed Up and Go [TUG] test) [ Time Frame: 52 weeks ]
  • Change from baseline manual muscle tests [MMT]) test) [ Time Frame: 52 weeks ]
  • Change from baseline forced vital capacity [FVC] [ Time Frame: 52 weeks ]
  • Change from baseline supine and sitting, slow vital capacity [SVC] [ Time Frame: 52 weeks ]
  • Change from baseline supine and sitting, maximal inspiratory pressure [MIP] [ Time Frame: 52 weeks ]
  • Change from baseline maximal expiratory pressure [MEP] [ Time Frame: 52 weeks ]
  • Change from baseline Patient-reported Outcomes Measurement Information System (PROMIS®) [ Time Frame: 52 weeks ]
  • Change from baseline Gross Motor Function Measure-88 Items (GMFM-88) [ Time Frame: 52 weeks ]
  • Change from baseline Pompe-pediatric Evaluation of Disability Inventory (PompePedi) [ Time Frame: 52 weeks ]
  • Change from baseline Subject/Physician Global Impression of Change (SGIC/PGIC) [ Time Frame: 52 weeks ]
  • Change from baseline Visual Analog Scale (VAS) for pain assessment [ Time Frame: 52 weeks ]
  • Change from baseline time to initiation of use of assistive device Scale (VAS) for pain assessment [ Time Frame: 52 weeks ]
  • Biomarkers/Pharmacodynamics of muscle injury and disease substrate [ Time Frame: 52 weeks ]
    Change from baseline in Creatine Kinase and Urinary Hexose Tetrasaccharide
  • Immunogenicity [ Time Frame: 52 weeks ]
    Change in anti-rhGAA antibodies from baseline over time
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE ZIP Study - A Study to Evaluate the PK, Safety, Efficacy, and PD With ATB200/AT2221 in LOPD Subjects Aged 12 to <18
Official Title  ICMJE An Open-label Study of the Pharmacokinetics, Safety, Efficacy, and Pharmacodynamics of ATB200/AT2221 in Pediatric Subjects Aged 12 to < 18 Years With Late-onset Pompe Disease
Brief Summary

This is a Phase 3, open-label, uncontrolled, multicenter study to evaluate the PK, safety, efficacy, and PD of ATB200/AT2221 treatment in pediatric subjects aged 12 to < 18 years with LOPD.

Enzyme replacement therapy (ERT)-experienced subjects are those who have received at least 1 dose of alglucosidase alfa prior to enrolling in this study.

Detailed Description

The study will consist of a 30-day screening period, a 12-month treatment period, and a 30-day safety follow-up period, for a total duration of approximately 14 months. Subjects who complete this study may have an opportunity to enroll in a separate long-term extension study.

Pediatric subjects will be treated every other week with oral AT2221 followed by ATB200 IV. Subjects will undergo PK assessments at Day 1, Week 26, and Week 52.

Safety assessments include monitoring of adverse events, clinical laboratory tests, physical examinations, vital signs, echocardiograms, 12-lead electrocardiogram (ECG), and detection of ATB200 antibodies. Efficacy assessments include evaluation of ambulatory function (6-Minute Walk Test [6MWT]); motor function tests; muscle strength; pulmonary function tests; Patient-reported Outcomes Measurement Information System (PROMIS®) for dyspnea, fatigue, physical functioning, and upper extremity; Gross Motor Function Measure-88 Items (GMFM-88); Pompe-pediatric Evaluation of Disability Inventory (PompePedi); Subject/Physician Global Impression of Change (SGIC/PGIC); Visual Analog Scale (VAS) for pain assessment, and time to initiation of use of assistive device. The patient-reported outcomes are to be completed if available. Pharmacodynamic (PD) assessments include measurement of serum creatine kinase (CK) levels and urinary hexose tetrasaccharide (Hex4) levels.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pompe Disease (Late-onset)
Intervention  ICMJE
  • Biological: ATB200
    Enzyme Replacement Therapy via intravenous infusion
  • Drug: AT2221
    Participants received ATB200 co-administered with AT2221 (Miglustat)
    Other Name: Miglustat
Study Arms  ICMJE Experimental: ATB200/AT2221
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
Interventions:
  • Biological: ATB200
  • Drug: AT2221
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 9, 2019)
14
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 2021
Estimated Primary Completion Date September 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Male or female subjects (ERT-naïve or ERT-experienced), diagnosed with late-onset Pompe disease who are aged 12 to < 18 years at screening
  2. Subject weighs ≥ 25 kg and ≤ 115 kg
  3. Subject's parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable based on site and local regulations
  4. Subject must have a diagnosis of LOPD
  5. Subject has a sitting FVC ≥ 30% of the predicted value for healthy adolescents (Global Lung Function Initiative [GLI]) at screening
  6. Subject performs two 6MWTs at screening that are valid, as determined by the clinical evaluator, and that meet all of the following criteria:

    1. both screening values of 6-Minute Walk Distance (6MWD) are ≥ 75 meters
    2. the lower value of 6MWD is within 20% of the higher value of 6MWD

Exclusion Criteria:

  1. Subject has received any investigational/experimental drug, biologic or device within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening
  2. Subject has received any gene therapy at any time
  3. Female subject is pregnant or breast-feeding at screening
  4. Subject requires the use of ventilation support for > 6 hours per day while awake
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: For Site 609-662-2000 PompeSiteInfo@amicusrx.com
Contact: For Patient 609-662-2000 patientadvocacy@amicusrx.com
Listed Location Countries  ICMJE Canada,   Taiwan,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03911505
Other Study ID Numbers  ICMJE ATB200-04
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Amicus Therapeutics
Study Sponsor  ICMJE Amicus Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Amicus Therapeutics
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP