Clinical trial • Phase III • Neurology|Rare Disease
MIGLUSTAT for Late-onset Pompe disease
Phase III trial of MIGLUSTAT for Late-onset Pompe disease. open-label. 85 participants.
Overview
- Trial Therapeutic Area
- Neurology|Rare Disease
- Trial Disease
- Late-onset Pompe disease
- Trial Stage
- Phase III
- Drug Modality
- Peptide/protein/enzyme|Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 17-06-2024
- First CTIS Authorization Date
- 12-07-2024
Trial design
open-label Phase III trial in Greece, Netherlands, Hungary and others.
- Open Label
- Yes
- Target Sample Size
- 85
Eligibility
Recruits 85 Subjects must provide signed informed consent prior to any study-related procedures. If the subject is under 20 years of age, the subject must provide written informed consent. In Japan the subject's caregiver (or legal representative) must also sign the informed consent form (as noted in the protocol translations)..
- Pregnancy Exclusion
- Subject, if female, is pregnant or breastfeeding
- Vulnerable Population
- Subjects must provide signed informed consent prior to any study-related procedures. If the subject is under 20 years of age, the subject must provide written informed consent. In Japan the subject's caregiver (or legal representative) must also sign the informed consent form (as noted in the protocol translations).
Inclusion criteria
- {"criterion_text":"- Subject must provide signed informed consent prior to any study- related procedures being performed. If the subject is under 20 years of age, the subject must provide written informed consent"}
- {"criterion_text":"- Subject must have completed Study ATB200-03. Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of ATB200/AT2221 (eg, hospitalization for a car accident, COVID-19 pandemic, or emergency surgery) and which resulted in several consecutive missed doses may be eligible to participate in this study upon approval by the Amicus medical monitor"}
- {"criterion_text":"- Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug"}
Exclusion criteria
- {"criterion_text":"- Subject plans to receive gene therapy or participate in another interventional study for Pompe disease"}
- {"criterion_text":"- Subject has a hypersensitivity to any of the excipients in ATB200 or AT2221, or has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms"}
- {"criterion_text":"- Subject, if female, is pregnant or breastfeeding"}
- {"criterion_text":"- Subject, whether male or female, is planning to conceive a child during the study"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Long-term safety: incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug, frequency and severity of immediate and late IARs, and any abnormalities noted in other safety assessments (eg, clinical laboratory tests, ECGs, vital signs). Immunogenicity to ATB200 will also be described","definition_or_measurement_approach":"Assessment by incidence counts of TEAEs, SAEs and AEs leading to discontinuation, frequency and severity grading of immediate and late infusion-associated reactions (IARs); abnormalities in clinical labs, ECGs, vital signs; immunogenicity described (anti‑drug antibody/immunogenicity assessments)"}
Secondary endpoints
- {"endpoint_text":"- Change from baseline in 6-minute walk distance (6MWD)","definition_or_measurement_approach":"Change from baseline measured as difference in 6-minute walk distance (meters)"}
- {"endpoint_text":"- Change from baseline in 6MWD (% predicted)","definition_or_measurement_approach":"Change from baseline in 6MWD expressed as percent predicted"}
- {"endpoint_text":"- Change from baseline in sitting FVC (% predicted)","definition_or_measurement_approach":"Change from baseline in sitting forced vital capacity expressed as % predicted"}
- {"endpoint_text":"- Change from baseline in the manual muscle test score for the lower extremities","definition_or_measurement_approach":"Change from baseline in manual muscle test score for lower extremities"}
- {"endpoint_text":"- Change from baseline in the total score for the PROMIS – physical function","definition_or_measurement_approach":"Change from baseline in PROMIS physical function total score (patient-reported outcome)"}
- {"endpoint_text":"- Change from baseline in the total score for the PROMIS – fatigue","definition_or_measurement_approach":"Change from baseline in PROMIS fatigue total score (patient-reported outcome)"}
- {"endpoint_text":"- Change from baseline in the following variables related to motor function: - GSGC total score, - time to complete the 10-meter walk (ie, assessment of gait) of the GSGC test, - time to complete the 4-stair climb of the GSGC test, - time to complete the Gower's maneuver of the GSGC test, - time to arise from a chair as part of the GSGC test, - change from baseline in the time to complete the TUG test","definition_or_measurement_approach":"Change from baseline in listed GSGC components and TUG test times (motor function assessments)"}
- {"endpoint_text":"- Change from baseline in the following variables related to muscle strength: - manual muscle test score for the upper extremities, - manual muscle test total score (upper and lower extremities combined), - quantitative muscle test value (kg) for the upper extremities, - quantitative muscle test value (kg) for the lower extremities, - quantitative muscle test total value (kg) (upper and lower extremities combined)","definition_or_measurement_approach":"Change from baseline in manual muscle test scores and quantitative muscle test values (kg)"}
- {"endpoint_text":"- Change from baseline in the following variables from patient-reported outcome measures: - total score for the PROMIS-dyspnea, - total score for the PROMIS–upper extremity, - R-PAct Scale total score, - EQ-5D-5L health status","definition_or_measurement_approach":"Change from baseline in listed patient-reported outcome measures (PROMIS scales, R-PAct, EQ-5D-5L)"}
- {"endpoint_text":"- Actual value of the subject's functional status (improving, stable, or declining) pertaining to the effects of study drug in the following areas of life, as measured by the SGIC: - overall physical well-being, - effort of breathing, - muscle strength, - muscle function, - ability to move around, - activities of daily living, - energy level, - level of muscular pain","definition_or_measurement_approach":"SGIC (Subject Global Impression of Change) assessment categorizing functional status as improving, stable or declining across listed domains"}
- {"endpoint_text":"- Actual value of the subject's functional status (improving, stable, or declining), as measured by the PGIC","definition_or_measurement_approach":"PGIC (Patient Global Impression of Change) assessment categorizing overall functional status"}
- {"endpoint_text":"- Change from baseline in the following measures of pulmonary function, as follows: - sitting SVC (% predicted), - MIP (cmH2O), - MIP (% predicted), - MEP (cmH2O), - MEP (% predicted), - SNIP (cmH2O)","definition_or_measurement_approach":"Change from baseline in listed pulmonary function measures (SVC, MIP, MEP, SNIP), absolute and % predicted where specified"}
- {"endpoint_text":"- Change from baseline in serum CK level","definition_or_measurement_approach":"Change from baseline in serum creatine kinase (CK) level (laboratory measurement)"}
- {"endpoint_text":"- Change from baseline in urinary Hex4 level","definition_or_measurement_approach":"Change from baseline in urinary Hex4 level (biomarker measurement)"}
Recruitment
- Planned Sample Size
- 85
- Recruitment Window Months
- 96
- Consent Approach
- Subjects must provide signed informed consent prior to any study-related procedures. If the subject is under 20 years of age, the subject must provide written informed consent. In Japan the subject's caregiver (or legal representative) must also sign the informed consent form. Subject information and ICF documents are available in multiple languages (protocol and ICF files exist for EN, FR, DU/NL, HU, IT, PL, SI as indicated in the document list).
Geography
- Total Number Of Sites
- 13
- Total Number Of Participants
- 34
Greece
- Earliest CTIS Part Ii Submission Date
- 19-07-2024
- Latest Decision Or Authorization Date
- 19-07-2024
- Processing Time Days
- 0
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Eginitio Hospital
- Department Name
- 1st Department of Neurology
- Principal Investigator Name
- George-Konstantinos Papadimas
- Principal Investigator Email
- gkpapad@yahoo.gr
- Contact Person Name
- George-Konstantinos Papadimas
- Contact Person Email
- gkpapad@yahoo.gr
Netherlands
- Earliest CTIS Part Ii Submission Date
- 18-07-2024
- Latest Decision Or Authorization Date
- 18-07-2024
- Processing Time Days
- 0
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
- Department Name
- Department of Pediatrics
- Principal Investigator Name
- Ans van der Ploeg
- Principal Investigator Email
- a.vanderploeg@erasmusmc.nl
- Contact Person Name
- Ans van der Ploeg
- Contact Person Email
- a.vanderploeg@erasmusmc.nl
Hungary
- Earliest CTIS Part Ii Submission Date
- 18-07-2024
- Latest Decision Or Authorization Date
- 18-07-2024
- Processing Time Days
- 0
- Number Of Sites
- 3
- Number Of Participants
- 7
Sites
- Site Name
- University Of Pecs
- Department Name
- Neurology
- Principal Investigator Name
- Janszky József
- Principal Investigator Email
- hilbert.helga@pte.hu
- Contact Person Name
- Janszky József
- Contact Person Email
- hilbert.helga@pte.hu
- Site Name
- Semmelweis University
- Department Name
- Institute of Genomic Medicine and Rare Disorders
- Principal Investigator Name
- Molnár Mária Judit
- Principal Investigator Email
- molnar.mariajudit@med.semmelweis-univ.hu
- Contact Person Name
- Molnár Mária Judit
- Contact Person Email
- molnar.mariajudit@med.semmelweis-univ.hu
- Site Name
- University Of Szeged
- Department Name
- Neurology Clinic
- Principal Investigator Name
- László Vécsei
- Principal Investigator Email
- vecsei.laszlo@med.u-szeged-hu
- Contact Person Name
- László Vécsei
- Contact Person Email
- vecsei.laszlo@med.u-szeged-hu
Poland
- Earliest CTIS Part Ii Submission Date
- 06-08-2024
- Latest Decision Or Authorization Date
- 06-08-2024
- Processing Time Days
- 0
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Centrum Medyczne Medyk Sp. z o.o.
- Department Name
- Neurology
- Principal Investigator Name
- Halina Bartosik-Psujek
- Principal Investigator Email
- badaniakliniczne.rejtana@medyk.rzeszow.pl
- Contact Person Name
- Halina Bartosik-Psujek
- Contact Person Email
- badaniakliniczne.rejtana@medyk.rzeszow.pl
Belgium
- Earliest CTIS Part Ii Submission Date
- 17-07-2024
- Latest Decision Or Authorization Date
- 17-07-2024
- Processing Time Days
- 0
- Number Of Sites
- 1
- Number Of Participants
- 3
Sites
- Site Name
- UZ Leuven
- Department Name
- Neurology
- Principal Investigator Name
- Kristl Claeys
- Principal Investigator Email
- kristl.claeys@uzleuven.be
- Contact Person Name
- Kristl Claeys
- Contact Person Email
- kristl.claeys@uzleuven.be
France
- Earliest CTIS Part Ii Submission Date
- 17-07-2024
- Latest Decision Or Authorization Date
- 17-07-2024
- Processing Time Days
- 0
- Number Of Sites
- 3
- Number Of Participants
- 11
Sites
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Neurology
- Principal Investigator Name
- Céline TARD
- Principal Investigator Email
- celine.tard@chru-lille.fr
- Contact Person Name
- Céline TARD
- Contact Person Email
- celine.tard@chru-lille.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- ENMG and neuromuscular pathology
- Principal Investigator Name
- Françoise BOUHOUR
- Principal Investigator Email
- francoise.bouhour@chu-lyon.fr
- Contact Person Name
- Françoise BOUHOUR
- Contact Person Email
- francoise.bouhour@chu-lyon.fr
- Site Name
- Raymond-Poincare Hospital
- Department Name
- Neurology
- Principal Investigator Name
- Pascal LAFORET
- Principal Investigator Email
- pascal.laforet@aphp.fr
- Contact Person Name
- Pascal LAFORET
- Contact Person Email
- pascal.laforet@aphp.fr
Slovenia
- Earliest CTIS Part Ii Submission Date
- 18-07-2024
- Latest Decision Or Authorization Date
- 18-07-2024
- Processing Time Days
- 0
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- University Medical Center Ljubljana
- Department Name
- Neurophysiology
- Principal Investigator Name
- Blaž Koritnik
- Principal Investigator Email
- blaz.koritnik@kclj.si
- Contact Person Name
- Blaž Koritnik
- Contact Person Email
- blaz.koritnik@kclj.si
Italy
- Earliest CTIS Part Ii Submission Date
- 24-07-2024
- Latest Decision Or Authorization Date
- 24-07-2024
- Processing Time Days
- 0
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- Universita' Degli Studi Di Napoli Federico II
- Department Name
- Dipartimento di Pediatria
- Principal Investigator Name
- Giancarlo Parenti
- Principal Investigator Email
- parenti@unina.it
- Contact Person Name
- Giancarlo Parenti
- Contact Person Email
- parenti@unina.it
- Site Name
- Azienda Ospedaliera Universitaria Gaetano Martino Messina
- Department Name
- UOC di Neurologia e Malattie Neuromuscolari
- Principal Investigator Name
- Antonio Toscano
- Principal Investigator Email
- antonio.toscano@unime.it
- Contact Person Name
- Antonio Toscano
- Contact Person Email
- antonio.toscano@unime.it
Denmark
- Earliest CTIS Part Ii Submission Date
- 12-07-2024
- Latest Decision Or Authorization Date
- 12-07-2024
- Processing Time Days
- 0
- Number Of Sites
- 1
- Number Of Participants
- 6
Sites
- Site Name
- Aarhus Universitetshospital
- Department Name
- Neurologisk klinik
- Principal Investigator Name
- Henning Andersen
- Principal Investigator Email
- hennande@rm.dk
- Contact Person Name
- Henning Andersen
- Contact Person Email
- hennande@rm.dk
Sponsor
Primary sponsor
- Full Name
- Amicus Therapeutics Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Iqvia Rds Ireland Limited
- Name
- Medpace Finland Oy
- Name
- Hungarotrial Zrt.
- Responsibilities
- Regional CRO
- Name
- Everest Clinical Research Corporation
- Responsibilities
- IXRS
- Name
- Medpace Ellas Monoprosopi I.K.E.
Third parties
- {"country":"Ireland","full_name":"Iqvia Rds Ireland Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"ATOM International Limited","duties_or_roles":"Physiotherapist training","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"eResearchTechnology GmbH","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","duties_or_roles":"Urinalysis","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"Hungary","full_name":"Hungarotrial Zrt.","duties_or_roles":"Regional CRO","organisation_type":"Educational Institution"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient travel services/Patient reimbursement","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Canada","full_name":"Everest Clinical Research Corporation","duties_or_roles":"IXRS","organisation_type":"Pharmaceutical company"}
- {"country":"Greece","full_name":"Medpace Ellas Monoprosopi I.K.E.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- AT2221
- Active Substance
- MIGLUSTAT
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- oral
- Authorisation Status
- prodAuthStatus=1
- Orphan Designation
- Yes
- Maximum Dose
- 260 mg (max daily dose)
- Investigational Product Name
- ATB200
- Active Substance
- CIPAGLUCOSIDASE ALFA
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- INTRAVENOUS
- Route
- intravenous
- Authorisation Status
- prodAuthStatus=1
- Orphan Designation
- Yes
- Maximum Dose
- 20 mg/kg (max daily dose)
- Combination Treatment
- Yes
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