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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02675465
Registration number
NCT02675465
Ethics application status
Date submitted
26/01/2016
Date registered
5/02/2016
Date last updated
4/04/2024
Titles & IDs
Public title
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
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Scientific 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
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Secondary ID [1]
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ATB200-02
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Pompe Disease
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Condition category
Condition code
Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - ATB200
Treatment: Drugs - AT2221
Experimental: ATB200 - In Stage 1, safety, tolerability, and PK will be evaluated following sequential single ascending doses of intravenously infused ATB200 for 3 dosing periods.
Experimental: ATB200 + AT2221 - In Stage 2, safety, tolerability, and PK will be evaluated following single- and multiple-ascending dose combinations of ATB200 co-administered with AT2221 (Miglustat) In Stage 3, long term safety and efficacy will be assessed following 24 month treatment of ATB200 co-administered with AT2221 (Miglustat)
Treatment: Drugs: ATB200
Treatment: Drugs: AT2221
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Plasma GAA activity levels as measured by maximum observed plasma concentration (Cmax).
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Assessment method [1]
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Timepoint [1]
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18 Weeks
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Primary outcome [2]
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Plasma GAA activity levels as measured by time to reach the maximum observed plasma concentration (tmax).
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Assessment method [2]
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Timepoint [2]
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18 Weeks
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Primary outcome [3]
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Plasma GAA activity levels as measured by area under the plasma-drug concentration time curve.
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Assessment method [3]
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Timepoint [3]
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18 Weeks
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Primary outcome [4]
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Safety and tolerability as measured by counts of Treatment Emergent Adverse Events (TEAEs), including Infusion Associated Reactions (IARs).
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Assessment method [4]
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Timepoint [4]
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18 weeks
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Eligibility
Key inclusion criteria
Key
- Male and female subjects between 18 and 75years of age, inclusive
- Diagnosis of Pompe disease
Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
- Has received ERT with alglucosidase alfa for the previous 2-6 years, inclusive
- Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency
of once every other week
- Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
- Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value
ERT-experienced subjects (non-ambulatory):
- Has received ERT with alglucosidase alfa (Myozyme/Lumizyme) for =2 years
- Is wheelchair-bound
ERT-naïve subjects (ambulatory):
- Must be able to walk 200-500 meters on the 6MWT
- Has upright FVC must be 30% to 80% of predicted normal value
- Subject has never received alglucosidase alfa
Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
- Has received ERT with alglucosidase alfa for >7years, inclusive
- Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency
of once every other week
- Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
- Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value
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Minimum age
18
Years
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Maximum age
75
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Subject has received treatment with prohibited medications within 30 days of Baseline
Visit
- Subject, if female, is pregnant or breastfeeding at screening
- Subject, whether male or female, planning to conceive a child during the study
- Subject has 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
- Subject has a history of allergy or sensitivity to miglustat or other iminosugars
- Subjects with active systemic autoimmune disease such as lupus, scleroderma, or
rheumatoid arthritis. All subjects with autoimmune disease must be discussed with the
Amicus Medical Monitor
- Subjects with active bronchial asthma. All subjects with bronchial asthma must be
discussed with the Amicus Medical Monitor
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1/Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/01/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/06/2024
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Actual
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Sample size
Target
32
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
SA
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Recruitment hospital [1]
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Womens & Childrens Hospital, Adelaide - North Adelaide
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Recruitment postcode(s) [1]
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05006 - North Adelaide
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Recruitment outside Australia
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United States of America
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Arizona
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California
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Florida
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Georgia
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Michigan
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Montana
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New Jersey
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North Carolina
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Pennsylvania
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Virginia
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Germany
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Bochum
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Germany
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Munich
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Netherlands
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Rotterdam
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New Zealand
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Auckland
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United Kingdom
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Birmingham
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United Kingdom
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State/province [16]
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Salford
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Amicus Therapeutics
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This study is an international, multi-center, study of Pompe disease patients that are
currently receiving enzyme-replacement therapy (ERT). The purpose of this study is to find
out if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults
with Pompe disease.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02675465
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Address
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02675465
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