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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02675465




Registration number
NCT02675465
Ethics application status
Date submitted
26/01/2016
Date registered
5/02/2016
Date last updated
19/09/2024

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
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
Secondary ID [1] 0 0
ATB200-02
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pompe Disease 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
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)
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Plasma GAA activity levels as measured by maximum observed plasma concentration (Cmax).
Timepoint [1] 0 0
18 Weeks
Primary outcome [2] 0 0
Plasma GAA activity levels as measured by time to reach the maximum observed plasma concentration (tmax).
Timepoint [2] 0 0
18 Weeks
Primary outcome [3] 0 0
Plasma GAA activity levels as measured by area under the plasma-drug concentration time curve.
Timepoint [3] 0 0
18 Weeks
Primary outcome [4] 0 0
Safety and tolerability as measured by counts of Treatment Emergent Adverse Events (TEAEs), including Infusion Associated Reactions (IARs).
Timepoint [4] 0 0
18 weeks

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
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
20/08/2024
Sample size
Target
Accrual to date
Final
29
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Womens & Childrens Hospital, Adelaide - North Adelaide
Recruitment postcode(s) [1] 0 0
05006 - North Adelaide
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Montana
Country [7] 0 0
United States of America
State/province [7] 0 0
New Jersey
Country [8] 0 0
United States of America
State/province [8] 0 0
North Carolina
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Virginia
Country [11] 0 0
Germany
State/province [11] 0 0
Bochum
Country [12] 0 0
Germany
State/province [12] 0 0
Munich
Country [13] 0 0
Netherlands
State/province [13] 0 0
Rotterdam
Country [14] 0 0
New Zealand
State/province [14] 0 0
Auckland
Country [15] 0 0
United Kingdom
State/province [15] 0 0
Birmingham
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Amicus Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT02675465