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First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02675465
First Posted: February 5, 2016
Last Update Posted: November 6, 2017
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. Read our disclaimer for details.
Information provided by (Responsible Party):
Amicus Therapeutics
January 26, 2016
February 5, 2016
November 6, 2017
January 2016
June 2019   (Final data collection date for primary outcome measure)
  • Plasma GAA activity levels as measured by maximum observed plasma concentration (Cmax). [ Time Frame: 18 Weeks ]
  • Plasma GAA activity levels as measured by time to reach the maximum observed plasma concentration (tmax). [ Time Frame: 18 Weeks ]
  • Plasma GAA activity levels as measured by area under the plasma-drug concentration time curve. [ Time Frame: 18 Weeks ]
  • Safety and tolerability as measured by counts of Treatment Emergent Adverse Events (TEAEs), including Infusion Associated Reactions (IARs). [ Time Frame: 18 weeks ]
Same as current
Complete list of historical versions of study NCT02675465 on ClinicalTrials.gov Archive Site
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First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
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
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.

This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the effect of a highly targeted rhGAA (ATB200) co-administered with a chaperone (AT2221).

The study aims to evaluate safety, tolerability, pharmacodynamics (PD), and immunogenicity of ATB200 co-administered with AT2221. The study will be conducted in 3 stages.

In Stage 1, safety, tolerability, and PK will be evaluated following sequential single ascending doses of intravenously infused ATB200.

In Stage 2, safety, tolerability, and PK will be evaluated following single- and multiple-ascending dose combinations of ATB200 and AT2221.

In Stage 3, long term safety and efficacy will be assessed following 24 month treatment of ATB200 co-administered with AT2221 (Miglustat)

No Muscle biopsies will be performed in this study.

Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Pompe Disease
  • Drug: ATB200
  • Drug: AT2221
    Other Name: Miglustat
  • 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.
    Intervention: Drug: ATB200
  • 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)
    Interventions:
    • Drug: ATB200
    • Drug: AT2221
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
20
June 2019
June 2019   (Final data collection date for primary outcome measure)

Key Inclusion Criteria:

  • Male and female subjects between 18 and 65 years 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

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
Sexes Eligible for Study: All
18 Years to 65 Years   (Adult)
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Germany,   Netherlands,   United Kingdom,   United States
 
 
NCT02675465
ATB200-02
No
Not Provided
Not Provided
Amicus Therapeutics
Amicus Therapeutics
Not Provided
Not Provided
Amicus Therapeutics
June 2017

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