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Trial record 1 of 1 for:    atb200
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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
  Purpose
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.

Condition Intervention Phase
Pompe Disease Drug: ATB200 Drug: AT2221 Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:
  • 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 ]

Enrollment: 20
Study Start Date: January 2016
Estimated Study Completion Date: June 2019
Estimated Primary Completion Date: June 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
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)
Drug: ATB200 Drug: AT2221
Other Name: Miglustat

Detailed Description:

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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02675465


Locations
United States, Arizona
Neuromuscular Research Centre
Phoenix, Arizona, United States, 85028
United States, California
University of California Irvine
Orange, California, United States, 92868
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United States, Georgia
Emory University Division of Medical Genetics
Decatur, Georgia, United States, 30033
United States, Michigan
Infusion Associates
Grand Rapids, Michigan, United States, 49525
United States, Montana
Great Falls Clinic, LLP
Great Falls, Montana, United States, 59405
United States, New Jersey
Rutgers New Jersey Medical School
Newark, New Jersey, United States, 08103
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Pennsylvania
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Virginia
O&O Alpan LLC
Fairfax, Virginia, United States, 22030
Australia, South Australia
Womens & Childrens Hospital, Adelaide
North Adelaide, South Australia, Australia, 05006
Germany
University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital
Bochum, Germany, 44791
Friedrich-Baur-Institure, Dep of Neurology - University Munich
Munich, Germany, 80336
Netherlands
Erasmus Medical Center
Rotterdam, Netherlands
United Kingdom
University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center
Birmingham, United Kingdom, B15 2TH
Salford Royal NHS Foundation Trust
Salford, United Kingdom, M6 8HD
Sponsors and Collaborators
Amicus Therapeutics
  More Information

Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT02675465     History of Changes
Other Study ID Numbers: ATB200-02
First Submitted: January 26, 2016
First Posted: February 5, 2016
Last Update Posted: November 6, 2017
Last Verified: June 2017

Keywords provided by Amicus Therapeutics:
Pompe, rhGAA

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Miglustat
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents
Glycoside Hydrolase Inhibitors
Hypoglycemic Agents
Physiological Effects of Drugs