For Healthcare Professionals

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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About the study

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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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

  1. Male and female subjects between 18 and 75years of age, inclusive
  2. Diagnosis of Pompe disease Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
  3. Has received ERT with alglucosidase alfa for the previous 2-6 years, inclusive
  4. Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week
  5. Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
  6. Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value ERT-experienced subjects (non-ambulatory):
  7. Has received ERT with alglucosidase alfa (Myozyme/Lumizyme) for ≥2 years
  8. Is wheelchair-bound ERT-naïve subjects (ambulatory):
  9. Must be able to walk 200-500 meters on the 6MWT
  10. Has upright FVC must be 30% to 80% of predicted normal value
  11. Subject has never received alglucosidase alfa Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):
  12. Has received ERT with alglucosidase alfa for >7years, inclusive
  13. Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week
  14. Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
  15. Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

EXCLUSION CRITERIA

  1. Subject has received treatment with prohibited medications within 30 days of Baseline Visit
  2. Subject, if female, is pregnant or breastfeeding at screening
  3. Subject, whether male or female, planning to conceive a child during the study
  4. 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
  5. Subject has a history of allergy or sensitivity to miglustat or other iminosugars
  6. 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
  7. Subjects with active bronchial asthma. All subjects with bronchial asthma must be discussed with the Amicus Medical Monitor
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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.
Phone iconCall 609-662-2000Email iconEmail Study Center

Study’s details


Contition

Pompe Disease

Age

18+

Phase

Phase 1/Phase 2

Participants needed

32

Est. Completion Date

Dec 2023

Treatment type

Interventional


Sponsor

Amicus Therapeutics

ClinicalTrials.gov identifier

NCT02675465

Study number

ATB200-02

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