A dose finding study with intravenous administration of duvoglustat hydrochloride (AT2220) and Myozyme in Pompe patients

Phase 1

• Conditions: Pompe disease; MedDRA version: 20.0 Level: LLT Classification code 10036143 Term: Pompe's disease System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2013-002257-30-GB
• Lead Sponsor: Amicus Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-10-21
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 24

Inclusion Criteria

1.Male or female, diagnosed with Pompe disease and between 16 and 65 years of age, inclusive (where required by local authorities, the minimum age will be 18 years of age)
2.Subject has never been treated with rhGAA (rhGAA-naïve)
OR Subject has been treated with Lumizyme (rhGAA-experienced) for at least 12 months, AND for at least the 12 weeks before the Screening Visit, has been consistently treated with Lumizyme as per its local commercial labeling (or as per local regulations, with prior approval of the sponsor), including being on a stable dose, with a stable infusion duration (between 3 and 5 hours) and biweekly schedule (stable defined as not varying by more than ± 10%)
For subjects who fall outside of these criteria, entry into the clinical trial is at the sponsor’s discretion
3.Subject, whether male or female, agrees to use medically accepted methods of contraception during the study and for 30 days after their last administration of duvoglustat (unless subject is not of childbearing potential)
4.Subject is willing and able to provide written informed consent and, as applicable, assent. Subjects under 18 years of age will provide written informed assent, and written informed consent will be provided by their parent or legal guardian
5.Subject is able to comply with all study procedures
Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 21
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

1.Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 weeks before the Screening Visit
2.Subject has clinically significant unstable cardiac disease (ie, any cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure)
3.Subject requires any invasive mechanical ventilation
4.Subject is restricted to a wheelchair or is otherwise not fully ambulatory; subjects who require wheelchair assistance or walkers under special circumstances may be eligible at the discretion of the sponsor
5.Subject has a history of allergy or sensitivity to study drug (including excipients) or other iminosugars (eg, miglitol, miglustat)
6.Subject is pregnant or breastfeeding
7.Subject has a positive medical history for hepatitis A, B, or C, or HIV (human immunodeficiency virus)
8.Subject has received any investigational/experimental device within the 30 days before the Screening Visit
9.Subject is taking, or has taken at any time in the 12 weeks before the Screening Visit, a prohibited medication [miglitol (eg, Glyset); acarbose (eg, Precose, Glucobay); voglibose (eg, Volix, Vocarb, Volibo); oseltamivir (eg, Tamiflu); dietary supplements containing mulberry extract; chloroquine; any investigational/experimental drug]
10.Subject is taking, or has taken at any time in the 26 weeks before the Screening Visit, the prohibited medication miglustat (eg, Zavesca)
11.Subject is taking, or has taken at any time in the 12 months before the Screening Visit, any immunosuppressant drug (eg, cyclosporins, azathioprines, methotrexate, monoclonal antibodies that block the immune system, and long-term corticosteroids). Subjects who require corticosteroids for acute management of infusion reactions may be allowed with sponsor approval
12.Subject has any concurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the subject may have an unacceptable risk by participating in this study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified