Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00688597
• Date of registration: 30/05/2008
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
• Scientific title: An Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients With Pompe Disease
• Date of first enrolment: December 8, 2008
• Target sample size: 3
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00688597
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, Canada, France, Germany, Netherlands, United Kingdom, United States

Contacts

• Name: Medical Monitor Clinical Research
• Affiliation: Amicus Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Male or female, 18 to 74 years of age inclusive

- Diagnosis of Pompe disease based on clinical assessment, enzyme assay, and/or
genotyping. Confirmatory genotyping will be performed on all participants who are
screened for the study

- Naïve to enzyme replacement therapy (ERT) or has not received ERT in the 3 months
prior to screening

- Willing not to initiate ERT or other prohibited treatment during study participation

- Functional grade for arms and/or legs =2 OR sitting forced vital capacity =30% and
<80% of predicted value, reproducible between screening and baseline (±15%)

- Participants of reproductive potential agree to use reliable methods of contraception
during the study

- Participant or legal representative is willing and able to provide written informed
consent

Exclusion Criteria:

- Any intercurrent condition that may preclude accurate interpretation of study data

- Obstructive pulmonary disease

- Invasive ventilatory support

- Use of noninvasive ventilatory support >8 hours/day while awake

- History of QTc prolongation >450 milliseconds (msec) for males and >470 msec for
females

- History of allergy or sensitivity to the study drug, including any prior serious
adverse reaction to iminosugars (such as miglustat or miglitol)

- Pregnancy or breast-feeding

- Current or recent drug or alcohol abuse

- Treatment with another investigational drug within 30 days of study start

- Use of prohibited medications =3 months prior to screening

- Otherwise unsuitable for the study in the opinion of the Investigator



Age minimum: 18 Years
Age maximum: 74 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease

Intervention(s)

Drug: Duvoglustat

Primary Outcome(s)

Proportion Of Participants Experiencing Severe Treatment-emergent Adverse Events (TEAEs) [Time Frame: Baseline, Week 11]

Secondary Outcome(s)

Change In 6-minute Walk Test (6MWT) From Baseline To End Of Study [Time Frame: Baseline, Week 11]

Secondary ID(s)

POM-CL-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 17/08/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00688597