Phase 2 N=25 Treatment

Drug-drug Interaction Study

Pompe Disease

Bottom Line

View on ClinicalTrials.gov: NCT01380743 ↗

Enrolled (actual)

Serious AEs

4.0%

Results posted

Aug 2018

Primary outcome: Primary: Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) — 0; 0; 0; 1 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: duvoglustat (Drug); rhGAA (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Amicus Therapeutics
Primary completion: Jan 2013

Outcome Measures

Outcome	Result	p-value
PRIMARY Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs)	0; 0; 0; 1	—
PRIMARY Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease	17269; 22785; 18986; 18980; 20539; 28607	—
PRIMARY PK: Time To The Maximum Plasma Concentration (Tmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease	4.74; 4.00; 4.00; 4.00; 4.51; 4.00	—
PRIMARY PK: Elimination Half-life (T1/2) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease	3.81; 3.82; 3.56; 3.70; 4.42; 4.77	—
PRIMARY PK: Area Under The Plasma Concentration Versus Time Curve From Time 0 To The Time Of The Last Measurable Concentration (AUC0-t) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease	108578; 141515; 107489; 118483; 159994; 226198	—
PRIMARY PK: AUC From Time 0 Extrapolated To Infinity (AUCinf) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease	110388; 144056; 108862; 120604; 165983; 237613	—
SECONDARY Total GAA Activity In Skeletal Muscle	1409; 1926; 2622; 1887; 1952; 2320	—
SECONDARY Duvoglustat Concentration In Skeletal Muscle	0; 5.9; 38.8; 83.6; 8.7; 0	—

Summary

This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe Disease.

Eligibility Criteria

Inclusion Criteria

Male or female, diagnosed with Pompe disease and between 18 and 65 years of age, inclusive
Participant has been on a stable regimen and dose of rhGAA for at least 3 months before screening (stable regimen defined as currently receiving rhGAA every 2 weeks and stable dose defined as not varying by more than ± 10%)
Participant has an estimated glomerular filtration rate (eGFR) ≥ 50 mL/min at Screening; eGFR to be estimated using the 4-parameter Modification of Diet in Renal Disease (MDRD) equation:

eGFR (mL/min/1.73 m^2) = 175 x (Scr)^(-1.154) x (Age)^(-0.203) x (0.742 if female) x (1.212 if African-American)

Male and female participants of childbearing potential agree to use medically accepted methods of contraception during the study and for 30 days after study completion
Participant is willing and able to provide written informed consent and is able to comply with all study procedures

Exclusion Criteria

Participant has had a documented transient ischemic attack, ischemic stroke, unstable angina, or myocardial infarction within the 3 months before Screening
Participant has clinically significant unstable cardiac disease (for example, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association class III or IV congestive heart failure)
Participant requiring mechanical ventilation or is confined to a wheelchair
Participant has a history of allergy or sensitivity to study drug (including excipients) or other iminosugars (for example, miglustat, miglitol)
Participant is pregnant or breastfeeding
Participant tests positive for hepatitis B surface antigen or hepatitis C antibody
Participant has received any investigational/experimental drug or device within 30 days of Screening
Participant has any intercurrent illness or condition that may preclude the participant from fulfilling the protocol requirements or suggests to the investigator that the potential participant may have an unacceptable risk by participating in this study

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01380743). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.