Avalglucosidase Alfa for Pompe Disease
(Mini-COMET Trial)

Recruiting in Palo Alto (17 mi)

+22 other locations

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Genzyme, a Sanofi Company

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.

Eligibility Criteria

This trial is for children under 18 with infantile-onset Pompe disease who've been on alglucosidase alfa for at least 6 months. They must show signs of the disease worsening or not improving enough, and be able to follow the study plan. Pregnant or breastfeeding females can't join, nor those allergic to the study drugs or on certain other medications.

Inclusion Criteria

I am of legal age or have consent from my guardian to participate.

I have Pompe Disease and my breathing, movement, or heart health is getting worse.

I (and my guardian, if I'm a minor) can follow the study's requirements.

+6 more

Exclusion Criteria

I am using effective birth control or am willing to be tested for pregnancy.

You are at high risk for a severe allergic reaction to neoGAA (avalglucosidase alfa).

I have high antibody levels against alglucosidase alfa.

+2 more

Participant Groups

The trial tests avalglucosidase alfa's safety and effectiveness in kids previously treated with alglucosidase alfa for Pompe Disease. It compares how both drugs work in the body (pharmacokinetics) and their impact on symptoms like heart size, muscle function, breathing, and eyelid drooping.

4Treatment groups

Experimental Treatment

Group I: Cohort 3b: Alglucosidase Alfa in PAPExperimental Treatment2 Interventions

After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose \[between 20 mg/kg qow and 40 mg/kg weekly as per physician\] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP. After PAP, participants received avalglucosidase alfa 40mg/kg IV infusion qow from Week 26 up to Week 371 in ETP.

Group II: Cohort 3a: Avalglucosidase Alfa 40 mg//kgExperimental Treatment1 Intervention

Group III: Cohort 2: Avalglucosidase Alfa 40 mg/kgExperimental Treatment1 Intervention

Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in ETP.

Group IV: Cohort 1: Avalglucosidase Alfa 20 mg/kgExperimental Treatment1 Intervention

Avalglucosidase alfa, 20 mg/kg intravenous (IV) infusion every other week (qow) for 25 weeks in the Primary Analysis Period (PAP), followed by same treatment from Week 26 up to Week 371 in extension treatment period (ETP).