Avalglucosidase Alfa for Pompe Disease

(Mini-COMET Trial)

Recruiting at 22 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Genzyme, a Sanofi Company

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 25 weeks

22 Participants Needed

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.

Research Team

Clinical Sciences & Operations

Principal Investigator

Sanofi

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.

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.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive avalglucosidase alfa or alglucosidase alfa via IV infusion every other week for 25 weeks

25 weeks

Extension Treatment

Participants continue to receive avalglucosidase alfa IV infusion every other week

Up to 430 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• alglucosidase alfa GZ419829
• avalglucosidase alfa GZ402666

Trial Overview 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.

Participant Groups

Group I: Cohort 3b: Alglucosidase Alfa in PAP Experimental Treatment 2 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//kg Experimental Treatment 1 Intervention

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 avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP.

Group III: Cohort 2: Avalglucosidase Alfa 40 mg/kg Experimental Treatment 1 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/kg Experimental Treatment 1 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).

Who Is Running the Clinical Trial?

Genzyme, a Sanofi Company

Lead Sponsor

Trials

528

Recruited

186,000+

David Meeker

Genzyme, a Sanofi Company

Chief Executive Officer since 2011

MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School

Jean-Paul Kress

Genzyme, a Sanofi Company

Chief Medical Officer since 2015

MD from Faculte Necker-Enfants Malades, Paris