Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

Phase 1

• Conditions: Pompe disease (acid alpha-glucosidase deficiency); MedDRA version: 24.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2016-000942-77-DE
• Lead Sponsor: Genzyme Corporation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-06-27
• Last Update Posted: 4 December 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

-The patient has confirmed GAA enzyme deficiency from any tissue
source and/or 2 confirmed GAA gene mutations.
-The patient must provide signed, informed consent prior to performing
any study related procedures. Consent of a legally authorized
guardian(s) is (are) required for legally minor patients as defined by
local regulation. If the patient is legally minor, signed written consent
shall be obtained from parent(s)/legal guardian and assent obtained
from patients, if applicable.
-The patient (and patient's legal guardian if patient is legally minor as
defined by local regulation) must have the ability to comply with the
clinical protocol.
-The patient, if female and of childbearing potential, must have a
negative pregnancy test (beta-human chorionic gonadotropin) at
baseline.
-The patient is <3 years of age.
-The patient has known Pompe specific cardiac hypertrophy.
-The patient is wheelchair dependent.
-The patient is not able to ambulate 40 meters (approximately 130 feet)
without stopping and without an assistive device.
-The patient requires invasive-ventilation (non-invasive ventilation is
allowed).
-The patient is not able to successfully perform repeated forced vital
capacity (FVC) measurements in upright position of =30% predicted and
=85% predicted.
-The patient has had previous treatment with alglucosidase alfa or any
investigational therapy for Pompe disease.
-The patient has prior or current use of immune tolerance induction
therapy
Are the trial subjects under 18? yes
Number of subjects for this age range: 7
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 122
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 17

Exclusion Criteria

-The patient is <3 years of age.
-The patient has known Pompe specific cardiac hypertrophy.
-The patient is wheelchair dependent.
-The patient is not able to ambulate 40 meters (approximately 130 feet) without topping and without an assistive device.
-The patient requires invasive-ventilation (non-invasive ventilation is allowed).
-The patient is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of =40% predicted and =85% predicted.
-The patient has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease.
-The patient has prior or current use of immune tolerance induction therapy

Study & Design

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