Avalglucosidase alfa French post-trial access for participants with Pompe disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Sanofi Winthrop Industrie

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

11 Jul 2022 → ongoing

Decision date (initial)

2024-08-06

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Sanofi Winthrop Industrie

External identifiers

EU CT number

2024-514773-22-00

EudraCT number

2021-002590-26

WHO UTN

U1111-1266-5434

ClinicalTrials.gov

NCT05164055

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To assess long-term safety in patients in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France, or until September 2026, whichever comes first.

Secondary objectives 1

1. To assess long-term efficacy outcomes in patients in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France, or until September 2026, whichever comes first.

Conditions and MedDRA coding

Glycogen storage disease type II

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Patient with LOPD or IOPD who has previously completed Study EFC14028, LTS13769, or ACT14132 in France, and reimbursement for avalglucosidase alfa is not yet granted in France.
2. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
3. The patient (and patient's legal guardian if patient is <18 years of age) must have the ability to comply with the clinical protocol.
4. The patient, if female and of childbearing potential, must have a negative pregnancy test result [urine beta-human chorionic gonadotropin (β-HCG)] at enrollment.
5. Sexually active female patients of childbearing potential and male patients are required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception.

Exclusion criteria 7

1. Patient with life-threatening hypersensitivity (anaphylactic reaction) to one of avalglucosidase alfa's excipients.
2. Patient who permanently discontinued avalglucosidase alfa in a previous clinical study
3. Pregnant or breastfeeding female patient
4. The patient is concurrently participating in another clinical study of investigational treatment
5. The patient, in opinion of the Investigator, is unable to comply with the requirements of the study
6. The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatobiliary, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival.
7. Individuals accommodated in an institution because of regulatory or legal order; prisoners, or patients who are legally institutionalized.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Number of adverse events (AE), treatmentemergent adverse events (TEAE), including infusion associated reactions (IAR) and death

Secondary endpoints 9

1. Assessment of six-minute walk test (distance in meters and % predicted value) for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants
2. Assessment of quick motor function test (QMFT) for LOPD participants
3. Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants
4. Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants
5. Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants
6. Quality of life evaluation: Pompe Disease Impact Scale (PDIS) for LOPD participants
7. Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) score for IOPD participants
8. PedsQL score for IOPD participants
9. Left Ventricular Mass Index (LVMI) Z-score in IOPD participants

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Sanofi Winthrop Industrie

Sponsor organisation

Sanofi Winthrop Industrie

Address

82 Avenue Raspail

City

Gentilly

Postcode

94250

Country

France

Scientific contact point

Organisation

Sanofi Winthrop Industrie

Contact name

Clinical Sciences and Operations

Public contact point

Organisation

Sanofi Winthrop Industrie

Contact name

Clinical Sciences and Operations

Third parties 4

Locations

1 EU/EEA country · 11 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 18 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications