Trial Profile

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

Status: Recruiting

Phase of Trial: Phase III

Latest Information Update: 01 Jun 2025

Price :

$35 ^*

Note:

Adis is an information provider.
Final gross price and currency may vary according to local VAT and billing address.
Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

Drugs Cipaglucosidase alfa (Primary) ; Miglustat (Primary) ; Alglucosidase alfa
Indications Glycogen storage disease type II
Focus Adverse reactions; Registrational
Acronyms ROSSELLA
Sponsors Amicus Therapeutics

04 May 2023 Planned initiation date changed from 1 Apr 2023 to 1 Jun 2023.
18 Jan 2023 Planned End Date changed from 1 Feb 2025 to 1 Apr 2027.
18 Jan 2023 Planned primary completion date changed from 1 Feb 2025 to 1 Apr 2027.

If your organization or you do not have a subscription, try one of the following:

Contacting your organization’s admin about adding this content to your AdisInsight subscription
Buying a PDF version of any individual profile
Request a free trial (opens in a new window)

If your organization has a subscription, there are several access options, even while working remotely:

Working within your organization’s network
Login with username/password or try to access via your institution
Persisted access using your organization’s identifier stored in your user browser for 90 days

For assistance, contact us at asktheexpert.adisinsight@springer.com