Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

NCT03729362

Pompe Disease (Late-onset)

COMPLETED PHASE3

Clinical Study of the CardioFocus Endoscopic Ablation System for the Treatment of Symptomatic AF

NCT00958165

Paroxysmal Atrial Fibrillation

COMPLETED PHASE3

Post-Market Clinical Study of the CardioFocus Endoscopic Ablation System - Adaptive Contact (CF-EAS-AC) for the Treatment of Symptomatic Atrial Fibrillation

NCT01196923

Atrial Fibrillation

COMPLETED PHASE4

Substrate Targeted Ablation Using the FlexAbility™ Ablation Catheter System for the Reduction of Ventricular Tachycardia

NCT02130765

Monomorphic Ventricular Tachycardia

TERMINATED NA

Sole-Therapy Treatment of Atrial Fibrillation

NCT00566176

Atrial Fibrillation

UNKNOWN PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This program is being offered on a patient by patient basis and will require company, IRB/IEC and single patient IND approval.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Pompe Disease

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ATB200

Intervention Type BIOLOGICAL

AT2221

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Subject must have a diagnosis of Pompe disease based on documentation of one of the following:

1. deficiency of GAA enzyme
2. GAA genotyping
2. Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
3. The patient must be willing to receive treatment with ATB200/AT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC.

Exclusion Criteria

1. Patient, whether male or female, is planning to conceive a child during the treatment program.
2. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
3. Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with or adversely impact protocol requirements.
4. Patient has received any gene therapy at any time.
5. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)

Eligible Sex

ALL

Accepts Healthy Volunteers

No