Efficacy of enzyme replacement therapy with alpha-glucosidase in patient with late-onset Pompe disease

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

IRCT

Registry ID

IRCT2017111120981N2

Enrollment

Registered

2017-11-19

Start date

2014-03-05

Completion date

Unknown

Last updated

2018-07-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pompe. Glycogen storage disease

Interventions

20 mg of alpha-glucosidase enzyme every 14 days.

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Late-onset Pumpe disease No received enzyme replacement therapy

Exclusion criteria

Exclusion criteria: Pregnancy Unwillingness to participate in the study Advanced liver disease and renal failure

Design outcomes

Primary

Measure	Time frame
Respiratory function. Timepoint: Every 6 months. Method of measurement: spirometery.;Muscular force. Timepoint: evry 6 months. Method of measurement: physical exam.	—

Secondary

Measure	Time frame
Muscular Enzymes. Timepoint: evry 6 months. Method of measurement: lab data.	—

Countries

Iran (Islamic Republic of)

Contacts

Public ContactKhadijeh Hajinaghi Tehrani

Bu-ali Hospital

Khtehrani@iautmu.ac.ir+98 912 149 3324

Outcome results

None listed

Source: IRCT (via WHO ICTRP) · Data processed: Feb 4, 2026