Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

National Taiwan University

Status and phase: Completed; Phase 4

Conditions: Pompe Disease

Treatments: Drug: Salbutamol

Study type: Interventional

Funder types: Other

Identifiers

NCT02405598

201308031MINC

Details and patient eligibility

About

evaluate if beta 2-adrenergic agonist can have adjuvant effect to patients with infantile-onset Pompe disease under enzyme replacement therapy

Enrollment: 14 patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Confirm diagnosis as Pompe disease
2. age 2 years or above
3. under at least 1 year regular recombinant human acid alpha glucosidase, and no dosage/frequency change in recent 3 months
4. Provide inform consent

Exclusion criteria

1. chronic heart disease such as arrhythmia, cardiomyopathy, cardiac infarction
2. history of seizure
3. history of diabetes mellitus
4. history of hyperthyroidism
5. hypokalemia
6. pregnant
7. allergy to β-agonists
8. under medications including diuretics, digoxin, beta-blockers, etc

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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14 participants in 1 patient group

Salbutamol

Experimental group

Description:

1. age 2-6 year: 0.1mg/kg tid x 2 weeks, then gradually increase to 0.2mg/kg tid (daily total maximal12mg) 2. age 6-12 year: 2mg tid x 2 weeks, then gradually increase to 4mg tid (daily total maximal 24mg) 3. age 12 year and above: 4mg tidx 2 weeks, then gradually increase to 8mg tid (daily total maximal 32mg)

Treatment:

Drug: Salbutamol