---
title: Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
nct_id: NCT02405598
overall_status: COMPLETED
phase: PHASE4
sponsor: National Taiwan University Hospital
study_type: INTERVENTIONAL
primary_condition: Pompe Disease
countries: Taiwan
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT02405598.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT02405598"
ct_last_update_post_date: 2016-09-20
last_seen_at: "2026-05-12T06:37:36.285Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease

**NCT ID:** [NCT02405598](https://clinicaltrials.gov/study/NCT02405598)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE4
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 14
- **Lead Sponsor:** National Taiwan University Hospital
- **Conditions:** Pompe Disease
- **Start Date:** 2013-10
- **Completion Date:** 2016-06
- **CT.gov Last Update:** 2016-09-20

## Brief Summary

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

## Eligibility

- **Minimum age:** 2 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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
```

## Arms

- **Salbutamol** (EXPERIMENTAL) — 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)

## Interventions

- **Salbutamol** (DRUG) — 1. Albuterol Syrup (1mL= Salbutamol 0.4mg, 60ml/bot)
2. VENTOLIN TABLETS 2MG (SALBUTAMOL SULPHATE)

## Primary Outcomes

- **10% decrement of creatinine kinase** _(time frame: 6 months)_ — measure and compare serum creatinine kinase levels
- **Improvement in 6-minutes walk test** _(time frame: 6 months)_ — measure and compare 6-minutes walk test

## Locations (1)

- National Taiwan University Hospital, Taipei, Taiwan, Taiwan

## Recent Field Changes (last 30 days)

- `design.enrollmentCount` — added _(2026-05-12)_
- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.national taiwan university hospital|taipei|taiwan|taiwan` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT02405598.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT02405598*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*