NCT01203592

Brief Summary

The study tests the notion that patients suffering from certain types of congenital myasthenic syndromes are benefitted by the use of Albuterol at doses used in clinical practice.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Sep 2010

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2010

Completed
14 days until next milestone

First Submitted

Initial submission to the registry

September 15, 2010

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 16, 2010

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2013

Completed
Last Updated

January 1, 2016

Status Verified

December 1, 2015

Enrollment Period

2.9 years

First QC Date

September 15, 2010

Last Update Submit

December 31, 2015

Conditions

Congenital Myasthenic Syndrome

Keywords

Congenital myasthenic syndromeAlbuterolNeuromuscular transmission

Outcome Measures

Primary Outcomes (1)

  • Name: Efficacy of albuterol in the treatment of congenital myasthenic syndromes

    The primary outcome measures pertain to evaluating the improvement in the patient's strength

    3 years

Study Arms (1)

Albuterol

EXPERIMENTAL

4 mg twice daily by mouth for adults. The dose for children 6 to 12 years is 2 mg two or three times daily; the dose for children 2 to 6 years is 0.1 mg/kg/day (maximum 2 mg) three times daily.

Drug: Albuterol

Interventions

AlbuterolDRUG

4 mg twice daily by mouth for adults. The dose for children 6 to 12 years is 2 mg two or three times daily; the dose for children 2 to 6 years is 0.1 mg/kg/day (maximum 2 mg) three times daily.

Also known as: Ventolin
Albuterol

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of congenital myasthenic syndrome substantiated by typical clinical history, seronegativity to AChR and MuSK, and evidence of a decremental EMG response.

You may not qualify if:

  • Uncontrolled hypertension, arrhythmias, or other significant cardiac disease.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Myasthenic Syndromes, Congenital

Interventions

Albuterol

Condition Hierarchy (Ancestors)

Neuromuscular Junction DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

EthanolaminesAmino AlcoholsAlcoholsOrganic ChemicalsAminesPhenethylaminesEthylamines

Study Officials

  • Andrew G Engel, MD

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
PI

Study Record Dates

First Submitted

September 15, 2010

First Posted

September 16, 2010

Study Start

September 1, 2010

Primary Completion

August 1, 2013

Study Completion

August 1, 2013

Last Updated

January 1, 2016

Record last verified: 2015-12

Locations

US(1)