COMPIS - Congenital myopathy intervention study. An open-label, cross over, randomized, controlled study using salbutamol

Conditions

Congenital myopathy

Brief summary

Increase of MFM32 test after 6 months of treatment as compared to no treatment.

Detailed description

Increased walking distance during 6-minute walking test after 6 months of treatment., Less fatigability during physical activity after 6 months of treatment: assessed by comparing subjects speed (m/min) during the first and last minute of 6 minute walking test., Decreased time needed to complete timed function tests (10 m walk/run, 4 steps and down) after 6 months treatment., Increased hand strength on hand held myometry after 6 months on treatment., Less fatigability in upper extremities during physical examination: assessed with 5 consecutive 9 hole peg tests, where time of completion of first set is compared with time of completion of the last set., Increase in forced vital capacity both sitting and lying down after 6 months of treatment., Improvement of quality of life after 6 months of treatment.

Related papers

No related papers found yet.

Interventions

DRUGVentoline 0

DRUG4 mg/ml oral lösning.

DRUGSALBUTAMOL WZF

DRUG2 mg

DRUGtabletki

DRUG4 mg

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Increase of MFM32 test after 6 months of treatment as compared to no treatment.	—

Secondary

Measure	Time frame
Increased walking distance during 6-minute walking test after 6 months of treatment., Less fatigability during physical activity after 6 months of treatment: assessed by comparing subjects speed (m/min) during the first and last minute of 6 minute walking test., Decreased time needed to complete timed function tests (10 m walk/run, 4 steps and down) after 6 months treatment., Increased hand strength on hand held myometry after 6 months on treatment., Less fatigability in upper extremities during physical examination: assessed with 5 consecutive 9 hole peg tests, where time of completion of first set is compared with time of completion of the last set., Increase in forced vital capacity both sitting and lying down after 6 months of treatment., Improvement of quality of life after 6 months of treatment.	—

Measure

Time frame

Increased walking distance during 6-minute walking test after 6 months of treatment., Less fatigability during physical activity after 6 months of treatment: assessed by comparing subjects speed (m/min) during the first and last minute of 6 minute walking test., Decreased time needed to complete timed function tests (10 m walk/run, 4 steps and down) after 6 months treatment., Increased hand strength on hand held myometry after 6 months on treatment., Less fatigability in upper extremities during physical examination: assessed with 5 consecutive 9 hole peg tests, where time of completion of first set is compared with time of completion of the last set., Increase in forced vital capacity both sitting and lying down after 6 months of treatment., Improvement of quality of life after 6 months of treatment.

—

Countries

Sweden

Outcome results

None listed