carbamates and Becker Muscular Dystrophy

carbamates has been researched along with Becker Muscular Dystrophy in 9 studies

Research

Studies (9)

Authors

Clinical Trials (1)

Trial Overview

Trial Outcomes

Change From Baseline in Muscular Function After 12 Months of Treatment With Givinostat at the Selected Daily Dose Based on the 6-Minute Walk Test

"This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.~The 6-Minute Walk Test is a useful measure of functional capacity targeted at people with at least moderately severe impairment.~The longer the walked distance the better the outcome." (NCT01761292)
Timeframe: At 12 months

Change From Baseline in Muscular Function After 12 Months of Treatment With Givinostat at the Selected Daily Dose Based on the North Star Ambulatory Assessment (NSAA)

"The NSAA, which is composed by 17 items, was graded, for each item, using the standard scorecard with each assessment rated as 0 - unable to achieve independently, 1 - modified method but achieves goal independent of physical assistance from another, or 2 - normal with no obvious modification of activity.~The subscales scores are summed up to compute a total score, ranging from 0 to 34. The higher the total score, the better the outcome.~The mean Change From Baseline to EoS in NSAA total score is reported hereunder." (NCT01761292)
Timeframe: At 12 months

Change From Baseline in Muscular Function After 12 Months of Treatment With Givinostat at the Selected Daily Dose Based on the Performance of Upper Limb (PUL)

"The PUL (version 1.2) was used to assess the change in motor performance of the upper limb over time in patients with Becker and Duchenne muscular dystrophy, from when they are still ambulant, until they loose all arm function when non-ambulant.~The revised version of the PUL included 22 items. These include one entry item to define the starting functional level, and 21 items subdivided into:~shoulder level (Question B to E; minimum score 0 and maximum score 16)~elbow level (Question F to N; minimum score 0 and maximum score 34)~distal level dimension (Question O to V; minimum score 0 and maximum score 24) The total score is calculated by the sum of all the scores of the three subscales (total score range: 0-74) (scores from Question A entry item did not contribute). For all items, the higher the score, the better the outcome." (NCT01761292)
Timeframe: At 12 months

Change From Baseline to End of Study in Cross Sectional Area (CSA)

This histological parameter was evaluated on the brachial biceps biopsies taken prior to the first dose of study drug and after 12 months of treatment with givinostat. (NCT01761292)
Timeframe: At 12 months

Change From Baseline to End of Study in Number of Hypercontracted Fibers

This histological parameter was evaluated on the brachial biceps biopsies taken prior to the first dose of study drug and after 12 months of treatment with givinostat. The number of fibers is calculated per microscopic field (20x). (NCT01761292)
Timeframe: At 12 months

Change From Baseline to Part 2 in the Value of Muscle Fiber Area (MFA) % Comparing the Histology Biopsies Before and After 12 Months of Treatment With Givinostat.

"The primary endpoint was the change in histology comparing the brachial biceps biopsies before and after ≥12 months of treatment with Givinostat.~Muscle biopsies: A first brachial biceps biopsy (baseline) was taken prior to the first dose of study drug. A second brachial biceps biopsy was taken at Visit 10 (12 months) from the opposite arm.~The muscle biopsy samples from the biceps muscle were collected by open biopsy. The minimum amount of muscle tissue required was a piece of muscle of at least 0.5 × 0.5 × 0.5 cm." (NCT01761292)
Timeframe: After12 months of treatment

Change From Baseline in Muscular Function After 24 (Extension 1), 36 (Extension 2), and 52 Months (Extension 3) of Treatment With Givinostat at the Selected Daily Dose Based on the North Star Ambulatory Assessment (NSAA)

"The NSAA, which is composed by 17 items, was graded, for each item, using the standard scorecard with each assessment rated as 0 - unable to achieve independently, 1 - modified method but achieves goal independent of physical assistance from another, or 2 - normal with no obvious modification of activity.~The subscales scores are summed up to compute a total score, ranging from 0 to 34. The higher the total score, the better the outcome.~The mean Change From Baseline to EoS in NSAA total score is reported hereunder." (NCT01761292)
Timeframe: At 24, 36, and 52 months

Change From Baseline in Muscular Function After 24 (Extension 1), 36 (Extension 2), and 52 Months (Extension 3) of Treatment With Givinostat at the Selected Daily Dose Based on the Performance of Upper Limb (PUL)

"The PUL (version 1.2) was used to assess the change in motor performance of the upper limb over time in patients with Becker and Duchenne muscular dystrophy, from when they are still ambulant, until they loose all arm function when non-ambulant.~The revised version of the PUL included 22 items taking. These include one entry item to define the starting functional level, and 21 items subdivided into:~shoulder level (Question B to E; minimum score 0 and maximum score 16)~elbow level (Question F to N; minimum score 0 and maximum score 34)~distal level dimension (Question O to V; minimum score 0 and maximum score 24) The total score is calculated by the sum of all the scores of the three subscales (total score range: 0-74) (scores from Question A entry item did not contribute). For all items, the higher the score, the better the outcome." (NCT01761292)
Timeframe: At 24, 36, and 52 months

Change From Baseline in Muscular Function After After 24 (Extension 1), 36 (Extension 2), and 52 Months (Extension 3) of Treatment With Givinostat at the Selected Daily Dose Based on the 6-Minute Walk Test

"This test measures the distance that a patient can quickly walk on a flat, hard surface in a period of 6 minutes.~The 6-Minute Walk Test is a useful measure of functional capacity targeted at people with at least moderately severe impairment.~The longer the walked distance the better the outcome." (NCT01761292)
Timeframe: At 24, 36, and 52 months

Change From Baseline to End of Study in Fibrosis, Necrosis, Fatty Replacement

These histological parameters were evaluated on the brachial biceps biopsies taken prior to the first dose of study drug and after 12 months of treatment with givinostat. (NCT01761292)
Timeframe: After 12 months

Number of Children Experiencing Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Type and Severity of TEAEs

Summary of Treatment-emergent Adverse Events (TEAE) Reporting from Baseline to the End of Extension 3 (Month 52). In the analysis were included: Any TEAE, Any treatment-related TEAE, Any mild or moderate or severe TEAE, Any life-threatening or disabling TEAE, Any TEAE resulting in death, any serious adverse event, and Any TEAE resulting in study discontinuation. (NCT01761292)
Timeframe: Part 1, Part 2, and Extensions 1, 2, and 3

Reviews

2 reviews available for carbamates and Becker Muscular Dystrophy

Trials

3 trials available for carbamates and Becker Muscular Dystrophy

Other Studies

4 other studies available for carbamates and Becker Muscular Dystrophy