prednisolone and Muscular Atrophy, Spinal

prednisolone has been researched along with Muscular Atrophy, Spinal in 5 studies

Prednisolone: A glucocorticoid with the general properties of the corticosteroids. It is the drug of choice for all conditions in which routine systemic corticosteroid therapy is indicated, except adrenal deficiency states.
prednisolone : A glucocorticoid that is prednisone in which the oxo group at position 11 has been reduced to the corresponding beta-hydroxy group. It is a drug metabolite of prednisone.

Muscular Atrophy, Spinal: A group of disorders marked by progressive degeneration of motor neurons in the spinal cord resulting in weakness and muscular atrophy, usually without evidence of injury to the corticospinal tracts. Diseases in this category include Werdnig-Hoffmann disease and later onset SPINAL MUSCULAR ATROPHIES OF CHILDHOOD, most of which are hereditary. (Adams et al., Principles of Neurology, 6th ed, p1089)

Research

Studies (5)

Authors

Clinical Trials (7)

Trial Overview

Trial Outcomes

Cohort 1: Event-free Survival at 14 Months of Age

Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age. (NCT03505099)
Timeframe: From Day 1 up to 14 months of age

Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds

Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 26, confirmed by video recording, as a participant who sits for at least 30 seconds without assistance from another person or object. The participant was allowed to use their upper extremities. (NCT03505099)
Timeframe: From Day 1 up to 18 months of age visit

Cohort 1: Number of Participants Who Achieved the Ability to Maintain Weight at or Above the Third Percentile Without the Need for Non-Oral or Mechanical Feeding Support

"The ability to maintain weight at or above the third percentile without the need for non-oral or mechanical feeding support was defined by meeting the following criteria at each visit up to 18 months of age:~Did not receive nutrition through mechanical support (i.e., feeding tube)~Maintained weight (≥ third percentile for age and sex as defined by World Health Organization [WHO] guidelines) consistent with the participant's age at the assessment." (NCT03505099)
Timeframe: From Day 1 up to 18 months of age

Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds

Defined by the BSID GM subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported. (NCT03505099)
Timeframe: From Day 1 up to 24 months of age visit

Cohort 2: Number of Participants Who Achieved the Ability to Walk Alone

Defined by the BSID GM subtest performance criteria number 43, confirmed by video recording, as a participant who takes 5 coordinated independent steps. (NCT03505099)
Timeframe: From Day 1 up to 24 months of age visit

Number of Participants That Experienced One Grade III or Higher Unanticipated, Treatment-related Toxicity That Presents With Clinical Symptoms and Requires Medical Treatment

(NCT02122952)
Timeframe: 2 years

Number of Participants Who Experienced Permanent Ventilation or Death

Permanent ventilation was defined as the requirement of ≥ 16-hour respiratory assistance, including non-invasive ventilatory support, per day continuously for ≥ 2 weeks in the absence of an acute reversible illness, excluding perioperative ventilation. (NCT02122952)
Timeframe: Up to 13.6 months of age

Number of Participants With Assessed Improvement in Motor Function

Improvement in motor function was determined by achievement of developmental milestones, specifically achievement of ability to sit unassisted for at least 30 seconds, determined by physical therapist and confirmed by an independent central video reviewer. Achievement of functional independent sitting was defined as the ability to maintain a sitting position independently for at least 30 seconds as confirmed per video evaluation by an expert central reviewer based on videos taken either at scheduled visits or provided by the parent/legal guardian. (NCT02122952)
Timeframe: 24 months post-dose

Percent Change From Baseline in Mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score

Score ranges from 0 to 64, where 64 is the maximum possible score. A higher score is indicative of higher/better motor function. CHOP-INTEND assessments were discontinued once patients achieved higher functioning status, so the number of available data points decreased over time. (NCT02122952)
Timeframe: Baseline to 24 months post-dose

Event-free Survival at 14 Months of Age

Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age. (NCT03461289)
Timeframe: Up to 14 months of age

Number of Participants Who Achieve Independent Sitting for at Least 10 Seconds

Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight with head erect for at least 10 seconds; participant does not use arms or hands to balance body or support position. (NCT03461289)
Timeframe: From Day 1 up to 18 Months of Age Visit (Up to a Maximum of Approximately 17 Months)

Event-free Survival at 14 Months of Age

Event-free survival at 14 months of age was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study by 14 months of age. (NCT03837184)
Timeframe: From Baseline up to 14 Months of Age

Number of Participants Who Achieved Sitting Alone for at Least 10 Seconds

Independent sitting is defined by the World Health Organization Multicentre Growth Reference Study, confirmed by video recording, as a participant who sits up straight unsupported for at least 10 seconds. (NCT03837184)
Timeframe: From Baseline up to 18 Months of Age Visit

Ability to Thrive

"Ability to thrive is defined as achieving all of the following at 18 months of age:~does not receive nutrition through mechanical support or other non-oral method~ability to tolerate thin liquids as demonstrated through a formal swallowing test~maintains weight~This is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed." (NCT03306277)
Timeframe: 18 months

Achievement of Independent Sitting for at Least 30 Seconds

"Independent sitting is defined as sitting up straight with head erect for at least 30 seconds.~This endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance, was the endpoint of event-free survival assessed." (NCT03306277)
Timeframe: Up to 18 months

Event-free Survival

"Survival is defined by the avoidance of combined endpoint of either death or permanent ventilation, which is defined by tracheostomy or by the requirement of ≥ 16 hours of respiratory assistance per day for ≥ 14 consecutive days in the absence of an acute reversible illness, excluding perioperative ventilation. Permanent ventilation is considered a surrogate for death. An acute reversible illness is defined as any condition other than SMA that results in increased medical intervention.~The endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance was the survival endpoint assessed." (NCT03306277)
Timeframe: 14 months

Ventilatory Support Independence

"Ventilatory support independence is defined as requiring no daily ventilator support/usage at 18 months of age, excluding acute reversible illness and perioperative ventilation, through assessment of actual usage data captured from the device (Phillips Trilogy BiPAP device). This endpoint is derived solely from the Phillips Trilogy BiPAP device.~This is a co-secondary endpoint. The two co-secondary endpoints were assessed in sequence: The endpoint of ability to thrive was assessed first and, only when this assessment met statistical significance was the endpoint of ventilatory support independence assessed." (NCT03306277)
Timeframe: Up to 18 months

Reviews

1 review available for prednisolone and Muscular Atrophy, Spinal

Trials

1 trial available for prednisolone and Muscular Atrophy, Spinal

Other Studies

3 other studies available for prednisolone and Muscular Atrophy, Spinal