Trial · NCT07444450
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 15 of 15
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Parts A: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | Part A: Up to Day 365 | |
| Secondary | Part B: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | Part B: Up to Day 1825 | |
| Secondary | Parts A and B: Change From Baseline in Plasma Levels of Neurofilament Light Chain (NfL) | Part A: At Days 180 and 365; Part B: Up to Day 1825 | Blood will be collected to characterize changes in plasma NfL following treatment with salanersen. NfL is a protein released from damaged neurons and is a biomarker of neurodegeneration. |
| Secondary | Parts A and B: Change from Baseline in Compound Muscle Action Potential (CMAP) Amplitudes | Part A: At Day 365 and Part B: Up to Day 1825 | CMAP is a well-validated method for tracking disease progression in neuromuscular disorders such as SMA and amyotrophic lateral sclerosis and has been proposed as a potential biomarker of a therapeutic effect in SMA. CMAPs will be performed for the following nerve-muscle pairs: ulnar-abductor digiti minimi and peroneal-tibialis anterior. |
| Secondary | Parts A and B: Percentage of Participants Attaining World Health Organization (WHO) Motor Milestones | Part A: At Day 365 and Part B: Up to Day 1825 | The WHO motor milestones will include six key developmental milestones: sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone, and walking alone. |
| Secondary | Parts A and B: Percentage of Participants Attaining Hammersmith Infant Neurological Examination Section 2 (HINE-2) Motor Milestones | Part A: At Day 365 and Part B: Up to Day 1825 | Section 2 of the HINE is used to assess motor milestones and includes 8 motor milestone categories: voluntary grasp (0 to 3), ability to kick in supine position (0 to 4), head control (0 to 2), rolling (0 to 3), sitting (0 to 4), crawling (0 to 4), standing (0 to 3), and walking (0 to 3). Total HINE-2 score is the sum of points from each item and can range from 0 to 26, with higher scores depicting a better level of ability. |
| Secondary | Parts A and B: Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Motor Function Scale | Part A: At Day 365 and Part B: Up to Day 1825 | The CHOP INTEND test is designed to evaluate the motor skills of participants with significant motor weakness. It includes 16 items (capturing neck, trunk, and proximal and distal limb strength) structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0-4. The total score ranges from 0-64, with higher scores depicting better motor function. |
| Secondary | Parts A and B: Percentage of Participants who Remain Free of Clinically Manifested Spinal Muscular Atrophy (SMA) | Part A: At Day 365 and Part B: At Day 545 | |
| Secondary | Part B: Percentage of Participants who Develop Spinal Muscular Atrophy (SMA) Subtypes (Non-Sitters, Sitters and Walkers) as Assessed by the Investigator | Part B: Up to Day 1825 | |
| Secondary | Part B: Hammersmith Functional Motor Scale Expanded (HFMSE) Total Score | Part B: Up to Day 1825 | The HFMSE is a tool used to assess motor function in individuals with SMA. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all 33 items, with a maximum score of 66, with higher scores depicting better ability to perform activities. |
| Secondary | Part B: Revised Upper Limb Module (RULM) Total Score | Part B: Up to Day 1825 | The RULM is developed to assess upper limb functional abilities of participants with SMA. This test consists of a total of 20 upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function. |
| Secondary | Parts A and B: Time to Death (Overall Survival) | Part A: Up to Day 365 and Part B: Up to Day 1825 | |
| Secondary | Parts A and B: Time to Death or Permanent Ventilation | Part A: Up to Day 365 and Part B: Up to Day 1825 | Permanent ventilation is defined as tracheostomy or ≥16 hours ventilation/day continuously for \>21 days in the absence of an acute reversible event. |
| Secondary | Parts A and B: Concentration of Salanersen in Cerebrospinal Fluid (CSF) | Part A: Up to Day 365 and Part B: Up to Day 1460 | |
| Secondary | Parts A and B: Concentration of Salanersen in Serum | Part A: Up to Day 365 and Part B: Up to Day 1825 |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
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