Trial · NCT04488133
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 | Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score | Up to Day 778 | Section 2 of the HINE was used to assess motor milestones of the participants. It is composed of 8 motor milestone categories: voluntary grasp, ability to kick in supine position, head control, rolling, sitting, crawling, standing, and walking. Within each category, 3 to 5 levels can be achieved. Total HINE section 2 motor milestones score was calculated as the sum of each level and can range from 0 to 26. A higher score indicated good neurological function. |
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From first dose of study drug up to end of study (up to Day 778) | An AE was any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE was any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE was any untoward medical occurrence that at any dose resulted in death; placed the participant at immediate risk of death (according to the investigator's judgment); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in a birth defect or was a medically important event. |
| Secondary | Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Haematology Parameters) | Baseline up to Day 778 | Hematology parameters included hemoglobin, hematocrit, erythrocytes, platelets, leukocytes, neutrophils, eosinophils, basophils, lymphocytes and monocytes count. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high postbaseline values. |
| Secondary | Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Blood Chemistry Parameters) | Baseline up to Day 778 | Blood chemistry parameters included bilirubin, direct and indirect bilirubin, alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, gamma-glutamyl transferase, creatinine, urea nitrogen, cystatin C, sodium, potassium, chloride, bicarbonate, protein, albumin, calcium, phosphate, glucose, and creatine kinase. These parameters were flagged as low, normal, or high relative to the parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high postbaseline values. The categories with at least one participant with shift from baseline in these parameters are reported. |
| Secondary | Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Coagulation Parameters) | Baseline up to Day 778 | Coagulation parameters included activated partial thromboplastin time, prothrombin international (Intl.) normalized ratio and prothrombin time. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high postbaseline values. The categories with at least one participant with shift from baseline in these parameters are reported. |
| Secondary | Number of Participants With Shifts From Baseline in Clinical Laboratory Parameters (Urinalysis Parameters) | Baseline up to Day 778 | Urinalysis included assessments of specific gravity, pH, protein, glucose, ketones, direct bilirubin, occult blood, erythrocytes, leukocytes, epithelial cells, transitional epithelial cells, hyaline casts, ammonium biurate, amorphous crystals, calcium oxalate crystals, triple phosphate crystals and uric acid crystals. These parameters were flagged as low, normal, or high relative to parameter's normal range or as unknown if no result was available. Here, shift to low indicated values that were normal, high or unknown at baseline and shifted to low values postbaseline. Shift to high indicated values that were normal, low or unknown at baseline and shifted to high postbaseline values. The categories with at least one participant with shift from baseline in these parameters are reported. |
| Secondary | Number of Participants With Shift From Baseline in Clinically Significant Abnormalities in Electrocardiograms (ECGs) | Baseline up to Day 778 | Clinical significance of abnormalities in ECG was determined based on the investigator's discretion. Shift to abnormal indicated values that were normal or unknown at baseline and shifted to abnormal values postbaseline. |
| Secondary | Number of Participants With Abnormalities in Vital Signs | Up to Day 778 | Vital sign assessment included temperature, heart rate, systolic blood pressure, and diastolic blood pressure. As pre-specified in SAP, the criteria for determining potentially clinically relevant abnormalities in vital signs included: temperature \> 38.0 degrees Celsius (C), heart rate increments or decrements of 15 and 30 beats per minute (bpm), systolic blood pressure increments or decrements of 20 and 40 \[millimeters of mercury (mmHg)\], diastolic blood pressure increments or decrements of 10 and 20 mmHg. The categories with at least one participant with clinically relevant vital sign abnormalities are reported. |
| Secondary | Number of Participants Who Achieved Motor Milestones as Assessed by World Health Organization (WHO) Criteria | Day 778 | The WHO motor milestones were a set of six milestones in motor development, all of which would be expected to be attained by 24 months of age in healthy children. The individual milestones are: sitting without support, standing with assistance, hands and knees crawling, walking with assistance, standing alone and walking alone. |
| Secondary | Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Score | Baseline, Day 778 | The CHOP-INTEND test was designed to evaluate the motor skills of infants with significant motor weakness. It was evaluated for participants \< 2 years of age at the time of informed consent and for participants 2 to ≤ 3 years of age at the time of informed consent if they did not achieve independent sitting prior to Screening. It included 16 items (capturing neck, trunk, and proximal and distal limb strength), nine of which were scored 0, 1, 2, 3, or 4 with greater scores indicating greater muscle strength, five were scored as 0, 2, or 4, one was scored as 0, 1, 2, or 4, and one as 0, 2, 3, or 4. Total score was calculated as the sum of scores for each item. Total score ranged from 0 (worst possible score) and 64 (best possible score). CHOP-INTEND assessments were discontinued once participants achieved a maximum score of 64 after Day 421, so the number of participants with data points decreased over time. |
| Secondary | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score | Baseline, Day 778 | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. It was assessed only in participants aged 2 years and older. Participants were asked to do a specific activity (such as rolling) and they were then graded on the quality and execution of that movement on a scale of 0 = being unable, 1 = performed with some compensation, and 2 = unaided. The overall score is the sum of the scores for all activities and ranged from 0 to 66. Higher scores indicated increased motor function. |
| Secondary | Change From Baseline in Revised Upper Limb Module (RULM) Score | Baseline, Day 778 | RULM test was developed to assess upper limb functional abilities. It was assessed only in participants aged 2 years and older. RULM test: total 19 items. 18 items were assessed on 3-point scale: 0-Unable to achieve independently; 1-Modified method but achieves goal independent of physical assistance from another person; 2-Normal -achieves goal without any assistance. One remaining item was assessed as either 0 or 1. For each item, score was collected on left \& right side. Derived total score=summing scores from these 19 individual items ranging from 0-if participant fails all activities to 37-if participant achieves all activities. If, for an individual item, response was recorded for both left \& right side, highest score was used in calculating total score. Higher scores=increased upper limb function. |
| Secondary | Time to Death or Permanent Ventilation | Up to Day 778 | Time to death or permanent ventilation is defined as the time from first dose to death or permanent ventilation (tracheostomy or 16 hours of ventilation/day continuously for \> 21 days in the absence of an acute reversible event), whichever comes first. Time to death or permanent ventilation was estimated using the Kaplan-Meier method. |
| Secondary | Change From (Ratio to) Baseline in Cerebrospinal Fluid (CSF) Levels of Neurofilament Light Subunit (NF-L) | Baseline, Day 659 | The change from baseline data was reported in terms of geometric mean ratio to baseline. Lower ratios to baseline represent greater reductions in concentrations of NF-L from baseline. |
| Secondary | Change From (Ratio to) Baseline in Plasma Levels of NF-L | Baseline, Day 778 | The change from baseline data was reported in terms of geometric mean ratio to baseline. Lower ratios to baseline represent greater reductions in concentrations of NF-L from baseline. |
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.
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