Trial · NCT02292537
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 | Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score at Month 15 | Baseline and Month 15 | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. 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 with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. |
| Secondary | Proportion of Participants Who Achieved a 3-Point Increase From Baseline in HFMSE Score at Month 15 | Baseline and Month 15 | The HFMSE consists of 33 scored activities used to assess motor function in children with SMA. The scale was originally developed with 20 scored activities and was devised for use in children with SMA Type 2 and Type 3 with limited ambulation to give objective information on motor ability and clinical progression. The expanded scale includes an additional module of 13 items developed to allow for evaluation of ambulatory SMA patients. 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 with a maximum achievable score of 66. Higher scores indicate increased motor function. A positive change from Baseline indicates improvement. |
| Secondary | Proportion of Participants That Achieved Any New Motor Milestone at Month 15 | Month 15 | New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. |
| Secondary | Number of New Motor Milestones Achieved Per Participant | Month 15 | New motor milestones are defined as sitting without support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. |
| Secondary | Change From Baseline in Revised Upper Limb Module (RULM) Test | Baseline and Month 15 | The RULM Test is used in patients with SMA to assess upper limb functional ability items that are reflective of activities of daily living (i.e., raise a can to mouth as if drinking, take a coin and place it in a box, remove the lid of a container). The RULM test has a total of 20 items with an entry item that serves as functional class identification and does not contribute to the total score. The remaining 19 scorable items reflect different functional domains and are graded on a 3-point system with a score of 0 (unable), 1 (able, with modification), and a maximum of 2 (able, no difficulty). There is only 1 item (item I) that is scored as a can/cannot score, with 1 as the highest score. Scorable items are summed for a total score range of 0-37, with higher scores increased great upper limb function. A positive change from Baseline indicates improvement. |
| Secondary | Proportion of Participants That Achieved Standing Alone | Month 15 | If the participant was unable to achieve standing alone at Baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder. |
| Secondary | Proportion of Participants That Achieved Walking With Assistance | Month 15 | If the participant was unable to achieve walking with assistance at baseline but could achieve this at Month 15 then they were considered a responder. If they could not achieve this or if a participant terminated the study prior to the 15-month assessment due to treatment failure or death, then any imputed value was ignored and the participant was considered as a non-responder. |
| Secondary | Number of Participants That Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs) | Baseline through Month 15 | AEs: any sign, symptom, or diagnosis/disease that is unfavorable or unintended, that is new, or if pre-existing, worsens in participants administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. SAEs: an event that results in death; an event that, in the view of the investigator, places the participant at immediate risk of death; an outcome that results in a congenital anomaly/birth defect diagnosed in a child of a participant; an event that requires or prolongs inpatient hospitalization; an event that results in persistent or significant disability/incapacity. Any other medically important event that, in the opinion of the investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed in the definition above. |
| Secondary | Number of Participants With Clinically Significant Vital Sign Abnormalities | Baseline through Month 15 | Vital signs assessed for clinical significance include resting blood pressure, pulse, respiratory rate, and temperature. |
| Secondary | Number of Participants With Clinically Significant Weight Abnormalities | Baseline through Month 15 | Weight changes assessed from Baseline to Month 15. |
| Secondary | Number of Participants With Clinically Significant Neurological Examination Abnormalities | Baseline through Month 15 | Neurological changes assessed for clinical significance include assessment of mental status, level of consciousness, sensory function, motor function, cranial nerve function, and reflexes. |
| Secondary | Number of Participants With Clinically Significant Physical Examination Abnormalities | Baseline through Month 15 | Physical examination changes were assessed for clinical significance. |
| Secondary | Number of Participants With Clinically Significant Laboratory Parameter Abnormalities | Baseline through Month 15 | Laboratory parameter changes assessed for clinical significance include serum chemistry, hematology, coagulation and urinalysis. |
| Secondary | Number of Participants With Abnormal, Clinically Relevant Post-Baseline Worsening in Electrocardiogram (ECG) in Results | Baseline through Month 15 | The number of participants with abnormal, clinically relevant worsening, defined as participants with an ECG interpreted as abnormal and clinically relevant, with a comparison with Baseline value is reported. |
| Secondary | Number of Participants Taking Any Concomitant Medication Related to Dosing Procedure or Sham Procedure | Baseline through Month 15 | Concomitant medications include prescription and over-the-counter medications administered to participants on or after the first day of study treatment. |
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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