Trial · NCT04174157
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 in probability of survival of all patients with SMA using Kaplan Meier method to estimate | Based on information collected at Baseline and every 6 months through 2 years of follow-up, then annually through 15 years of follow up. | |
| Primary | Change from baseline Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) in infants with pre-symptomatic or type I SMA | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | CHOP INTEND score ranges from 0 to 64 with higher scores indicating higher motor function |
| Primary | Change from baseline Hammersmith Infant Neurological Examination (HINE) in infants with pre-symptomatic, type I or type II SMA | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | HINE score range from 0 to 26 with higher scores indicating more development. |
| Primary | Change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) for patients with type II and III SMA | Baseline and every 6months through 2 years of follow up, then annually through 15 years of follow up | HFMSE score range from 0 to 66 with the higher scores indicating more development. |
| Primary | Incidence of treatment emergent adverse events | Through 15 years of follow up | |
| Primary | Incidence of treatment emergent serious adverse events | Through 15 years of follow up | |
| Primary | Incidence of treatment emergent adverse events related to therapy | Through 15 years of follow up | |
| Primary | Incidence of treatment emergent thrombocytopenia, hepatotoxicity and cardiac adverse events | Through 15 years of follow up | |
| Secondary | Change from baseline in rates of hospitalization | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | |
| Secondary | Change from baseline in Zarit Burden Interview | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | Zarit Burden Total Score ranges from 0 to 88. A higher score correlates with higher level of burden. |
| Secondary | Change from baseline in PedsQL Patient interview | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | PedsQL Total Scale Score is average of all items and ranges from 0 to 100. A higher score correlates with better Health-Related Quality of Life |
| Secondary | Change from baseline in PedsQL Parent interview | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | PedsQL Total Scale Score is average of all items and ranges from 0 to 100. A higher score correlates with better Health-Related Quality of Life |
| Secondary | Change from baseline in percent of patients requiring ventilator support (BiPAP, Endotracheal tube) | : Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | |
| Secondary | Change from baseline in in percent of patients requiring nutritional support (Gastrostomy Tube, Gastrojejunal tube (GT) with Nissen fundoplication, GT without Nissen fundoplication, Nasogastrictube, Nasojejunaltube or Percutaneous endoscopic gastrostomy) | Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up | |
| Secondary | Change from baseline in in percent of patients requiring mobility device support (Ankle-Foot Orthoses, Supramalleolar Orthosis, Orthotic/shoe inserts, Knee immobilizers, Knee-Ankle-Foot Orthoses , Hand splints, Spinal bracing) | : Baseline and every 6 months through 2 years of follow up, then annually through 15 years of follow up |
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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GET https://conductscience.org/api/trials/v1/NCT04174157/registered