Trial · NCT07903376
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 20 of 21
Download this page as JSON| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Occurrence and Severity of Adverse Events and Serious Adverse Events | Through Month 12 | Number, nature, severity, seriousness, timing, outcome, and assessed relationship to SPX-01 of adverse events, serious adverse events, and dose-limiting toxicities using DAIDS grading criteria |
| Primary | Change in Alanine Aminotransferase (ALT) Levels From Baseline | Baseline through Month 12 | Change in serum ALT levels from baseline to each scheduled post-treatment assessment. ALT concentrations will be measured in U/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Aspartate Aminotransferase (AST) Levels From Baseline | Baseline through Month 12 | Change in serum AST levels from baseline to each scheduled post-treatment assessment. AST concentrations will be measured in U/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Platelet Count From Baseline | Baseline through Month 12 | Change in platelet count from baseline to each scheduled post-treatment assessment. Platelet counts will be measured in ×10⁹/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Serum Creatinine Levels From Baseline | Baseline through Month 12 | Change in serum creatinine levels from baseline to each scheduled post-treatment assessment as an indicator of renal function. Serum creatinine will be measured in mg/dL, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Cardiac Troponin I Levels From Baseline | Baseline through Month 12 | Change in cardiac troponin I levels from baseline to each scheduled post-treatment assessment as a marker of myocardial injury. Troponin I levels will be measured in ng/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Lactate Dehydrogenase (LDH) Levels From Baseline | Baseline through Month 12 | Change in serum lactate dehydrogenase (LDH) levels from baseline to each scheduled post-treatment assessment as a marker of hemolysis and tissue injury. LDH levels will be measured in U/L, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Hemoglobin Levels From Baseline | Baseline through Month 12 | Change in blood hemoglobin concentration from baseline to each scheduled post-treatment assessment as an indicator of hematologic safety and potential hemolysis. Hemoglobin levels will be measured in g/dL, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Change in Total Bilirubin Levels From Baseline | Baseline through Month 12 | Change in serum total bilirubin levels from baseline to each scheduled post-treatment assessment as an indicator of hepatic safety and potential hemolysis. Total bilirubin levels will be measured in mg/dL, and absolute changes from baseline will be summarized at each assessment time point. |
| Primary | Occurrence of Thrombotic Microangiopathy (TMA) Following SPX-01 Administration | Through Month 12 | Assessment of the occurrence of thrombotic microangiopathy (TMA) in the participant following SPX-01 administration, based on clinical and laboratory findings consistent with microangiopathic hemolytic anemia, thrombocytopenia, and acute kidney injury. Suspected TMA will undergo clinical evaluation and diagnostic confirmation according to the study protocol. TMA occurrence will be reported as present or absent (Yes/No) during the follow-up period. |
| Secondary | Overall Survival Status | Through Month 12 | Documentation of whether the participant remains alive during the 12-month follow-up period |
| Secondary | Event-Free Survival Status | Through Month 12 | Documentation of death or sustained respiratory support of at least 16 hours per day for at least 14 days, in the absence of an acute reversible illness. |
| Secondary | Change in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Total Score From Baseline | Baseline; Months 1, 3, 6, and 12 | Change from baseline in the total score of the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), a 16-item scale designed to assess motor function in infants with neuromuscular disorders, including spinal muscular atrophy (SMA) Type 1. Each item is scored from 0 to 4, yielding a total score ranging from 0 to 64. Higher scores indicate better motor function, and a positive change from baseline indicates improvement. Individual changes from baseline will be assessed at Months 1, 3, 6, and 12. |
| Secondary | Age at First Achievement of Full Head Control as Assessed by HINE-2 | Through Month 12 | Age in months at first achievement of full head control, as assessed using the head control item of the Hammersmith Infant Neurological Examination, Section 2 (HINE-2). Achievement will be defined as a head control item score of 2, indicating the ability to maintain the head upright consistently. Age at achievement will be calculated based on the participant's date of birth and the date of the first assessment confirming milestone achievement. |
| Secondary | Age at First Achievement of Independent Rolling as Assessed by HINE-2 | Through Month 12 | Age in months at first achievement of independent rolling, as assessed using the rolling item of the Hammersmith Infant Neurological Examination, Section 2 (HINE-2). Achievement will be defined as a rolling item score of 3, indicating the ability to roll independently from supine to prone. Age at achievement will be calculated based on the participant's date of birth and the date of the first assessment confirming milestone achievement. |
| Secondary | Age at First Achievement of Independent Sitting as Assessed by HINE-2 | Through Month 12 | Age in months at first achievement of independent sitting, as assessed using the sitting item of the Hammersmith Infant Neurological Examination, Section 2 (HINE-2). Achievement will be defined as a sitting item score of at least 3, indicating the ability to maintain a stable sitting position without external support. Age at achievement will be calculated based on the participant's date of birth and the date of the first assessment confirming milestone achievement. |
| Secondary | Age at First Achievement of Standing With Support as Assessed by HINE-2 | Through Month 12 | Age in months at first achievement of standing with support, as assessed using the standing item of the Hammersmith Infant Neurological Examination, Section 2 (HINE-2). Achievement will be defined as a standing item score of at least 2, indicating the ability to maintain a standing position with support. Age at achievement will be calculated based on the participant's date of birth and the date of the first assessment confirming milestone achievement. |
| Secondary | Change in Compound Muscle Action Potential (CMAP) Amplitude From Baseline | Baseline; Months 1, 3, 6, and 12 | Change from baseline in compound muscle action potential (CMAP) amplitude, measured using motor nerve conduction studies (electrophysiological assessment). CMAP amplitude will be recorded in millivolts (mV) and reflects the summed electrical response of muscle fibers following peripheral motor nerve stimulation. Absolute changes in CMAP amplitude from baseline will be summarized at Months 1, 3, 6, and 12. A positive change from baseline indicates an increase in CMAP amplitude. |
| Secondary | Ventilatory Support Status | Baseline through Month 12 | Documentation of the participant's requirement for invasive or non-invasive ventilatory support during the 12-month follow-up period. Ventilatory support status will be assessed through clinical evaluations and medical records, including the type of respiratory support required and any changes from baseline. |
| Secondary | Swallowing Function Status | Baseline through Month 12 | Documentation of the participant's swallowing function during the 12-month follow-up period following SPX-01 administration. Swallowing status will be assessed through clinical evaluation and documented as the presence or absence of swallowing dysfunction, including clinical evidence of impaired swallowing or aspiration during feeding. Changes from baseline will be recorded at scheduled follow-up assessments. |
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
Read this list from software.
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GET https://conductscience.org/api/trials/v1/NCT07903376/registered