Registered outcomes11

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

1 to 11 of 11

List	Outcome	Time frame
Primary	Change in Scale for Assessment and Rating of Ataxia (SARA) modified total score	From baseline to Week 52
Primary	Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score	From baseline to Week 52
Secondary	Percent change in spleen volume	From baseline to Week 52
Secondary	Percent change in liver volume	From baseline to Week 52
Secondary	Change in hemoglobin level	From baseline to Week 52
Secondary	Percent change in platelet count	From baseline to Week 52
Secondary	Percent change in CSF GL-1 and lyso-GL-1 levels	From baseline to Week 52
Secondary	Percent change in plasma GL-1 and lyso-GL-1 levels	From baseline to Week 52
Secondary	Number of patients with treatment emergent adverse events (TEAEs)/ serious adverse events (SAEs)/ adverse events of special interest (AESIs)	From baseline to max of 4.5 years
Secondary	Change in score of Beck Depression Inventory II (BDI-II) during the treatment-emergent period (for participants 13 years of age and above at baseline)	From baseline to Week 52
Secondary	Change in Patient Health Questionnaire 9 (PHQ-9) during the treatment-emergent period (for participants 12 years of age at baseline)	From baseline to Week 52

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

Twenty rows a page, as JSON. No key.

GET https://conductscience.org/api/trials/v1/NCT05222906/registered