Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
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Phase
—
Status
Recruiting
Study type
Observational
Enrollment
150estimated
Sites
11
Countries
Ireland, Qatar, United Kingdom +1
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | Jan 2017 | actual | When the study began enrolling |
| Primary completion | Apr 2026 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Aug 2026 | estimated | The whole study's end, after follow-up |
| First posted | 2016-12-20 | estimated | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2024-11-22 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Homocystinuria Due to CBS Deficiency
Primary outcomes
what the primary-completion date above is the date OFChanges in Met cycle metabolites levels - tHcy
measured 6.5 years
Changes in Met cycle metabolites levels - total Cys
measured 6.5 years
Changes in Met cycle metabolites levels - Met
measured 6.5 years
Changes in Met cycle metabolites levels - Cth
measured 6.5 years
Patient Reported Outcome (PRO): Quality of Life in Neurological Disorders [Neuro-QoL]
measured 6.5 years
Patient Reported Outcome (PRO): Quality of Life by 36-Item Short Form Survey [SF-36]
measured 6.5 years
National Institutes of Health (NIH) Toolbox Cognition Battery
measured 6.5 years
EuroQol EQ-5D™ questionnaire to measure health and quality-of-life
measured 6.5 years
Dual-Energy X-Ray Absorptiometry to measure bone mineral density
measured 6.5 years
Eye assessments to evaluate ocular health: Visual acuity examination will be performed to determine the clarity or sharpness of vision
measured 6.5 years
Eye assessments to evaluate ocular health: Slit lamp eye examination will be performed to look for any diseases or abnormalities in the anterior portion of the eye
measured 6.5 years
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