Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
Conditions
Brief summary
Safety • Treatment-Emergent Adverse Events and Serious Adverse Events, vital signs, ECG, physical and neurological examination, safety laboratory blood and urinalysis, Efficacy • Skin deposition of cystatin C/amyloid protein complex, including monomer vs dimer (and other high-molecular versions of same) ratios, together with skin collagen deposition and cell surface marker activation (vimentin, SMAD/WNT-1) which are correlated, Serious cerebral bleedings events
Detailed description
• Clinical Dementia Rating (CDR) Scale, • Deaths, • Levels of cystatin C/amyloid dimers/oligomiers/polymers vs monomers of same, • Levels of glutathione and GSSG/GSH ratios in plasma, • hCC levels in urine, Plasma concentrations of NPI-001, • Change in amyloid deposition in the brain
Interventions
None listed
Sponsors
Arctic Therapeutics ehf.
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety • Treatment-Emergent Adverse Events and Serious Adverse Events, vital signs, ECG, physical and neurological examination, safety laboratory blood and urinalysis, Efficacy • Skin deposition of cystatin C/amyloid protein complex, including monomer vs dimer (and other high-molecular versions of same) ratios, together with skin collagen deposition and cell surface marker activation (vimentin, SMAD/WNT-1) which are correlated, Serious cerebral bleedings events | — |
Secondary
| Measure | Time frame |
|---|---|
| • Clinical Dementia Rating (CDR) Scale, • Deaths, • Levels of cystatin C/amyloid dimers/oligomiers/polymers vs monomers of same, • Levels of glutathione and GSSG/GSH ratios in plasma, • hCC levels in urine, Plasma concentrations of NPI-001, • Change in amyloid deposition in the brain | — |
Countries
Iceland
Outcome results
None listed