AMX0035 in Adult Patients With Wolfram Syndrome | NCT05676034 | Trialant

AMX0035 in Adult Patients With Wolfram Syndrome | NCT05676034 | Trialant

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
AMX0035	Experimental	AMX0035 administered by mouth for 208 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participant	Drug: AMX0035

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
To evaluate the effect of AMX0035 on residual beta-cell function by monitoring C-peptide levels during a 0-240 minutes mixed-meal tolerance test (MMTT)	C-peptide area under the curve (AUC) response at Week 24 using a 0-240 minute MMTT Change from Baseline in area under the curve (AUC) in delta C-peptide at Week 24 using a 0-240 minute MMTT	24 weeks
To assess the safety and tolerability of AMX0035 administered orally for up to 208 weeks in adult participants with Wolfram syndrome	Incidence and severity of Adverse Events and Serious Adverse Events Incidence of abnormalities in clinical laboratory assessments	208 weeks

Secondary Outcomes

Measure	Description	Time Frame
To evaluate the effect of AMX0035 during a 0-240 minute MMTT	C-peptide AUC response to a 240-minute MMTT at Week 48	48 weeks
To assess the effect of AMX0035 on visual acuity	Change from Baseline on best-corrected visual acuity for both eyes measured on the LogMAR scale by sight tests using Snellen chart at Week 48

Contacts/Locations Module

Condition Browse Module