Treatment Trials

5 Clinical Trials for Various Conditions

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TERMINATED
Screening Study to Identify Patients With Complement Factor I Deficiencies
Description

This is screening study to identify patients with Complement Factor I deficiencies. The primary objective is to identify participants with CFI deficiencies and assess the prevalence in the screened population.

COMPLETED
First-In-Human Clinical Study of the C3 Complement Inhibitor AMY- 101 in Healthy Male Volunteers
Description

Safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of a Single Ascending Dose (SAD) and a Multiple Dose (MD) of the complement inhibitor AMY-101. A prospective, single-center, open-label, First-In-Human (FIH) clinical study in healthy male volunteers.

TERMINATED
A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471
Description

The primary purpose of this study was to evaluate the efficacy of 12 months of oral ACH-0144471 (also known as danicopan and ALXN2040) in participants with C3G or IC-MPGN based on histologic scoring and proteinuria.

COMPLETED
A Proof-of-Concept Study of Danicopan for 6 Months of Treatment in Participants With C3 Glomerulopathy (C3G)
Description

The primary purpose of this proof-of-concept clinical study was to evaluate the efficacy and safety of the study drug, ACH-0144471 (also known as danicopan and ALXN2040), in participants with C3G who also had significant proteinuria attributable to C3G.

TERMINATED
A Natural History Study to Assess the Clinical Outcomes of Patients With Complement Factor I Deficiency-Mediated Disease
Description

This study will follow participants with a disease which is associated with an absence or deficiency in Complement Factor I (CFI) as confirmed in the CFI-001 screening study.