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. 2022 Oct 11;313(1):339–357. doi: 10.1111/imr.13143

TABLE 2.

Phase 2 and Phase 3 clinical trials with low molecular weight antagonists of Factor D or Factor B. Source: clinicaltrials.gov

Compound Indication Phase Comparator/combination Identifier (status, 07/22)
A. Factor D inhibitors

Danicopan (Achillion/Alexion)

PNH 3 C5 inhibitor co‐administration

NCT04469465 (ALPHA, recruiting), NCT05389449 (not yet recruiting)

NCT03472885 (active, not recruiting)

2 None

NCT03053102 (completed)

NCT03181633 (active, not recruiting)

C3G 2 None NCT03369236 (completed)
C3G or IC‐MPGN 2 None

NCT03124368 (completed)

NCT03459443 (completed)

Geographic Atrophy secondary to AMD 2 none NCT05019521 (recruiting)
COVID‐19 2 Remdisivir NCT04988035 (ACTIV‐5, BET‐C, completed)
Vemircopan (Achillion/Alexion) PNH 2 None NCT04170023 (recruiting)
Myasthenia Gravis 2 None NCT05218096 (recruiting)
Proliferative Lupus Nephritis and IgAN 2 None NCT05097989 (recruiting)
BCX9930 (BioCryst)

PNH

2 None

NCT05116774 (REDEEM‐1; active, not recruiting)

NCT05116787 (REDEEM‐2; active, not recruiting)

NCT04702568 (active, not recruiting)

NCT04330534 (completed)

C3G, IgAN and MN 2 None NCT05162066 (RENEW; active, not recruiting)
B. Factor B inhibitors
Iptacopan (Novartis) PNH 3 Anti C5 antibody head‐to‐head NCT04558918 (APPLY‐PNH; active, not recruiting)
None

NCT04820530

(APPOINT‐PNH, recruiting)

NCT04747613 (recruiting)

2 C5 inhibitor co‐administration

NCT03439839 (completed)

None

NCT03896152 (completed)

aHUS 3 None NCT04889430 (APPELHUS; recruiting)

C3G

3 None NCT04817618 (APPEAR‐C3G; recruiting)
2 None

NCT03955445 (recruiting)

NCT03832114 (completed)

IgAN

3 None

NCT04578834 (APPLAUSE‐IgAN; recruiting)

NCT04557462 (recruiting)

2 None NCT03373461 (completed)
Autoimmune Benign Hematological Disorders (ITP and CAD) 2 None NCT05086744 (recruiting)
Lupus Nephritis 2 None NCT05268289 (recruiting)
AMD 2 None NCT05230537 (recruiting)
Idiopathic MN 2 None NCT04154787 (recruiting)