Drugs / CL20-i4-EF1α-hγc-OPT
last change Aug 2026 re-read 2 minutes ago

CL20-i4-EF1α-hγc-OPT

targets IL2RG via restoration

Developed for
combined immunodeficiency, X-linked
Investigated by
Assisi Foundation · California Institute for Regenerative Medicine (CIRM) · National Heart, Lung, and Blood Institute (NHLBI) · St. Jude Children's Research Hospital

Trials 1 · a red edge is where a trial was stopped

2017201820192020202120222023202420252026
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT01512888 Aug 2016 → Aug 2026 combined immunodeficiency, X-linked St. Jude Children's Research Hospital Suspended No outcome recorded

Evidence & citations 3 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

FieldValueCited text
Known as CL20-i4-EF1α-hγc-OPT ClinicalTrials.gov intervention name — accepted as the source's own label NCT01512888
Action Restore “This method involves transferring a normal copy of the common gamma chain gene into the participant's bone marrow stem cells.” NCT01512888
Target IL2RG “a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells” PMID 30995372 Apr 2019