leniolisib (CDZ 173) Clinical Trials

Size	Price	Qty
5mg	USD 227
25mg	USD 670

Clinical Trials

Leniolisib (CDZ173) is being evaluated across several clinical trials targeting primary immunodeficiency disorders, primarily Activated PI3K-delta Syndrome (APDS), PASLI disease, and Common Variable Immunodeficiency (CVID). Spanning Phase 2, Phase 3, and combined Phase 2/3 designs, these studies assess safety, pharmacokinetics, and therapeutic efficacy in pediatric and adult populations. Sponsored by industry entities including Pharming Technologies B.V. and Novartis Pharmaceuticals, the trials encompass completed early-stage dose-finding protocols alongside active, recruiting, and non-recruiting extension evaluations.

NCT Number	Recruitment	Conditions	Sponsor/Collaborators	Start Date	Phases
NCT06990529	RECRUITING	PIDs Linked to PI3K	Pharming Technologies B.V.	2025-04-29	PHASE2
NCT05693129	ACTIVE_NOT_RECRUITING	APDS	Pharming Technologies B.V.	2023-08-30	PHASE3
NCT05438407	ACTIVE_NOT_RECRUITING	APDS	Pharming Technologies B.V.	2023-02-01	PHASE3
NCT06549114	ACTIVE_NOT_RECRUITING	Primary Immunodeficiency Disorders (PIDs)	Pharming Technologies B.V.	2024-10-21	PHASE2
NCT06897358	ACTIVE_NOT_RECRUITING	Common Variable Immunodeficiency (CVID)	Pharming Technologies B.V.	2025-02-12	PHASE2
NCT06249997	UNKNOWN	APDS Gene Mutation	Pharming Technologies B.V.	2023-08-03	PHASE3
NCT06249997	Recruiting	APDS Gene Mutation	Pharming Technologies B.V.\|Laboratory Corporation of America\|Axial Biotech Inc\|CMIC Co Ltd. Japan	2023-08-03	Phase 3
NCT05693129	Recruiting	APDS	Pharming Technologies B.V.\|CMIC Co Ltd. Japan\|Labcorp Central Laboratory\|Fortrea\|Aixial Group	2023-08-30	Phase 3
NCT05438407	Active not recruiting	APDS	Pharming Technologies B.V.\|CMIC Co Ltd. Japan\|Labcorp Central Laboratory\|Fortrea\|Aixial Group	2023-02-01	Phase 3
NCT02435173	COMPLETED	Common Variable Immunodeficiency (CVID), APDS / PASLI	Novartis Pharmaceuticals	2015-08-24	PHASE2; PHASE3
NCT02859727	Active not recruiting	Activated PI3Kdelta Syndrome (APDS); PASLI Disease	Pharming Technologies B.V.	2016-09-08	Phase 2\|Phase 3
NCT02435173	Completed	Common Variable Immunodeficiency (CVID) APDS / PASLI	Novartis Pharmaceuticals\|Novartis	2015-08-24	Phase 2\|Phase 3

(data from https://clinicaltrials.gov, updated on 2026-02-13)

Check the leniolisib (CDZ 173) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Leniolisib selectively binds to and inhibits phosphoinositide 3-kinase delta (PI3Kδ), preventing the phosphorylation of PIP2 to PIP3 and downregulating downstream AKT/mTOR signaling pathways involved in lymphocyte activation and proliferation. By restoring normal immune cell homeostasis and reducing hyperactive B-cell and T-cell responses, this targeted inhibition addresses the underlying cellular pathology of primary immunodeficiency disorders such as Activated PI3K-delta Syndrome (APDS) and Common Variable Immunodeficiency.

Appendix RUO and cGMP Quality Standards

Quality Dimension	RUO (Research Use Only)	cGMP (Current Good Manufacturing Practice)
Clinical Applicability	Prohibited in human clinical trials or medical diagnostics.	Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status	Non-regulated grade; exempt from drug manufacturing laws.	Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment	Unclassified analytical or research laboratories.	Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control	Basic purity and activity testing.	Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation	Basic equipment calibration; no process validation required.	Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance	Vendor self-declared without required formal QMS.	Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact	High risk of IND rejection if used as a critical raw material.	Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).