Clinical Trials

Numerous clinical trials have evaluated LCL161 as both monotherapy and combination regimens across Phase 1 and Phase 2 studies for indications such as multiple myeloma, metastatic pancreatic cancer, triple-negative breast cancer, myelofibrosis, and advanced solid tumors. Sponsored by industry leaders like Novartis Pharmaceuticals alongside research institutions such as the Mayo Clinic and M.D. Anderson Cancer Center, these trials exhibit varied recruitment statuses ranging from completed to terminated or unknown.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT02098161 COMPLETED
Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase; Primary Myelofibrosis; Secondary Myelofibrosis
M.D. Anderson Cancer Center
 2014-12-18 PHASE2
NCT02890069 COMPLETED
Colorectal Cancer, Non-small Cell Lung Carcinoma (Adenocarcinoma), Triple Negative Breast Cancer, Renal Cell Carcinoma
Novartis Pharmaceuticals
 2016-10-14 PHASE1
NCT02649673 TERMINATED
Small Cell Lung Cancer; Ovarian Cancer
SCRI Development Innovations, LLC
 2016-03-23 PHASE1
NCT03111992 COMPLETED
Multiple Myeloma
Novartis Pharmaceuticals
 2017-12-18 PHASE1
NCT03111992 Completed
Multiple Myeloma
Novartis Pharmaceuticals|Novartis
 2017-12-18 Phase 1
NCT01955434 COMPLETED
Recurrent Plasma Cell Myeloma; Refractory Plasma Cell Myeloma
Mayo Clinic
 2013-11 PHASE2
NCT01934634 UNKNOWN
Metastatic Pancreatic Cancer
US Oncology Research
 2014-03 PHASE1
NCT01968915 COMPLETED
Neoplasms
Novartis Pharmaceuticals
 2013-11 PHASE1
NCT01240655 COMPLETED
Solid Tumors
Novartis Pharmaceuticals
 2011-04 PHASE1
NCT01617668 COMPLETED
Breast Cancer
Novartis Pharmaceuticals
 2012-08 PHASE2
NCT01934634 Unknown status
Metastatic Pancreatic Cancer
US Oncology Research|Novartis Pharmaceuticals|Delta Clinical Research LLC
 2014-03 Phase 1
NCT01968915 Completed
Neoplasms
Novartis Pharmaceuticals|Novartis
 2013-11 Phase 1
NCT01617668 Completed
Breast Cancer
Novartis Pharmaceuticals|Novartis
 2012-08 Phase 2
NCT01098838 COMPLETED
Advanced Solid Tumors
Novartis Pharmaceuticals
 2008-11 PHASE1

(data from https://clinicaltrials.gov, updated on 2023-06-06)

Check the LCL161 product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

LCL161 functions as a small-molecule second mitochondrial activator of caspase (SMAC) mimetic that potently binds to inhibitor of apoptosis proteins, including XIAP and c-IAP, promoting their autoproteolytic degradation and blocking downstream anti-apoptotic signaling pathways. This targeted inhibition restores pro-apoptotic signaling and triggers programmed cell death in malignant cells, ultimately curbing tumor growth in clinical conditions such as multiple myeloma, triple-negative breast cancer, and advanced solid tumors.

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>).

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Note: Technical data last updated: Sep 1, 2026.