Nirogacestat (PF-03084014) has been evaluated across numerous clinical trials spanning Phase 1 through Phase 4, primarily focusing on desmoid tumors, triple-negative breast cancer, metastatic pancreatic cancer, and relapsed or refractory multiple myeloma. Sponsored by pharmaceutical entities including Pfizer, Janssen, GlaxoSmithKline, and SpringWorks Therapeutics alongside academic groups such as the Children's Oncology Group, these studies range in recruitment status from active and recruiting to completed or terminated. Specific protocols have assessed nirogacestat in combination with cryoablation for desmoid fibromatosis and alongside targeted therapies for multiple myeloma.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07176689 | RECRUITING | Desmoid Tumor; Aggressive Fibromatosis |
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany |
2025-09-17 | PHASE4 |
| NCT07539454 | NOT_YET_RECRUITING | AIDS-Related Kaposi Sarcoma; Skin Kaposi Sarcoma |
AIDS Malignancy Consortium |
2026-09-17 | PHASE2 |
| NCT05137054 | RECRUITING | Multiple Myeloma |
Regeneron Pharmaceuticals |
2022-08-17 | PHASE1 |
| NCT05949099 | RECRUITING | Desmoid Tumor |
Nam Bui |
2023-08-15 | PHASE2 |
| NCT05556798 | ACTIVE_NOT_RECRUITING | Multiple Myeloma |
Memorial Sloan Kettering Cancer Center |
2022-10-04 | PHASE1 |
| NCT07170644 | ACTIVE_NOT_RECRUITING | Desmoid Tumor; Aggressive Fibromatosis |
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany |
2025-08-08 | PHASE2 |
| NCT05879146 | RECRUITING | Tumor |
M.D. Anderson Cancer Center |
2024-11-26 | PHASE2 |
| NCT05573802 | RECRUITING | Multiple Myeloma; Neoplasms; Neoplasm, Plasma Cell; Gammopathy, Monoclonal; Paraproteinemias; Blood Protein Disorders; Haematologic Disease; Corneal Disease |
Hellenic Society of Hematology |
2023-07-14 | PHASE1; PHASE2 |
| NCT04195399 | ACTIVE_NOT_RECRUITING | Desmoid Fibromatosis; Recurrent Desmoid Fibromatosis; Unresectable Desmoid Fibromatosis |
Children's Oncology Group |
2020-10-07 | PHASE2 |
| NCT07259330 | COMPLETED | Healthy |
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany |
2025-10-27 | PHASE1 |
| NCT07171619 | COMPLETED | Healthy |
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany |
2025-08-21 | PHASE1 |
| NCT04722146 | ACTIVE_NOT_RECRUITING | Multiple Myeloma |
Janssen Research & Development, LLC |
2021-03-12 | PHASE1 |
| NCT05348356 | COMPLETED | Ovarian Granulosa-Stromal Tumor; Ovarian Granulosa Cell Tumor; Ovarian Cancer |
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany |
2022-08-30 | PHASE2 |
| NCT07084896 | ACTIVE_NOT_RECRUITING | Multiple Myeloma |
GlaxoSmithKline |
2020-06-08 | PHASE1; PHASE2 |
| NCT07150091 | ACTIVE_NOT_RECRUITING | Multiple Myeloma |
GlaxoSmithKline |
2022-07-19 | PHASE1; PHASE2 |
| NCT07150104 | ACTIVE_NOT_RECRUITING | Multiple Myeloma |
GlaxoSmithKline |
2022-08-31 | PHASE1; PHASE2 |
| NCT04126200 | ACTIVE_NOT_RECRUITING | Multiple Myeloma |
GlaxoSmithKline |
2019-10-07 | PHASE1; PHASE2 |
| NCT05090566 | TERMINATED | Multiple Myeloma |
Pfizer |
2021-10-27 | PHASE2 |
| NCT04093596 | COMPLETED | Relapsed/Refractory Multiple Myeloma |
Allogene Therapeutics |
2019-09-23 | PHASE1 |
| NCT04171843 | TERMINATED | Relapsed/Refractory Multiple Myeloma |
Precision BioSciences, Inc. |
2020-04-30 | PHASE1 |
| NCT03785964 | COMPLETED | Desmoid Tumor; Aggressive Fibromatosis |
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany |
2019-04-17 | PHASE3 |
| NCT01981551 | COMPLETED | Desmoid Tumors; Aggressive Fibromatosis |
National Cancer Institute (NCI) |
2013-10-31 | PHASE2 |
| NCT02338531 | WITHDRAWN | Breast Cancer |
Jules Bordet Institute |
2015-06 | PHASE2 |
| NCT02462707 | WITHDRAWN | Advanced Solid Tumors |
Pfizer |
2015-07 | PHASE1 |
| NCT02299635 | TERMINATED | Triple Negative Breast Neoplasms |
Pfizer |
2015-02-03 | PHASE2 |
| NCT01876251 | TERMINATED | Breast Cancer Metastatic |
Pfizer |
2013-11-04 | PHASE1 |
| NCT02137564 | WITHDRAWN | AIDS-related Kaposi Sarcoma; HIV Infection; Recurrent Kaposi Sarcoma |
AIDS Malignancy Consortium |
2015-07 | PHASE2 |
| NCT02338531 | Withdrawn | Breast Cancer |
Jules Bordet Institute |
2015-06 | Phase 2 |
| NCT02299635 | Terminated | Triple Negative Breast Neoplasms |
Pfizer |
2015-02-03 | Phase 2 |
| NCT02109445 | TERMINATED | Metastatic Cancer Pancreas |
Pfizer |
2014-09-03 | PHASE2 |
| NCT02109445 | Terminated | Metastatic Cancer Pancreas |
Pfizer|Academic GI Cancer Consortium (AGICC) |
2014-09-03 | Phase 2 |
| NCT01876251 | Terminated | Breast Cancer Metastatic |
Pfizer |
2013-11-04 | Phase 1 |
| NCT00878189 | COMPLETED | Neoplasms by Histologic Type |
Pfizer |
2009-06-25 | PHASE1 |
(data from https://clinicaltrials.gov, updated on 2026-08-17)
Check the
Nirogacestat (PF-03084014)
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Mechanism and Biochemical Profile
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>).
- Nirogacestat (PF-03084014) Mechanism of Action: Gamma-secretase Inhibition in Mesenchymal Cell Research
- Nirogacestat (PF-03084014) Working Concentration | Cell Culture | Treatment
- Nirogacestat (PF-03084014) Solubility in DMSO
- Nirogacestat (PF-03084014) Stock Solution
- Nirogacestat (PF-03084014) Storage
- Nirogacestat (PF-03084014) Stability
- Nirogacestat (PF-03084014) Combination Database
- Nirogacestat (PF-03084014) Molecular Weight
- Nirogacestat (PF-03084014) SMILES
- Nirogacestat (PF-03084014) CAS Number
- Nirogacestat (PF-03084014) Chemical Structure (2D and 3D)
- Nirogacestat (PF-03084014) IC50 for Cell-based and Cell-free Assays
- Nirogacestat (PF-03084014) Sigma (PZ0298)? Try a better option.
- Nirogacestat (PF-03084014) MedChemExpress (HY-15185)? Try a better option.
- Nirogacestat (PF-03084014) Cayman (27061)? Try a better option.
- Nirogacestat (PF-03084014) APExBIO (B1193)? Try a better option.
- Nirogacestat (PF-03084014) SDS｜MSDS
Note: Technical data last updated: Sep 1, 2026.