Clinical Trials

Numerous clinical trials evaluate lonafarnib across Phase 1 to Phase 3 for indications including chronic hepatitis delta, progeria, and solid malignancies such as breast cancer and recurrent glioblastoma. Sponsored by academic, governmental, and industry entities—including the National Institute of Diabetes and Digestive and Kidney Diseases, Boston Children's Hospital, and Eiger BioPharmaceuticals—these studies exhibit recruitment statuses ranging from completed to active not recruiting and enrolling by invitation.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT02579044 ENROLLING_BY_INVITATION
Progeria
Boston Children's Hospital
 2015-12 PHASE1; PHASE2
NCT06775041 ACTIVE_NOT_RECRUITING
Hutchinson-Gilford Progeria Syndrome
PRG Science & Technology Co., Ltd.
 2025-01-13 PHASE2
NCT00102648 ACTIVE_NOT_RECRUITING
Malignant Supratentorial Neoplasm; Recurrent Glioblastoma; Recurrent Gliosarcoma
M.D. Anderson Cancer Center
 2004-12-21 PHASE1
NCT05229991 COMPLETED
Hepatitis D, Chronic
Soroka University Medical Center
 2021-05-15 PHASE3
NCT05953545 WITHDRAWN
Chronic Hepatitis Delta
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
 2024-05-22 PHASE2
NCT05953545 Not yet recruiting
Chronic Hepatitis Delta
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|National Institutes of Health Clinical Center (CC)
 2024-05-15 Phase 2
NCT03719313 COMPLETED
Hepatitis Delta Virus
Eiger BioPharmaceuticals
 2018-12-01 PHASE3
NCT00916747 UNKNOWN
Progeria
Boston Children's Hospital
 2009-08 PHASE2
NCT02968641 WITHDRAWN
Chronic Delta Hepatitis
Eiger BioPharmaceuticals
 PHASE2
NCT03600714 COMPLETED
Liver Disease; Hepatitis D
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
 2018-08-01 PHASE2
NCT02430194 COMPLETED
Chronic Hepatitis D Infection
Eiger BioPharmaceuticals
 2014-12 PHASE2
NCT02511431 COMPLETED
Hepatitis D
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
 2015-07-29 PHASE2
NCT02527707 COMPLETED
Chronic Delta Hepatitis
Eiger BioPharmaceuticals
 2015-09 PHASE2
NCT02579044 Enrolling by invitation
Progeria
Boston Children''s Hospital
 2015-12 Phase 1|Phase 2
NCT02430181 COMPLETED
Chronic Hepatitis D Infection
Eiger BioPharmaceuticals
 2014-11 PHASE2
NCT01495585 COMPLETED
Hepatitis D
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
 2011-12 PHASE2
NCT02430181 Completed
Chronic Hepatitis D Infection
Eiger BioPharmaceuticals
 2014-11 Phase 2
NCT01495585 Completed
Hepatitis D
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|National Institutes of Health Clinical Center (CC)
 2011-12 Phase 2
NCT00281515 COMPLETED
Epithelial Ovarian Cancer
AGO Study Group
 2006-01 PHASE2
NCT00773474 TERMINATED
Metastatic Breast Cancer
George Sledge
 2008-10 PHASE2
NCT00539968 TERMINATED
Prostate Cancer; Breast Cancer; Ovarian Cancer; Lung Cancer; Gastric Cancer
Merck Sharp & Dohme LLC
 2007-06 PHASE1; PHASE2
NCT00425607 COMPLETED
Progeria; Hutchinson-Gilford Syndrome
Monica E. Kleinman
 2007-05 PHASE2
NCT01232881 Terminated
Breast Cancer
Hoosier Cancer Research Network|United States Department of Defense|Indiana University School of Medicine|Emory University
 2009-08 --
NCT00916747 Active not recruiting
Progeria
Boston Children''s Hospital|Schering-Plough|Merck Sharp & Dohme LLC|Eiger BioPharmaceuticals
 2009-08 Phase 2
NCT00083096 UNKNOWN
Brain and Central Nervous System Tumors
European Organisation for Research and Treatment of Cancer - EORTC
 2004-03 PHASE1
NCT00081510 COMPLETED
Breast Cancer
Merck Sharp & Dohme LLC
 2003-12 PHASE2
NCT00879034 COMPLETED
Progeria; Hutchinson-Gilford Syndrome
Boston Children's Hospital
 2009-03 PHASE2
NCT00612651 COMPLETED
Gliosarcoma; Glioblastoma; Anaplastic Astrocytoma
Duke University
 2005-10 PHASE1
NCT00109538 TERMINATED
Myelodysplastic Syndromes; Leukemia, Myelomonocytic, Chronic; Myelodysplasia; Myelomonocytic
Merck Sharp & Dohme LLC
 2005-05 PHASE3
NCT00288444 TERMINATED
Lung Cancer; Soft Tissue Sarcoma; Colorectal Carcinoma; Breast Cancer; Prostate Cancer
Emory University
 2006-01 PHASE1
NCT00068757 COMPLETED
Breast Cancer
European Organisation for Research and Treatment of Cancer - EORTC
 2003-08 PHASE1
NCT00047502 COMPLETED
Chronic Myelogenous Leukemia
M.D. Anderson Cancer Center
 2002-11-01 PHASE1
NCT00102635 TERMINATED
Head and Neck Cancer
M.D. Anderson Cancer Center
 2005-01-20 PHASE1
NCT00015899 COMPLETED
Brain and Central Nervous System Tumors
Pediatric Brain Tumor Consortium
 2002-01 PHASE1
NCT00038493 COMPLETED
Glioblastoma Multiforme
M.D. Anderson Cancer Center
 2001-09-21 PHASE2
NCT00038584 COMPLETED
Carcinoma, Squamous Cell; Cancer of Head and Neck
M.D. Anderson Cancer Center
 1999-06 PHASE1
NCT00073450 TERMINATED
Carcinoma, Squamous Cell; Head and Neck Neoplasms
Merck Sharp & Dohme LLC
 2003-09 PHASE2
NCT00038597 COMPLETED
Myelogenous Leukemia, Chronic
M.D. Anderson Cancer Center
 2001-04-30 PHASE2
NCT00050336 TERMINATED
Carcinoma, Non-small-cell Lung; Metastases, Neoplasm
Merck Sharp & Dohme LLC
 2002-12 PHASE3
NCT00020774 WITHDRAWN
Liver Cancer
Jonsson Comprehensive Cancer Center
 1998-10 PHASE2
NCT00006351 COMPLETED
Bladder Cancer; Transitional Cell Cancer of the Renal Pelvis and Ureter; Urethral Cancer
European Organisation for Research and Treatment of Cancer - EORTC
 2000-06 PHASE2
NCT00003956 COMPLETED
Lymphoma; Unspecified Adult Solid Tumor, Protocol Specific
Memorial Sloan Kettering Cancer Center
 1999-04 PHASE1
NCT00005030 WITHDRAWN
Colorectal Cancer; Metastatic Cancer
M.D. Anderson Cancer Center
 1999-09-29 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-04-02)

Check the Lonafarnib (SCH66336) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Lonafarnib selectively inhibits farnesyl protein transferase, thereby preventing the essential post-translational prenylation of H-Ras, K-Ras-4B, and N-Ras proteins and suppressing downstream Akt kinase phosphorylation and survival pathways. This enzymatic block triggers caspase-8 activation and induces programmed cell death, providing therapeutic rationale for inhibiting oncogenic Ras signaling in breast cancer and glioblastoma while disrupting critical lipid-dependent processes in progeria and chronic hepatitis delta infection.

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.