Clinical Trials

Numerous clinical trials evaluate mitotane across oncology indications, predominantly focusing on adrenocortical carcinoma, recurrent adrenal cortical cancer, and advanced prostate cancer. Spanning Phase 1 safety studies, Phase 2 combination evaluations, and Phase 3 randomized trials assessing recurrence-free survival, these investigations are supported by governmental entities, cooperative clinical networks, and academic medical centers. Overall recruitment statuses for these trials range from completed and active to recruiting and not yet recruiting.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT03583710 RECRUITING
ENSAT Stage I Adrenal Cortex Carcinoma; ENSAT Stage II Adrenal Cortex Carcinoma; ENSAT Stage III Adrenal Cortex Carcinoma
M.D. Anderson Cancer Center
 2018-08-20 PHASE3
NCT05913427 RECRUITING
Adrenocortical Carcinoma
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
 2022-06-08 PHASE2
NCT05036434 ACTIVE_NOT_RECRUITING
Adrenocortical Carcinoma
National Cancer Center, Korea
 2021-11-15 PHASE2
NCT06831175 RECRUITING
Adrenal Cortical Carcinoma; Adrenal Cortical Cancer; Adrenal Cancer
West China Hospital
 2025-03-15 PHASE2
NCT05634577 TERMINATED
Adrenocortical Carcinoma
M.D. Anderson Cancer Center
 2023-03-23 PHASE2
NCT06279442 Not yet recruiting
Carcinoma Adrenal|Carcinoma Adrenocortical Recurrent
Latin American Cooperative Oncology Group
 2024-06 --
NCT03723941 COMPLETED
Adrenocortical Carcinoma
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
 2018-07-20 PHASE3
NCT05344027 Completed
Adrenocortical Carcinoma
King''s College Hospital NHS Trust
 2022-04-11 --
NCT03083834 UNKNOWN
Adrenal Insufficiency; Adrenal Cancer
Bnai Zion Medical Center
 2017-03-14
NCT00777244 UNKNOWN
Adrenocortical Carcinoma
University of Turin, Italy
 2008-04 PHASE3
NCT00304070 COMPLETED
Stage I Adrenal Cortical Carcinoma AJCC v7; Stage II Adrenal Cortical Carcinoma AJCC v7; Stage III Adrenal Cortical Carcinoma AJCC v7; Stage IV Adrenal Cortical Carcinoma AJCC v7
Children's Oncology Group
 2007-05-03 PHASE3
NCT02057237 COMPLETED
Prostate Cancer
University Health Network, Toronto
 2013-09 PHASE1
NCT02057237 Completed
Prostate Cancer
University Health Network Toronto
 2013-09 Phase 1
NCT00778817 TERMINATED
Recurrent Adrenocortical Carcinoma; Stage III Adrenocortical Carcinoma; Stage IV Adrenocortical Carcinoma
National Cancer Institute (NCI)
 2008-12 PHASE2
NCT00094497 COMPLETED
Carcinoma, Adrenal Cortical
Collaborative Group for Adrenocortical Carcinoma Treatment
 2004-06 PHASE3
NCT00071058 COMPLETED
Adrenal Cortex Neoplasms
National Cancer Institute (NCI)
 2003-10 PHASE2
NCT00001339 COMPLETED
Adrenal Cortical Carcinoma
National Cancer Institute (NCI)
 1993-08 PHASE2

(data from https://clinicaltrials.gov, updated on 2026-07-16)

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

Compliance for Clinical Use

Mechanism and Biochemical Profile

Mitotane selectively disrupts mitochondrial bioenergetics and adrenal steroidogenesis, leading to targeted adrenocortical cellular necrosis and hormonal synthesis inhibition. This cytotoxic disruption impairs tumor cell proliferation and steroid hypersecretion, providing significant clinical benefit in patients with advanced, persistent, or recurrent adrenocortical carcinoma.

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.