Clinical Trials
The therapeutic potential of this compound has been evaluated across multiple clinical trials targeting various advanced solid neoplasms, pancreatic adenocarcinoma, biliary tract cancers, and autosomal dominant hypocalcemia. These investigations encompass Phase 1, Phase 2, and Phase 3 studies sponsored by industrial entities such as Adherex Technologies, Inc. and Calcilytix Therapeutics, Inc., as well as academic institutions including the University of Nebraska. Specific evaluations, such as NCT00421811 and NCT01825603, have investigated safety, tolerability, and anti-tumor efficacy both as monotherapy and in combination with chemotherapeutic agents like gemcitabine and cisplatin. The recruitment status across these clinical trials spans completed, terminated, recruiting, and active but not recruiting stages.
| NCT Number | Recruitment | Conditions | Sponsor/Collaborators | Start Date | Phases |
|---|---|---|---|---|---|
| NCT07080385 | RECRUITING | Autosomal Dominant Hypocalcemia Type 1 (ADH1) |
Calcilytix Therapeutics, Inc., a BridgeBio company |
2026-01-30 | PHASE2; PHASE3 |
| NCT05680818 | ACTIVE_NOT_RECRUITING | Autosomal Dominant Hypocalcemia (ADH) |
Calcilytix Therapeutics, Inc., a BridgeBio company |
2023-01-06 | PHASE3 |
| NCT01825603 | COMPLETED | Ampulla of Vater Adenocarcinoma; Gallbladder Adenocarcinoma; Metastatic Pancreatic Adenocarcinoma; Pancreatic Adenocarcinoma; Stage III Ampulla of Vater Cancer; Stage III Intrahepatic Cholangiocarcinoma; Stage III Pancreatic Cancer; Stage IIIA Gallbladder Cancer; Stage IIIA Hilar Cholangiocarcinoma; Stage IIIB Gallbladder Cancer; Stage IIIB Hilar Cholangiocarcinoma; Stage IV Ampulla of Vater Cancer; Stage IVA Gallbladder Cancer; Stage IVA Hilar Cholangiocarcinoma; Stage IVA Intrahepatic Cholangiocarcinoma; Stage IVA Pancreatic Cancer; Stage IVB Gallbladder Cancer; Stage IVB Hilar Cholangiocarcinoma; Stage IVB Intrahepatic Cholangiocarcinoma; Stage IVB Pancreatic Cancer |
University of Nebraska |
2013-04-09 | PHASE1 |
| NCT00421811 | COMPLETED | Neoplasms |
Adherex Technologies, Inc. |
2007-04 | PHASE1; PHASE2 |
| NCT00225550 | TERMINATED | Neoplasms |
Adherex Technologies, Inc. |
PHASE1; PHASE2 | |
| NCT00390676 | COMPLETED | Neoplasms |
Adherex Technologies, Inc. |
2006-11 | PHASE1 |
| NCT00264433 | COMPLETED | Neoplasms |
Adherex Technologies, Inc. |
PHASE2 | |
| NCT00265057 | COMPLETED | Neoplasms |
Adherex Technologies, Inc. |
2005-06 | PHASE1; PHASE2 |
(data from https://clinicaltrials.gov, updated on 2026-04-22)
Compliance for Clinical Use
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>).