Fennec Pharmaceuticals is backing a new investigator-sponsored Phase I/II trial at the University of Arizona Cancer Center evaluating intravenous sodium thiosulfate (PEDMARK) as an otoprotectant in adolescent and young adult (AYA) and adult patients with head and neck and testicular cancers receiving cisplatin. The open-label study will assess reduction in hearing impairment against historical controls and measure systemic elimination of unbound cisplatin up to six hours post-infusion. This follows recent institution-led starts at Tampa General Hospital, focused on real-world utility in AYA and adult cohorts, and at City of Hope in adult men with stage II–III metastatic testicular germ cell tumors. PEDMARK is currently approved only in pediatric patients with localized, non-metastatic solid tumors, with an NCCN 2A recommendation extending to AYA use.
The move underscores a deliberate expansion strategy: build adult data and operational know-how through academic centers to close the evidence gap that has limited broader adoption beyond pediatrics. By anchoring the Arizona protocol to both clinical audiology endpoints and pharmacokinetics of unbound cisplatin, investigators are targeting the central tension that has tempered clinician uptake—protecting hearing without diminishing cisplatin’s antitumor effect. The six-hour post-cisplatin administration window that supported pediatric outcomes is operationally feasible, but adult regimens vary widely in infusion duration and scheduling. Clarifying PK under adult dosing patterns is directly linked to prescriber confidence, protocol standardization, and any future label expansion.
For research sites, these studies put a spotlight on infrastructure and workflow. Reliable ototoxicity assessment demands baseline and serial audiometry, consistent grading, and often centralized adjudication—capabilities that are unevenly distributed across community and academic centers. Coordination between chemotherapy infusion and delayed sodium thiosulfate dosing requires tight pharmacy, nursing, and scheduling controls, with added monitoring for electrolyte shifts and nausea management. Investigator-sponsored mechanics can lower barriers for academic centers, but funding cadence, audiology capacity, and PK sampling logistics will dictate enrollment speed and data quality. CROs and tech vendors will see demand for audiology data capture, ePROs for hearing-related quality of life, and sample handling services tailored to narrow PK windows.
Regulatory and market dynamics set a high bar. FDA has shown openness to supportive-care indications when endpoints are objective and oncologic efficacy is preserved, but prevention claims in adults typically hinge on prospective randomized data rather than historical controls. NCCN’s 2A endorsement for AYA could nudge selective off-label use, yet a broad adult label will likely require a sponsor-initiated, multicenter randomized trial with harmonized audiology endpoints and predefined non-inferiority margins for tumor outcomes. In Europe, where commercialization runs through a partner, parallel evidence in adult head and neck and germ cell settings could influence HTA stances if otoprotection is robust without compromising cure rates in highly curable tumors.
Near term, watch for three signals: the magnitude of audiometric protection versus historical rates, any deviation in disease control or survival metrics, and PK confirmation that a six-hour window maintains cisplatin exposure across variable infusion practices. Strong results would set up a pivotal adult trial, with head and neck chemoradiation and first-line germ cell therapy as logical entry points given the ototoxicity burden. Operational risk centers on heterogeneity in cisplatin administration and audiology workflows across sites, which could limit generalizability and slow scale-up. The broader takeaway is that supportive-care innovation is moving up the adult oncology agenda, but converting institutional pilots into guideline upgrades and a formal label will require rigor beyond real-world studies and a clear demonstration that hearing preservation does not come at an oncologic cost.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
