DOM-INNATE: Study of SGX942 for the Treatment of Oral Mucositis in Patients With Concomitant Chemoradiation Therapy for Head and Neck Cancer

• SGX942 (Drug), also known as Dusquetide: 1.5 mg/mL SGX942 administered as a 4 minute IV infusion, twice per week starting within 3 days after initiating radiation therapy and continuing through 2 weeks after radiation therapy ends.
• Placebo (Drug): Placebo is 0.9% sodium chloride (normal saline). The treatment preparation, frequency and duration of therapy are identical to that of the active drug.

Primary outcomes

• Duration of Severe Oral Mucositis (SOM) [approx. 13 weeks]: To assess the efficacy of SGX942 compared to placebo in decreasing the duration of severe oral mucositis (SOM; defined as World Health Organization \[WHO\] Grade ≥3). Duration of SOM is defined as the number of days from the onset of SOM until resolution of SOM. OM is evaluated using the published WHO OM grading scale that uses a scale of 0 to 4, with SOM defined as a score ≥3.

Sex: All, ages: 18 Years to any

Inclusion Criteria:

* Biopsy-proven squamous cell carcinoma of the oral cavity or oropharynx without distant organ metastases
* Scheduled to receive cisplatin chemotherapy of 80-100 mg/m²
* Scheduled to receive a continuous course of fractionated, conventional external beam with a cumulative radiation dose between 55 and 72 Gy at each site

Exclusion Criteria:

* Current mucositis
* Current, clinically significant, active infection that in the opinion of the Investigator would make them an unfit participant in the trial
* Planned to receive Erbitux™ (Cetuximab) or similar targeted therapy between Baseline and 6 weeks post-RT
* Prior radiation to the head and neck
* Chemotherapy treatment within the previous 12 months
* Tumors of the lips, sinuses, salivary glands, nasopharynx, hypopharynx, or larynx
* Evidence of significant renal, hepatic, hematologic, or immunologic disease determined by any one of the following: Estimated creatinine clearance <30 mL/min; ALT or AST level greater than 10-fold the upper limit of normal or total bilirubin greater than 3-fold the upper limit of normal; Manifestations of end-stage liver disease, such as ascites or hepatic encephalopathy; Thrombocytopenia; or CD4+ T cell count below 200 cells per μL
* Evidence of immediate life-threatening disease or a life expectancy of less than 3 months
* Women who are pregnant or breast-feeding
* Participation in any study involving administration of an investigational agent within 30 days of randomization into this study

Participant flow

Outcome results

• Duration of Severe Oral Mucositis (SOM) (Primary), Median, days: SGX942 12.5 (4.0 to 22.0); Placebo 20.0 (12.0 to 29.0) (p 0.1798)

Adverse events

Time frame: Adverse event (AE) data was collected between randomization through 6 weeks after radiation therapy treatment ended. Participants received up to 7 weeks of radiation, for a maximum AE collection time of 13 weeks.

Serious adverse events by term

Other adverse events by term

2 more terms not shown.

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• Kudrimoti M, Curtis A, Azawi S, Worden F, Katz S, Adkins D, Bonomi M, Elder J, Sonis ST, Straube R, Donini O. Dusquetide: A novel innate defense regulator demonstrating a significant and consistent reduction in the duration of oral mucositis in preclinical data and a randomized, placebo-controlled phase 2a clinical study. J Biotechnol. 2016 Dec 10;239:115-125. doi: 10.1016/j.jbiotec.2016.10.010. Epub 2016 Oct 13. PMID 27746305
• Kudrimoti M, Curtis A, Azawi S, Worden F, Katz S, Adkins D, Bonomi M, Scott Z, Elder J, Sonis ST, Straube R, Donini O. Dusquetide: Reduction in oral mucositis associated with enduring ancillary benefits in tumor resolution and decreased mortality in head and neck cancer patients. Biotechnol Rep (Amst). 2017 May 17;15:24-26. doi: 10.1016/j.btre.2017.05.002. eCollection 2017 Sep. PMID 28649557

• Study Protocol (PDF, 2019-09-17)
• Statistical Analysis Plan (PDF, 2020-11-24)