Immunotherapy + Pembrolizumab for Head and Neck Cancer

(BelieveIT-201 Trial)

Recruiting at 54 trial locations

Overseen By Ascendis Oncology Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ascendis Pharma A/S

Must not be taking: Immunosuppressants, Steroids

Disqualifiers: Autoimmune, Cardiac disease, Bleeding, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 4

Duration: 6 weeks

27 Participants Needed

Trial Summary

What is the purpose of this trial?

The purpose of this trial is to evaluate the safety and efficacy of TransCon TLR7/8 Agonist, TransCon IL-2 β/γ, and pembrolizumab given prior to curative intent surgery in treatment of participants with newly diagnosed Stage III/IVA resectable locoregionally advanced head and neck squamous cell carcinoma (LA-HNSCC). After surgery, participants will receive local standard-of-care treatment and will be followed for safety, efficacy, and survival for up to 2 years. This trial contains a safety run-in to evaluate the safety and tolerability of the two treatment arms: Arm A (TransCon TLR7/8 Agonist plus pembrolizumab) and Arm B (TransCon TLR7/8 Agonist plus TransCon IL-2 β/γ). The safety run-in will be followed by the randomized Phase 2, open-label part of the trial comparing the safety, efficacy and survival of treatment Arm A or Arm B compared to treatment Arm C (pembrolizumab monotherapy).

Research Team

Joan Morris

Principal Investigator

Ascendis Pharma Oncology Division A/S

Eligibility Criteria

This trial is for adults with newly diagnosed, resectable Stage III/IVA head and neck squamous cell carcinoma. Participants must have a tumor suitable for surgery, adequate organ function, an ECOG performance status of 0 or 1, and no immunodeficiency or significant cardiac disease. They should not be on high-dose steroids or other immune-suppressing drugs.

Inclusion Criteria

My cancer is a new, operable, non-spreading squamous cell carcinoma.

My organs are working well.

I am scheduled for surgery on my head or neck cancer.

Exclusion Criteria

Active autoimmune conditions

I have a serious heart condition.

Known bleeding disorder that poses unacceptable risk for bleeding complications from IT injections or biopsies

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Safety Run-in

Evaluate the safety and tolerability of the two treatment arms: Arm A (TransCon TLR7/8 Agonist plus pembrolizumab) and Arm B (TransCon TLR7/8 Agonist plus TransCon IL-2 β/γ)

6 weeks

2 visits (in-person)

Treatment

Participants receive study drug(s) every 21 days for 2 cycles followed by curative-intent surgery

6 weeks

2 visits (in-person)

Follow-up

Participants are monitored for safety, efficacy, and survival after treatment

Up to 2 years

Treatment Details

Interventions

• Pembrolizumab
• TransCon IL-2 β/γ
• TransCon TLR7/8 Agonist

Trial Overview The trial tests the safety and effectiveness of two experimental treatments (TransCon TLR7/8 Agonist with pembrolizumab in Arm A; TransCon TLR7/8 Agonist with TransCon IL-2 β/γ in Arm B) against pembrolizumab alone (Arm C). It includes a safety run-in phase followed by randomized comparison over up to two years post-surgery.

Participant Groups

Group I: TransCon TLR7/8 Agonist in combination with pembrolizumab Experimental Treatment 2 Interventions

Participants receive 2 cycles, once every 3 weeks of TransCon TLR7/8 Agonist intratumoral (IT) injection in combination with pembrolizumab as a 30-minute intravenous (IV) infusion

Group II: TransCon TLR7/8 Agonist in combination with TransCon IL-2 β/γ Experimental Treatment 2 Interventions

Participants receive 2 cycles, once every 3 weeks of TransCon TLR7/8 Agonist (IT injection) in combination with TransCon IL-2 β/γ as a 30-minute IV infusion

Group III: Pembrolizumab Active Control 1 Intervention

Participants receive 2 cycles, once every 3 weeks of pembrolizumab alone as a 30-minute IV infusion

Pembrolizumab is already approved in United States, European Union, United Kingdom for the following indications:

Approved in United States as KEYTRUDA for:

• Head and neck squamous cell carcinoma (HNSCC) with PD-L1 CPS ≥1
• Melanoma
• Non-small cell lung cancer (NSCLC)
• Urothelial carcinoma
• Colorectal cancer
• Gastric cancer
• Hepatocellular carcinoma
• Renal cell carcinoma
• Cervical cancer
• Endometrial carcinoma

Approved in European Union as KEYTRUDA for:

• Head and neck squamous cell carcinoma (HNSCC) with PD-L1 CPS ≥1
• Melanoma
• Non-small cell lung cancer (NSCLC)
• Urothelial carcinoma
• Colorectal cancer
• Gastric cancer
• Hepatocellular carcinoma
• Renal cell carcinoma
• Cervical cancer
• Endometrial carcinoma

Approved in United Kingdom as KEYTRUDA for:

• Untreated metastatic or unresectable recurrent head and neck squamous cell carcinoma (HNSCC) with PD-L1 CPS ≥1

Who Is Running the Clinical Trial?

Ascendis Pharma A/S

Lead Sponsor

Trials

15

Recruited

1,600+

Ascendis Pharma Oncology Division A/S

Lead Sponsor

Trials

3

Recruited

560+