ASP-1929 Photoimmunotherapy (PIT) Study in Recurrent Head/Neck Cancer for Patients Who Have Failed at Least Two Lines of Therapy

A Phase 3, Randomized, Double-Arm, Open-Label, Controlled Trial of ASP-1929 Photoimmunotherapy Versus Physician's Choice Standard of Care for the Treatment of Locoregional, Recurrent Head and Neck Squamous Cell Carcinoma in Patients Who Have Failed or Progressed On or After at Least Two Lines of Therapy, of Which at Least One Line Must Be Systemic Therapy

A Phase 3, Randomized, Double-Arm, Open-Label, Controlled Trial of ASP-1929 vs Physician's Choice Standard of Care for the Treatment of Locoregional, Recurrent Head and Neck Squamous Cell Carcinoma in Patients Who Have Failed or Progressed On or After at Least Two Lines of Therapy

Study Overview

• Status: Recruiting
• Conditions: Head and Neck Cancer
• Intervention / Treatment: Combination product: ASP-1929 Photoimmunotherapy; Drug: Physician's Choice SOC

Detailed Description

The study will have an Experimental Arm and a Control Arm.

Experimental Arm:

Patients may receive repeated ASP-1929 PIT interventions for up to a maximum of 8 cycles within a period of up to 12 months after randomization until the patient has complete remission, progressive disease that is no longer amendable to study treatment, patient experiences intolerable side effects, or patient discontinues study treatment. Repeat ASP-1929 treatment cycles will be administered no less than 4 weeks from the previous ASP-1929 infusion.

Control Arm:

Patients may be treated with physician's choice standard of care until the patient has progressive disease, patient experiences intolerable side effects, discontinues study treatment, or chooses to withdraw.

• Study Type: Interventional
• Enrollment (Estimated): 275
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Bogdan Veresh, MD; Phone Number: 858-207-3113; Email: clinicaltrialinfo@rakuten-med.com

Study Locations

• India
  • Gujarat
    • Vadodara, Gujarat, India, 391760
      • Recruiting
      • Kailash Cancer Hospital and Research Center
      • Contact: Yash Damor; Phone Number: 91-8469554460
      • Principal Investigator: Rajesh Kantharia
  • Tamilnadu
    • Chennai, Tamilnadu, India, 60036
      • Recruiting
      • Cancer Institute (W.I.A)
      • Contact: S. Deyva Manohari; Phone Number: 91-8681823668
      • Principal Investigator: Venkatraman Radhakrishnan
• Japan
  • Aichi
    • Nagoya-shi, Aichi, Japan, 464-8681
      • Active, not recruiting
      • Aichi Cancer Center
  • Chiba
    • Kashiwa-shi, Chiba, Japan, 277-8577
      • Active, not recruiting
      • National Cancer Center Hospital East
  • Hokkaido
    • Sapporo-shi, Hokkaido, Japan, 060-8648
      • Active, not recruiting
      • Hokkaido University Hospital
  • Okayama
    • Okayama-shi, Okayama, Japan, 700-8558
      • Active, not recruiting
      • Okayama University Hospital
  • Tokyo
    • Chuo-ku, Tokyo, Japan, 104-0045
      • Active, not recruiting
      • National Cancer Center Hospital
• Taiwan
  • Taichung, Taiwan, 40705
    • Recruiting
    • Taichung Veterans General Hospital
    • Principal Investigator: Chen-Chi Wang
    • Contact: I-Chen Tsai; Phone Number: 886-911-102-866
  • Taichung, Taiwan, 40705
    • Recruiting
    • China Medical University Hospital
    • Principal Investigator: Chun-Hung Hua
    • Contact: Chia Hui Lin; Phone Number: 886-920-211-277
  • Taoyuan, Taiwan, 333
    • Recruiting
    • Chang Gung Memorial Hospital
    • Principal Investigator: Kai-Ping Chang
    • Contact: Lora Chang; Phone Number: 886-919-977-034
  • Taipei
    • Zhongzheng, Taipei, Taiwan, 100
      • Recruiting
      • National Taiwan University Hospital
      • Principal Investigator: Pei-Jen Lou
      • Contact: Christy Lee; Phone Number: 88858 886-223-123-456
• Ukraine
  • Dnipro, Ukraine, 49102
    • Active, not recruiting
    • City Clinical Hospital #4
• United States
  • Florida
    • Tampa, Florida, United States, 33606
      • Recruiting
      • University of South Florida
      • Principal Investigator: Matthew J. Mifsud, MD
      • Contact: Tien Nguyen; Phone Number: 813-844-7745
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Recruiting
      • Grady Health
      • Principal Investigator: Charles E. Moore, MD
      • Contact: Frank Akuchie; Phone Number: 404-942-8907
  • Kentucky
    • Lexington, Kentucky, United States, 40536
      • Active, not recruiting
      • University of Kentucky, Albert B. Chandler Medical Center
  • Michigan
    • Royal Oak, Michigan, United States, 48073
      • Recruiting
      • William Beaumont Hospital
      • Principal Investigator: Jeffrey Hotaling, MD
      • Contact: Grace San Agustin; Phone Number: 248-551-6679
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Recruiting
      • University of Pennsylvania
      • Principal Investigator: Karthik Rajasekaran, MD
      • Contact: Ethan Skinner; Phone Number: 215-615-1611
    • Philadelphia, Pennsylvania, United States, 19107
      • Recruiting
      • Thomas Jefferson University - Sidney Kimmel Medical College
      • Principal Investigator: David Cognetti, MD
      • Contact: Camilo Henao; Phone Number: 215-982-0009
  • Tennessee
    • Nashville, Tennessee, United States, 37203
      • Active, not recruiting
      • Vanderbilt University
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • MD Anderson Cancer Center
      • Principal Investigator: Ann Gillenwater, MD
      • Contact: Bridget Reeves; Phone Number: 713-745-1897

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Overall Inclusion Criteria:

• Have a histologically confirmed locoregional persistent, recurrent or second primary squamous cell carcinoma of the head and neck, not amenable to curative treatment
• Have failed or progressed on or after at least 2 lines of therapy for squamous cell carcinoma of the head and neck.
• Failed or progressed following prior platinum chemotherapy
• Have completed prior curative radiation therapy for treatment of their head and neck region
• Have locoregional head and neck tumor site(s) that are all accessible to illumination
• Have target tumors that are clearly measurable by contract enhanced CT scan
• Have a life expectancy of > 6 months, based on Investigator judgment
• Male participants must agree to use contraception during the treatment period and for at least 6 months after the last ASP-1929 infusion
• Female patients of childbearing potential must not be pregnant or breastfeeding and agree to follow the contraceptive guidance during the treatment period and for at least 6 months after the last dose of trial intervention and must refrain from breastfeeding for at least 2 months after the last ASP-1929 infusion
• Have an Eastern Cooperative Oncology Group (ECOG) score of 0 or 1

Overall Exclusion Criteria:

• Have a history of significant (>= Grade 3) cetuximab infusion reactions
• Have been treated with prior systematic chemotherapy or targeted small molecule therapy or radiation therapy within 2 weeks of trial Day 1 or not recovered from adverse events due to a previously administered agent
• Have been treated with an anticancer monoclonal antibody therapy within 4 weeks of trial Day 1 or have not recovered from adverse events due to previously administered agent
• Have been treated with an investigational agent or intervention within 4 weeks of trial Day 1 or have not recovered from adverse events, due to previously administered agent or intervention
• Have a present history of distant metastatic disease (M1)
• Have an active undergoing treatment or have a diagnosis of an active cancer other than nonmelanoma skin cancer or HNSCC
• Have a tumor in enhanced CT or MRI scan invading a major blood vessel, unless the vessel has been embolized, stented or surgically ligated to prevent potential bleeding from a blood vessel
• Have impaired hepatic function
• Have impaired renal function
• Have uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with trial requirements
• Have been previously treated or randomized to any trial using ASP-1929 or RM-1929 PIT as the study treatment

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Physician's Choice SOC; docetaxel, cetuximab, methotrexate, paclitaxel	Drug: Physician's Choice SOC; docetaxel, cetuximab, methotrexate, paclitaxel
Experimental: ASP-1929 Photoimmunotherapy; Use of ASP-1929 Photoimmunotherapy	Combination product: ASP-1929 Photoimmunotherapy; Use of ASP-1929 PIT therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression-Free Survival (PFS)	PFS, defined as the time from randomization to the first occurrence of disease progression (PD), as determined by the investigator according to RECIST 1.1, or death from any cause, whichever one occurs first.	24 months
Overall Survival (OS)	OS, defined by the time interval between the patient randomization and death due to any cause.	24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Complete Response (CR)	CR, defined as disappearance of all target lesions, as determined by Central Radiographic Review according to RECIST 1.1 criteria with modifications. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to <10mm.	24 months
Complete Response by Biopsy (CRb)	CR by histopathologic biopsy of target tumor(s) and a repeat confirmatory histopathologic CR biopsy at least 3 months after initial CR histopathologic biopsy of the target tumor(s) for patients that do not demonstrate a CR by RECIST 1.1 with modifications. CR: Disappearance of all target lesions.	24 months
Duration of Response (DoR)	DoR, defined as the time from first objective response (CR or PR) to the date of the first documented tumor progression, as determined by Central Radiographic Review according to RECIST 1.1 criteria with modifications or date of death due to any cause, whichever occurs first. CR: Disappearance of all target lesions. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.	24 months
Event-Free Survival (EFS)	EFS, defined as the time interval from randomization to a >20% increase in tumor size from baseline (defined as CT scan at screening) by RECIST 1.1 with modifications, development of new locoregional disease, distant metastatic disease, or death.	24 months
Eastern Cooperative Oncology Group (ECOG) performance status		24 months
Quality of Life (QoL) assessment - EQ 5D-5L	Change from baseline in EuroQol 5-Dimension 5-Level (EQ 5D-5L) Questionnaire. EQ-5D-5L is a 5-dimension (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) questionnaire developed to assess the patient-generated health state index score from the societal perspective and subjective perceived health. For each dimension, levels of perceived problems are coded from level 1 (minimum) to level 5 (maximum): no problems (1), slight problems (2), moderate problems (3), severe problems (4), and extreme problems/inability (5). A second part of the questionnaire provides a visual analogue scale (VAS) for individually perceived health from 0 to 100 (corresponding to the worst to the best imaginable health).	24 months
Population Pharmacokinetics (PK) of ASP-1929	Estimate covariate effects of ASP-1929 PK through compartmental PK modeling.	12 months
Presence of Anti-Drug Antibodies (ADA)		24 months
Objective Response Rate (ORR)	ORR, defined by the percentage of patients with the best overall response of complete response (CR) or partial response (PR) by RECIST 1.1. CR: Disappearance of all target lesions. PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.	24 months
Quality of Life (QoL) assessment - EORTC QLQ-C30	Change from baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). EORTC QLQ-C30 is a 30-item questionnaire developed to assess QoL of head and neck cancer participants and consists of five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea and vomiting), a global health status scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of the disease. It has four-point scales for the first 28 items. The minimum value is 1 and maximum value is 4. Higher scores mean a worse outcome. Overall health was evaluated as a 7-point response scale as the other two questions in that scale. The minimum value is 1 and maximum value is 7. Higher scores mean a better outcome.	24 months
Quality of Life (QoL) assessment - EORTC QLQ H&N 35	Change from baseline in EORTC head and neck specific module (EORTC QLQ H&N 35). EORTC QLQ-H&N35 is a 35-item questionnaire developed to assess QoL of head and neck cancer participants and consists of 7 multiple-item scales that assess the symptom of pain, swallowing, senses (taste/smell), speech, social eating, social contact and sexuality. Also, six single-item scales are included to survey the presence of symptomatic problems associated with teeth, mouth=opening, dry mouth, sticky saliva, coughing, and feeling ill. The minimum value is 1 and maximum value is 4. It has 2-point scales for the last 5 items. The minimum value is 1 and maximum value is 2. For all items and scales, high scores indicate more problems.	24 months

Collaborators and Investigators

• Sponsor: Rakuten Medical, Inc.
• Investigators: Study Director: Naomi Schechter, Rakuten Medical

Publications and helpful links

General Publications

• Kobayashi H, Furusawa A, Rosenberg A, Choyke PL. Near-infrared photoimmunotherapy of cancer: a new approach that kills cancer cells and enhances anti-cancer host immunity. Int Immunol. 2021 Jan 1;33(1):7-15. doi: 10.1093/intimm/dxaa037.
• Kobayashi H, Choyke PL. Near-Infrared Photoimmunotherapy of Cancer. Acc Chem Res. 2019 Aug 20;52(8):2332-2339. doi: 10.1021/acs.accounts.9b00273. Epub 2019 Jul 23.

Study record dates

Study Major Dates

• Study Start (Actual): May 9, 2019
• Primary Completion (Estimated): September 30, 2024
• Study Completion (Estimated): December 30, 2024

Study Registration Dates

• First Submitted: December 6, 2018
• First Submitted That Met QC Criteria: December 6, 2018
• First Posted (Actual): December 7, 2018

Study Record Updates

• Last Update Posted (Actual): September 8, 2023
• Last Update Submitted That Met QC Criteria: September 5, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: EGFR; Cetuximab; HNSCC; Erbitux; Head and neck; Rakuten Medical; ASP-1929; PIT; Photoimmunotherapy; HNC; RM-1929
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Head and Neck Neoplasms
• Other Study ID Numbers: ASP-1929-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes