Pan African Clinical Trials Registry

South African Medical Research Council, South African Cochrane Centre
PO Box 19070, Tygerberg, 7505, South Africa
Telephone: +27 21 938 0835 or +27 21 938 0967
Email: pactradmin@mrc.ac.za Website: pactr.samrc.ac.za
Trial no.: PACTR202201585469592 Date of Registration: 21/01/2022
Trial Status: Registered in accordance with WHO and ICMJE standards
TRIAL DESCRIPTION
Public title A study will evaluate the efficacy and safety of tiragolumab plus atezolizumab compared with placebo in patients with unresectable esophageal squamous cell carcinoma (or those who are unable or unwilling to undergo surgery) and whose cancers have not progressed following definitive concurrent chemoradiotherapy (dCRT).
Official scientific title A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF ATEZOLIZUMAB WITH OR WITHOUT TIRAGOLUMAB (ANTI-TIGIT ANTIBODY) IN PATIENTS WITH UNRESECTABLE ESOPHAGEAL SQUAMOUS CELL CARCINOMA WHOSE CANCERS HAVE NOT PROGRESSED FOLLOWING DEFINITIVE CONCURRENT CHEMORADIOTHERAPY
Brief summary describing the background and objectives of the trial Esophageal cancer is the seventh most commonly diagnosed cancer worldwide and the sixth most common cause of cancer-related death (572,000 new cases; 509,000 deaths in 2018); the latter signifying that esophageal cancer will be responsible for an estimated 1 in every 20 cancer deaths in 2018 (Bray et al. 2018). Approximately 70% of cases occur in men, and there is a 2- to 3-fold difference in incidence and mortality rates between the sexes worldwide (Bray et al. 2018). Most esophageal cancers can be categorized into two histological subtypes: squamous cell carcinoma and adenocarcinoma, with differences in prevalence depending on the region. Squamous cell carcinoma is the most common esophageal cancer in Asia and Africa, while adenocarcinoma is increasing in frequency in North America and Western Europe (Lin et al. 2013; Noone et al. 2017; Bray et al. 2018). Another major difference is the disease etiology: tobacco and alcohol abuse are the major risk factors for squamous cell carcinoma, whereas gastroesophageal reflux disease and Barrett’s esophagus are the two major risk factors for adenocarcinoma (Engel et al. 2003). Although the incidence of esophageal adenocarcinoma and esophagogastric junctional carcinoma has increased in the United States and Western Europe, esophageal squamous cell carcinoma accounts for ~78% of all esophageal cases worldwide, and for ~90% of patients in the highest-risk regions of Northern Iran through Central Asia to North-Central China (Arnold et al. 2015; Edgren et al. 2013; Lagergren et al 2017). Most esophageal cancer patients are diagnosed with advanced disease, where the disease is frequently recurrent and treatment options can include surgery, chemotherapy, and/or radiotherapy. Treatments can extend survival but are largely palliative; and median survival time is less than 1 year (Zhang 2013; Smyth et al. 2017). The prognosis of esophageal squamous cell carcinoma remains poor, and 5-year survival rates are between 10%-20% across the United States, Europe, and Asia (Weidmann and Mössner 2013; Arnold et al. 2015; Lordick et al. 2016; Wang et al. 2016; Murphy et al. 2017; Cheng et al. 2018; Ilson and van Hillegersberg 2018; NCCN 2019). The impact of esophageal cancer on patients is multi-faceted. Most affected individuals present with physical symptoms, primarily dysphagia, which can result in unintentional weight loss and loss of appetite (Daly et al. 2000). However, patients with esophageal cancer also frequently report poor emotional well-being, and in particular high rates of anxiety and depression (Hu et al. 2015). Each of these symptoms has a significant impact on different aspects of patients’ functioning and quality of life. Hence, there remains a significant need for novel therapeutic agents for this patient population. OBJECTIVES AND ENDPOINTS This study will evaluate the efficacy and safety of tiragolumab plus atezolizumab compared with placebo in patients with unresectable esophageal squamous cell carcinoma (or those who are unable or unwilling to undergo surgery) and whose cancers have not progressed following dCRT. In the protocol, "study treatment" refers to the combination of treatments assigned to patients as part of this study: Arm A: tiragolumab + atezolizumab Arm B: tiragolumab placebo + atezolizumab Arm C: tiragolumab placebo + atezolizumab placebo
Type of trial RCT
Acronym (If the trial has an acronym then please provide) SKYSCRAPER 07
Disease(s) or condition(s) being studied Cancer
Sub-Disease(s) or condition(s) being studied
Purpose of the trial Treatment: Drugs
Anticipated trial start date 31/03/2022
Actual trial start date
Anticipated date of last follow up 31/08/2026
Actual Last follow-up date
Anticipated target sample size (number of participants) 750
Actual target sample size (number of participants)
Recruitment status Active, not recruiting
Publication URL
Secondary Ids Issuing authority/Trial register
STUDY DESIGN
Intervention assignment Allocation to intervention If randomised, describe how the allocation sequence was generated Describe how the allocation sequence/code was concealed from the person allocating the participants to the intervention arms Masking If masking / blinding was used
Parallel: different groups receive different interventions at same time during study Randomised Stratified allocation where factors such as age, gender, center, or previous treatment are used in the stratification Central randomisation by phone/fax Masking/blinding used Care giver/Provider,Outcome Assessors,Participants
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Control Group placebo plus atezolizumab Atezolizumab at a fixed dose of 1200 mg administered by IV infusion Q3W on Day 1 of each 21-day cycle, followed by placebo administered by IV infusion Q3W on Day 1 of each 21-day cycle for up to 17 cycles. 21-day cycle up to 17 cycles Atezolizumab at a fixed dose of 1200 mg administered by IV infusion Q3W on Day 1 of each 21-day cycle, followed by placebo administered by IV infusion Q3W on Day 1 of each 21-day cycle 250 Placebo
Experimental Group Tiragolumab plus Atezolizum Atezolizumab at a fixed dose of 1200 mg administered by IV infusion every 3 weeks (Q3W) on Day 1 of each 21-day cycle, followed by tiragolumab at a fixed dose of 600 mg administered by IV infusion Q3W on Day 1 of each 21-day cycle for up to 17 cycles. 17 cycles Atezolizumab at a fixed dose of 1200 mg administered by IV infusion every 3 weeks (Q3W) on Day 1 of each 21-day cycle, followed by tiragolumab at a fixed dose of 600 mg administered by IV infusion Q3W on Day 1 of each 21-day cycle 250
Control Group Double placebo Placebo administered by IV infusion Q3W on Day 1 of each 21-day cycle in two consecutive administrations for up to 17 cycles. 17 cycles Placebo administered by IV infusion Q3W on Day 1 of each 21-day cycle in two consecutive administrations 250 Placebo
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
Patients must meet the following criteria for study entry: Signed Informed Consent Form Age 18 years at time of signing Informed Consent Form Ability to comply with the study protocol, in the investigator’s judgment ECOG Performance Status of 0 or 1 Histologically or cytologically confirmed diagnosis of squamous cell carcinoma of the esophagus Stage IIIVA per American Joint Committee on Cancer/Union for International CancerControl, 8th edition, unresectable locally advanced disease (medically or surgery is declined) prior to dCRT. dCRT treatment according to regional oncology guidelines (Such as National Comprehensive Cancer Network [NCCN; see Appendix 10 for recommended treatment], European Society for Medical Oncology [ESMO], Chinese Society of Clinical Oncology [CSCO], etc.) for esophageal cancer and with the following criteria: Patients with inoperable cancer must have received at least 2 cycles of platinum-based chemotherapy and radiation therapy consistent with definitive treatment (5064 Gy) without evidence of radiographic disease progression per RECIST v1.1, as documented by comparison of scans (pre- and post-dCRT) prior to randomization. Patients with cervical esophageal squamous cell carcinoma may receive higher radiation dose (50-66 Gy), as per local oncology guidelines. Randomization into the study must occur within 184 days after the last dose of radiation therapy. Use of herbal therapies/traditional Chinese medicines with anti-cancer activity intended to treat the disease under the study must be discontinued prior to randomization. Representative archival formalin-fixed, paraffin-embedded (FFPE) tumor specimens 12 months old, collected prior to initiation of dCRT in either paraffin blocks (preferred over slides) or approximately 10 15 slides (15 slides preferred) containing unstained, freshly cut, serial sections (of the 10-15 slides, 5 are for the stratification PD-L1 testing). The number of slides provided may also be governed by local regulations (e.g., Human Genetic Resources Administration of China) Adequate hematologic and end-organ function, defined by the following laboratory test results, obtained after the last dose of chemoradiotherapy and within 14 days prior to randomization. Negative HIV test at screening Patients without hepatitis B virus (HBV) infection or for patients with a positive hepatitis B surface antigen (HBsAg) test and/or a positive total hepatitis B core antibody (HBcAb) test in the absence of a positive hepatitis B surface antibody (HBsAb) test at screening: HBV DNA less than 500 IU/mL Patients with detectable HBV DNA should be managed per institutional guidelines. Initiation of anti-HBV therapy should be 14 days prior to initiation of study treatment, and patients should be willing to continue anti-HBV therapy for the duration of study treatment, and longer per institutional guidelines. Negative hepatitis C virus (HCV) antibody test at screening, or positive HCV antibody test followed by a negative HCV RNA test at screening. The HCV RNA test will be performed only for patients who have a positive HCV antibody test. For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm Patients who meet any of the following criteria will be excluded from study entry: Prior treatment with CD137 agonists or immune checkpoint blockade therapies, including antiCTLA-4, antiPD-1, antiPD-L1 and anti-TIGIT therapeutic antibodies Any unresolved toxicity of NCI CTCAE Grade more than or equal to 2 from the prior chemoradiation therapy. Patients with irreversible and manageable hearing loss are eligible. Evidence of complete esophageal obstruction not amenable to treatment Histology consistent with small cell esophageal carcinoma, esophageal adenocarcinoma, or mixed carcinoma High risk for developing esophageal fistula by clinical assessment or imaging, such as prior history or associated symptoms of esophageal fistula, or primary tumor invasion of the great vessels or trachea Prior esophagectomy Positive Epstein-Barr virus (EBV) viral capsid antigen IgM test at screening. An EBV polymerase chain reaction (PCR) test should be performed as clinically indicated to screen for active infection or suspected chronic active infection. Patients with a positive EBV PCR test are excluded. Uncontrolled tumor-related pain. Patients requiring pain medication must be on a stable regimen at study entry. Uncontrolled pleural effusion, pericardial effusion, or ascites requiring recurrent drainage procedures (once monthly or more frequently). Patients with indwelling catheters (e.g., PleurX) are allowed. Uncontrolled or symptomatic hypercalcemia Active or history of autoimmune disease or immune deficiency History of idiopathic pulmonary fibrosis, organizing pneumonia (e.g., bronchiolitis obliterans), drug-induced pneumonitis, or idiopathic pneumonitis, or evidence of active pneumonitis on screening chest computed tomography (CT) scan History of radiation pneumonitis in the radiation field (fibrosis) is permitted. Active tuberculosis Significant cardiovascular disease (such as New York Heart Association Class II or greater cardiac disease, myocardial infarction, or cerebrovascular accident) within 3 months prior to initiation of study treatment, unstable arrhythmia, or unstable angina Patients with known coronary artery disease, congestive heart failure not meeting the above criteria, or left ventricular ejection fraction 50% must be on a stable medical regimen that is optimized in the opinion of the treating physician, in consultation with a cardiologist if appropriate Major surgical procedure, other than for diagnosis, within 4 weeks prior to initiation of study treatment History of malignancy other than esophageal cancer within 2 years prior to screening, with the exception of malignancies with a negligible risk of metastasis or death (e.g., 5-year OS rate 90%), such as adequately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, localized prostate cancer, ductal carcinoma in situ, or Stage I uterine cancer Patients who received endoscopic mucosal resection or dissection for superficial mucosal cancers other than esophageal squamous cell carcinoma (ESCC) within 2 years prior to screening are eligible for the study. Patients with illness or conditions that interfere with their capacity to understand, follow, and/or comply with study procedures Severe infection within 4 weeks prior to randomization, including, but not limited to, hospitalization for complications of infection, bacteremia, or severe pneumonia, or any active infection that, in the opinion of the investigator, could impact patient safety Treatment with therapeutic oral or IV antibiotics within 2 weeks prior to randomization. Patients receiving prophylactic antibiotics (e.g., to prevent a urinary tract infection or chronic obstructive pulmonary disease exacerbation) are eligible for the study. Prior allogeneic stem cell or solid organ transplantation Treatment with a live, attenuated vaccine (e.g., FluMist) within 4 weeks prior to initiation of study treatment, or anticipation of need for such a vaccine during study treatment, within 5 months after the last dose of atezolizumab/placebo or 90 days after the last dose of tiragolumab/placebo, whichever is later Treatment with any other investigational agent, including EGFR inhibitors, with therapeutic intent for esophageal cancer prior to randomization Treatment with systemic immunostimulatory agents (including, but not limited to, interferon and interleukin-2 [IL-2]) within 4 weeks or 5 drug-elimination half-lives (whichever is longer) prior to randomization Treatment with systemic immunosuppressive medication (including, but not limited to, corticosteroids, cyclophosphamide, azathioprine, methotrexate, thalidomide, and antiTNF- agents) within 2 weeks prior randomization or anticipation of need for systemic immunosuppressive medication during study treatment History of severe allergic anaphylactic reactions to chimeric or humanized antibodies or fusion proteins Known hypersensitivity to Chinese hamster ovary cell products or to any component of the tiragolumab or atezolizumab formulation Pregnancy or breastfeeding, or intention of becoming pregnant during study treatment, within 5 months after the final dose of atezolizumab, or within 90 days after the final dose of tiragolumab, whichever is later Women of childbearing potential must have a negative serum pregnancy test result within 14 days prior to randomization. 80 and over: 80+ Year,Adult: 18 Year(s)-44 Year(s),Aged: 65 Year(s)-79 Year(s),Middle Aged: 45 Year(s)-64 Year(s) 18 Year(s) 100 Year(s) Both
ETHICS APPROVAL
Has the study received appropriate ethics committee approval Date the study will be submitted for approval Date of approval Name of the ethics committee
Yes 20/12/2021 KENYA MEDICAL RESEARCH INSTITUTE
Ethics Committee Address
Street address City Postal code Country
P.O. Box 54840-00200, NAIROBI, Kenya Nairobi 00200 Kenya
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Secondary Outcome Arm B vs Arm C: Investigator-Assessed PFS [ Time Frame: From randomization to the first occurrence of disease progression or death from any cause, whichever occurs first (up to approximately 50 months) ] Arm A vs Arm B: Investigator-Assessed PFS [ Time Frame: From randomization to the first occurrence of disease progression or death from any cause, whichever occurs first (up to approximately 50 months) ] Arm A vs Arm B: OS [ Time Frame: From randomization to death from any cause (up to approximately 50 months) ] Independent Review Facility (IRF)-Assessed PFS [ Time Frame: From randomization to the first occurrence of disease progression or death from any cause, whichever occurs first (up to approximately 50 months) ] Investigator-Assessed Confirmed Objective Response Rate (ORR) [ Time Frame: From randomization up to approximately 50 months ] IRF-Assessed Confirmed ORR [ Time Frame: From randomization up to approximately 50 months ] From randomization up to approximately 50 months
Secondary Outcome Investigator-Assessed Duration of Objective Response (DOR) [ Time Frame: From the first occurrence of a documented confirmed objective response to the first occurrence of disease progression or death from any cause, whichever occurs first (up to approximately 50 months) ] IRF-Assessed DOR [ Time Frame: From the first occurrence of a documented confirmed objective response to the first occurrence of disease progression or death from any cause, whichever occurs first (up to approximately 50 months) ] Percentage of Participants With Clinically Meaningful Changes in Physical Functioning, Role Functioning, Quality of Life (QoL) as Measured by EORTC QLQ-C30 [ Time Frame: Up to approximately 50 months ] Clinically meaningful changes in physical functioning, role functioning, global health status (GHS)/QoL as measured by the European Organisation for Research and Treatment of Cancer Quality of Life-Core 30 Questionnaire (EORTC QLQ-C30). EORTC QLQ-C30 is a self-reported measure, consisting of 30 questions that assess 5 aspects of participants functioning (physical, emotional, role, cognitive and social), 3 symptom scales (fatigue, nausea and vomiting, and pain), GHS and QoL, and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea and financial difficulties) within the previous week. Functioning and symptoms items are scored on a 4-point scale: 1=Not at all, 2=A little, 3=Quite a bit, 4=Very much. GHS and QoL items are scored on a 7-point scale: 1=Very poor, 2, 3, 4, 5, 6, 7=Excellent. Scores will be linearly transformed to a range of 0 to 100, with higher scores (i.e. closer to 100) reflecting better functioning, better GHS/QoL, and worse symptoms. Up to approximately 50 months
Secondary Outcome Percentage of Participants With Clinically Meaningful Changes in Dysphagia as Measured by EORTC QLQ-OES18 [ Time Frame: Up to approximately 50 months ] Clinically meaningful changes in dysphagia as measured by the EORTC Quality of Life-Esophageal Cancer, Module 18 Questionnaire (EORTC QLQ-OES18). EORTC QLQ-OES18 is a modular supplement to the EORTC QLQ-C30 questionnaire for use in participants with esophageal cancer. EORTC QLQ-OES18 consists of 4 multiple-item scale (dysphagia, eating, reflux, and pain) and 6 single items (trouble swallowing saliva, choked when swallowing, dry mouth, trouble with taste, trouble with coughing, and trouble talking) with a recall period of the previous week. Each symptom item is scored on a 4-point scale: 1=Not at all, 2=A little, 3=Quite a bit, 4=Very much. Scores will be linearly transformed to a range of 0 to 100, with higher transformed scores (i.e. closer to 100) reflecting worse symptoms. Percentage of Participants With Adverse Events (AEs) [ Time Frame: Up to approximately 50 months ] Serum Concentration of Tiragolumab [ Time Frame: Predose and postdose on Day 1 of Cycle 1 (each cycle=21 days) and predose on Day 1 of Cycles 2, 3, 4, 8, 12 and 16 and at treatment discontinuation (TD) visit (up to approximately 50 months) ] Serum Concentration of Atezolizumab [ Time Frame: Predose and postdose on Day 1 of Cycle 1 (each cycle=21 days) and predose on Day 1 of Cycles 2, 3, 4, 8, 12 and 16 and at TD visit (up to approximately 50 months) ] Percentage of Participants With Anti-drug Antibodies (ADAs) to Tiragolumab [ Time Frame: Predose on Day 1 of Cycles (each cycle=21 days) 1, 2, 3, 4, 8, 12 and 16 and at TD visit (up to approximately 50 months) ] Percentage of Participants With ADAs to Atezolizumab [ Time Frame: Predose on Day 1 of Cycles (each cycle=21 days) 1, 2, 3, 4, 8, 12 and 16 and at TD visit (up to approximately 50 months) ] Up to approximately 50 months
Primary Outcome Arm A vs Arm C: Investigator-Assessed Progression-Free Survival (PFS) [ Time Frame: From randomization to the first occurrence of disease progression or death from any cause, whichever occurs first (up to approximately 50 months) ] Arm A vs Arm C: Overall Survival (OS) [ Time Frame: From randomization to death from any cause (up to approximately 50 months) ] Arm B vs Arm C: OS [ Time Frame: From randomization to death from any cause (up to approximately 50 months) ] up to approximately 50 months
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
Aga Khan University Hospital Nairobi 3rd Parklands Avenue, Off Limuru Road Nairobi 00100 Kenya
International Cancer Institute Nandi Road 8/10, Eldoret, Kenya, 8088-30100 Eldoret 30100 Kenya
FUNDING SOURCES
Name of source Street address City Postal code Country
Hoffmann La Roche The Atrium, 6th Floor Chaka Road, off Lenana Road P.O. Box 44212-00100 Nairobi, Kenya Nairobi 00100 Kenya
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor Hoffmann La Roche The Atrium, 6th Floor Chaka Road, off Lenana Road P.O. Box 44212-00100 Nairobi, Kenya Nairobi 00100 Kenya Commercial Sector/Industry
COLLABORATORS
Name Street address City Postal code Country
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Saleh Mansoor mansoor.saleh@aku.edu +254709931500 Aga Khan University, Nairobi, 3rd Parklands Avenue, Nairobi-Kenya
City Postal code Country Position/Affiliation
Nairobi Kenya Professor of Hematology and Oncology
Role Name Email Phone Street address
Public Enquiries Eileen Koske eileen.koske@roche.com +254721375237 The Atrium, 6th Floor Chaka Road, off Lenana Road P.O. Box 44212-00100 Nairobi, Kenya
City Postal code Country Position/Affiliation
Nairobi Kenya Country Study and SSU Manager East Africa
Role Name Email Phone Street address
Principal Investigator Fredrick Asirwa director@intercancer.com +254700522149 International Cancer Institute, Nandi Road 8/10, Eldoret, Kenya, 8088-30100
City Postal code Country Position/Affiliation
Eldoret Kenya Professor of Hematology and Oncology
Role Name Email Phone Street address
Scientific Enquiries Leon Liu leon.liu.ll2@roche.com +8615800486751 Roche China Holding Ltd
City Postal code Country Position/Affiliation
Shanghai China Medical Director
REPORTING
Share IPD Description Additional Document Types Sharing Time Frame Key Access Criteria
Yes There is a plan to share IPD. Plan description: - Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). - Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). - For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm) ClinicalTrials.gov Identifier: NCT04543617 Where available, the following anonymized patient level data and information is provided for each clinical study: - Raw dataset. This is the dataset collected for each patient in the clinical study. - Analysis-ready dataset. This is the dataset used for Roche’s analysis. Please note: only clinical data that underlies the CSR is made available. Therefore some data types may not be routinely provided (eg. Exploratory Biomarker data, Images, Genomic data, PK data). If individual patient data from the same study is made available outside of Vivli this can not and should not be linked to data on the Vivli platform by researchers external to Roche due to a potential increase in risk of patient re-identification. The following information will be provided: 1. Protocol. 2. Annotated case report form. 3. Reporting and analysis plan. 4. Dataset specifications. This is the meta-data which describes the datasets e.g., variable labels, variable descriptions, code lists, formats. 5. Clinical study report. Analytic Code,Clinical Study Report,Statistical Analysis Plan,Study Protocol After the medicine studied has been approved by regulators for the indication in both the US and EU or terminated from development (all indications) 18 months after completion of the study report( to enable a publication to be submitted) Available studies are listed and available on the Vivli platform. Data requestors should use the Vivli data request form to request companies data Package(s). If approved requestors will need to sign a Data Use Agreement and the anonymized data will be shared in the Vivli secure research environment.
URL Results Available Results Summary Result Posting Date First Journal Publication Date
https://vivli.org/ourmember/roche/ No
Result Upload 1: Result Upload 2: Result Upload 3: Result Upload 4: Result Upload 5:
Result URL Hyperlinks Link To Protocol
Result URL Hyperlinks
Changes to trial information