A Phase 1 interventional study of Part A and Part B in Advanced Malignancies, sponsored by TopAlliance Biosciences. Completed at 14 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-11-22.
Sponsored by TopAlliance Biosciences · Phase 1, Interventional, and Treatment
The primary objective is to assess the safety and tolerability of Toripalimab in subjects with various advanced malignancies and to evaluate the recommended Phase 2 dose.
The secondary objectives are to: 1) describe the pharmacokinetic (PK) profile of Toripalimab, 2) evaluate antitumor activity of Toripalimab; 3) determine the immunogenicity of Toripalimab; 4) evaluate overall survival.
The exploratory objectives are to: 1) evaluate biomarkers that may correlate with activity of Toripalimab, 2) evaluate pharmacodynamic effects of Toripalimab on its target receptor, programmed cell death 1 (PD-1), as well as effects on the immune system. 3) evaluate the utility of PD-L1 \& additional exploratory markers as biomarkers that could aid in selection of appropriate subjects for TAB001 therapy, and 4) identification of additional biomarkers correlating with response to treatment with TAB001.
OVERVIEW: This is a Phase 1, open-label, 2-part (Part A = dose-escalation, Part B = multiple disease-specific cohort expansion) study to evaluate the safety, tolerability, PK, immunogenicity, and antitumor activity of toripalimab administered intravenously (IV) every 2 weeks (Q2W) of each 28-day cycle in Part A or 240 mg IV every 3 weeks (Q3W) of each 42-day cycle in Part B in adults with advanced solid tumors with disease progression following standard therapy or for which no standard therapy existed.
Part A used a 3+3 design and enrolled cohorts of 3-6 patients sequentially at escalating doses of 80, 240 an 480 mg Q2W to determine the MTD or MFD and the RP2D; up to 18 patients could be enrolled in the dose-escalation phase. Once a dose-limiting toxicity (DLT) occurred among the first 3 patients, the dose cohort would be expanded to a total of 6 patients. Patients who did not complete the 28-day DLT evaluation period for reasons other than toxicity were to be replaced. Dose escalation was to continue to the highest planned dose level (480 mg) or until identification of the MTD or the MFD. In addition, any cohort that had not exceeded the MTD could be expanded up to a maximum of 10 patients for further evaluation of safety and efficacy. Part A enrollment was limited to immunotherapy-naïve patients (defined as no prior exposure to immunotherapy, including but not limited to, anti-CTLA-4, anti-PD-1, or anti-PD-L1 antibodies; prior use of tumor vaccines was permitted).
In Part B, toripalimab administered at the RP2D, based on Part A, was to be evaluated for safety and antitumor activity. Enrollment was limited to patients who had not received a prior anti-PD-1, anti-PD-L1, or anti-PD-L2 antibody. Up to 280 patients with specified solid tumors could be enrolled in the cohort expansion phase with a maximum of 40 patients to be enrolled in each disease-specific cohort with the exception of the sarcoma cohort, in which a maximum of 80 patients could be enrolled.
The tumor status of all patients was to be evaluated by the investigator according to the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 and the immune-related Response Evaluation Criteria in Solid Tumors (irRECIST). In Part A, radiological assessment of tumor response status was to be performed approximately every 8 weeks (± 10 days) for the first 12 months and approximately every 12 weeks (± 10 days) thereafter, unless the investigator determined it was more appropriate to continue the radiological assessments approximately every 8 weeks. In Part B, radiological assessment of tumor response status was to be performed approximately every 9 weeks (± 10 days) for the first 12 months and approximately every 18 weeks (± 10 days) thereafter, unless the investigator determined it was more appropriate to continue the radiological assessments approximately every 9 weeks.
Patients received toripalimab Q2W (± 2 days) in Part A or Q3W (± 2 days) in Part B, until documentation of confirmed progressive disease (a repeat scan was to be conducted within 4 weeks ± 2 days in Part A and within 6 weeks ± 2 days in Part B to confirm disease progression per irRECIST), unacceptable toxicity, withdrawal of consent, intercurrent illness preventing further administration of toripalimab, or if the investigator considered it in the best interest of the patient to discontinue toripalimab. If feasible, all patients were to be followed for progression-free survival (PFS) and overall survival (OS) (Part B only) until the end of the study.
AEs and serious adverse events (SAEs) were to be captured from the start of the first dose of toripalimab through 90 days after the last dose of toripalimab unless a patient received another experimental or anticancer therapy, in which case only related AEs or SAEs were to be collected through 90 days after the last dose of toripalimab.
Serum samples and tumor tissues were collected for PK, pharmacodynamics, anti-drug antibody (ADA) and biomarkers determination during the study.
1,873 studies on the registry are indexed under Esophageal Neoplasms; 501 are open to participants now.
This study's enrollment of 184 is above the median of 58 across 1,347 interventional studies indexed under Esophageal Neoplasms.
Browse Esophageal Neoplasms studies →TopAlliance Biosciences is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Part B, must have a histologically or cytologically documented diagnosis of esophageal or gastric carcinoma, nasopharyngeal carcinoma (NPC), hepatocellular carcinoma (HCC), both soft tissue sarcoma (excluding leiomyosarcoma), chondrosarcoma, or with agreement of the sponsor, or other tumors who have received at least one line of standard systemic therapy for their respective tumor type in the metastatic setting with progressive locally advanced or metastatic disease that is not amenable to definitive local therapy with curative intent. Patient with MSI-H/dMMR Tumors are eligible to enroll.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
PD-1, PD-L1 and CTLA-4 are proteins that act as brakes on the immune system. Checkpoint inhibitors, such as pembrolizumab or nivolumab, block them so the immune system can attack a cancer.
PD-L1 can also be measured in a tumour sample. Some scores count stained tumour cells only (TPS); others also count certain immune cells (CPS). The study names the score and the minimum it uses.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
RECIST
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
PD-1, PD-L1 and checkpoint inhibitors
PD-1, PD-L1 and CTLA-4 are proteins that act as brakes on the immune system. Checkpoint inhibitors, such as pembrolizumab or nivolumab, block them so the immune system can attack a cancer.
PD-L1 can also be measured in a tumour sample. Some scores count stained tumour cells only (TPS); others also count certain immune cells (CPS). The study names the score and the minimum it uses.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Brain metastases
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Live vaccines
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Sequential dose escalation (3+3)
Biological: Part A
Indication specific dose cohorts: Esophageal Cancer, Gastric Cancer/GEJ, Biliary Tract Cancer, Soft Tissue Sarcoma (excluding Leiomyosarcoma) or Chondrosarcoma, Neuroendocrine Cancer, and other tumors (including Nasopharyngeal Cancer (NPC), Hepatocellular Carcinoma (HCC), MSI-H/dMMR tumors).
Biological: Part B
Part A: 80, 240, and 480 mg IV every 14 days.
Also known as: Toripalimab, TAB001, JS001
Part B: 240 mg IV every 3 weeks
Also known as: Toripalimab, TAB001, JS001
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
To assess the number of treatment-related adverse events in the toripalimab arm as assessed by CTCAE v4.0
Time frame: Through study completion, an estimated period of approximately 2 years.
Objective Response Rate (ORR)
The treatment effect of Toripalimab was assessed using RECIST 1.1 to determine objective response rate. ORR was defined and analyzed as the number of subjects achieving BOR of CR or PR, divided by the number of treated subjects. Subjects without at least one post-baseline radiological assessment were treated as non-responders. ORR was presented by dose cohort in Part A and tumor type in Part B, with 95% confidence intervals.
Time frame: Every 8 weeks (Part A) or every 9 weeks (Part B) through study completion, an estimated duration of 2 years.
Disease Control Rate (DCR)
The treatment effect of Toripalimab was assessed using RECIST 1.1 to determine objective response rate. ORR was defined and analyzed as the number of subjects achieving BOR of CR, PR, or SD divided by the number of treated subjects. Subjects without at least one post-baseline radiological assessment were treated as non-responders. DCR was presented by dose cohort in Part A and tumor type in Part B, with 95% confidence intervals.
Time frame: Every 8 weeks (Part A) or every 9 weeks (Part B) through study completion, an estimated duration of 2 years.
Progression-Free Survival (PFS)
The treatment effect of Toripalimab was assessed using RECIST 1.1 to determine progression-free survival time. PFS was defined as the time from the first dose of toripalimab to the first PD or death due to any cause, whichever occurred first and was analyzed separately for each cohort in Part B only,
Time frame: Every 8 weeks (Part A) or every 9 weeks (Part B) through study completion, an estimated duration of 2 years.
Overall Survival (OS)
The treatment effect of Toripalimab was assessed using RECIST 1.1 to determine overall survival (OS) analysis by tumor type in Part B. OS was defined as the time from date the first dose of toripalimab until death due to any cause. OS time for subjects not achieving the endpoint was censored at the last known alive date.
Time frame: Through study completion, an estimated duration of 2 years.
This study was initiated on 21 February 2018 (first patient signed the informed consent form) and completed on 07 June 2022. This study was conducted and screened patients in 14 sites in the United States.
| Milestone | Toripalimab 80 mg Repeat Dose Every 14 Days | Toripalimab 240 mg Repeat Dose Every 14 Days | Toripalimab 480 mg Repeat Dose Every 14 Days | Biliary Tract Cancer | Sarcoma | Esophageal Cancer | Gastric Cancer | Neuroendocrine Cancer | Other Tumors |
|---|---|---|---|---|---|---|---|---|---|
| Started | 3 | 8 | 7 | 42 | 59 | 11 | 29 | 22 | 3 |
| Completed | 0 | 0 | 2 | 8 | 7 | 1 | 3 | 6 | 1 |
| Not completed | 3 | 8 | 5 | 34 | 52 | 10 | 26 | 16 | 2 |
To assess the number of treatment-related adverse events in the toripalimab arm as assessed by CTCAE v4.0
| Participants | Part A: 80 mg | Part A: 240 mg | Part A: 480 mg | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophageal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine |
|---|---|---|---|---|---|---|---|---|---|
| Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 | 2 | 5 | 5 | 33 | 2 | 7 | 15 | 27 | 14 |
The treatment effect of Toripalimab was assessed using RECIST 1.1 to determine objective response rate. ORR was defined and analyzed as the number of subjects achieving BOR of CR or PR, divided by the number of treated subjects. Subjects without at least one post-baseline radiological assessment were treated as non-responders. ORR was presented by dose cohort in Part A and tumor type in Part B, with 95% confidence intervals.
| Participants | Part A: 80 mg | Part A: 240 mg | Part A: 480 mg | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophageal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine |
|---|---|---|---|---|---|---|---|---|---|
| Objective Response Rate (ORR) | 0 | 0 | 0 | 5 | 0 | 3 | 1 | 2 | 3 |
The treatment effect of Toripalimab was assessed using RECIST 1.1 to determine objective response rate. ORR was defined and analyzed as the number of subjects achieving BOR of CR, PR, or SD divided by the number of treated subjects. Subjects without at least one post-baseline radiological assessment were treated as non-responders. DCR was presented by dose cohort in Part A and tumor type in Part B, with 95% confidence intervals.
| Participants | Part A: 80 mg | Part A: 240 mg | Part A: 480 mg | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophageal Tumors | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine |
|---|---|---|---|---|---|---|---|---|---|
| Disease Control Rate (DCR) | 0 | 5 | 6 | 25 | 1 | 4 | 7 | 17 | 11 |
The treatment effect of Toripalimab was assessed using RECIST 1.1 to determine progression-free survival time. PFS was defined as the time from the first dose of toripalimab to the first PD or death due to any cause, whichever occurred first and was analyzed separately for each cohort in Part B only,
| months | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophageal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine |
|---|---|---|---|---|---|---|
| Progression-Free Survival (PFS) | 2.2 (1.8 to 6.2) | 1.4 (1.0 to 4.1) | 2.1 (2.1 to 6.2) | 2.1 (1.5 to 4.0) | 2.1 (1.8 to 4.1) | 6.4 (1.5 to NA) |
The treatment effect of Toripalimab was assessed using RECIST 1.1 to determine overall survival (OS) analysis by tumor type in Part B. OS was defined as the time from date the first dose of toripalimab until death due to any cause. OS time for subjects not achieving the endpoint was censored at the last known alive date.
| months | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophageal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine |
|---|---|---|---|---|---|---|
| Overall Survival (OS) | 4.9 (3.71 to 6.54) | 1.4 (1.12 to NA) | 5.7 (0.79 to 21.72) | 3.5 (2.33 to 6.93) | 4.4 (3.48 to 4.86) | 4.2 (2.60 to 8.25) |
Collected over All the TEAEs from first dose to 90 days after the last dose of toripalimab were included in the safety evaluation, an average duration of six months, up to two years.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Toripalimab 80 mg Every 14 Days | 1/3 (33.3%) | 2/3 (66.7%) | 2/3 (66.7%) |
| Toripalimab 240 mg Every 14 Days | 2/8 (25%) | 2/8 (25%) | 5/8 (62.5%) |
| Toripalimab 480 mg Every 14 Days | 0/7 (0%) | 4/7 (57.1%) | 5/7 (71.4%) |
| Sarcoma | 12/59 (20.3%) | 23/59 (39%) | 33/59 (55.9%) |
| Other Tumors | 0/3 (0%) | 1/3 (33.3%) | 2/3 (66.7%) |
| Esophogeal | 2/11 (18.2%) | 3/11 (27.3%) | 7/11 (63.6%) |
| Gastric/GEJ | 9/29 (31%) | 11/29 (37.9%) | 15/29 (51.7%) |
| Biliary Tract | 12/42 (28.6%) | 23/42 (54.8%) | 27/42 (64.3%) |
| Neuroendocrine | 3/22 (13.6%) | 6/22 (27.3%) | 14/22 (63.6%) |
| Event | Toripalimab 80 mg Every 14 Days | Toripalimab 240 mg Every 14 Days | Toripalimab 480 mg Every 14 Days | Sarcoma | Other Tumors | Esophogeal | Gastric/GEJ | Biliary Tract | Neuroendocrine |
|---|---|---|---|---|---|---|---|---|---|
| Respiratory, thoracic and mediastinal disordersRespiratory, thoracic and mediastinal disorders | 1/3 | 0/8 | 0/7 | 7/59 | 0/3 | 0/11 | 5/29 | 2/42 | 0/22 |
| Metabolism and Nutrition DisordersMetabolism and nutrition disorders | 0/3 | 0/8 | 1/7 | 3/59 | 1/3 | 0/11 | 2/29 | 1/42 | 2/22 |
| Nervous System DisordersNervous system disorders | 1/3 | 0/8 | 0/7 | 3/59 | 0/3 | 0/11 | 0/29 | 0/42 | 0/22 |
| Gastrointestinal DisordersGastrointestinal disorders | 0/3 | 0/8 | 1/7 | 1/59 | 0/3 | 1/11 | 6/29 | 5/42 | 1/22 |
| Infections and InfestationsInfections and infestations | 0/3 | 0/8 | 1/7 | 4/59 | 0/3 | 2/11 | 1/29 | 8/42 | 0/22 |
| Renal and Urinary DisordersRenal and urinary disorders | 0/3 | 0/8 | 1/7 | 1/59 | 0/3 | 0/11 | 0/29 | 3/42 | 1/22 |
| Musculoskeletal and Connective Tissue DisordersMusculoskeletal and connective tissue disorders | 0/3 | 1/8 | 0/7 | 0/59 | 0/3 | 0/11 | 0/29 | 0/42 | 0/22 |
| Reproductive System and Breast DisordersReproductive system and breast disorders | 0/3 | 1/8 | 0/7 | 0/59 | 0/3 | 0/11 | 0/29 | 0/42 | 0/22 |
| General DisordersGeneral disorders | 0/3 | 0/8 | 0/7 | 3/59 | 0/3 | 0/11 | 1/29 | 1/42 | 2/22 |
| Vascular DisordersVascular disorders | 0/3 | 0/8 | 0/7 | 3/59 | 0/3 | 0/11 | 0/29 | 0/42 | 0/22 |
| Event | Toripalimab 80 mg Every 14 Days | Toripalimab 240 mg Every 14 Days | Toripalimab 480 mg Every 14 Days | Sarcoma | Other Tumors | Esophogeal | Gastric/GEJ | Biliary Tract | Neuroendocrine |
|---|---|---|---|---|---|---|---|---|---|
| Skin and Subcutaneous DisorderSkin and subcutaneous tissue disorders | 0/3 | 2/8 | 2/7 | 9/59 | 2/3 | 0/11 | 6/29 | 2/42 | 5/22 |
| Blood and Lymphatic DisordersBlood and lymphatic system disorders | 1/3 | 3/8 | 1/7 | 5/59 | 0/3 | 0/11 | 2/29 | 6/42 | 1/22 |
| InvestigationsInvestigations | 1/3 | 0/8 | 1/7 | 12/59 | 0/3 | 3/11 | 4/29 | 12/42 | 3/22 |
| Endocrine DisordersEndocrine disorders | 0/3 | 1/8 | 0/7 | 10/59 | 1/3 | 2/11 | 1/29 | 1/42 | 2/22 |
| Nervous System DisordersNervous system disorders | 1/3 | 1/8 | 0/7 | 1/59 | 0/3 | 1/11 | 5/29 | 1/42 | 0/22 |
| General DisordersGeneral disorders | 0/3 | 2/8 | 1/7 | 10/59 | 0/3 | 3/11 | 4/29 | 11/42 | 6/22 |
| Gastrointestinal DisordersGastrointestinal disorders | 0/3 | 2/8 | 1/7 | 10/59 | 0/3 | 3/11 | 4/29 | 11/42 | 4/22 |
| Metabolism and nutrition disordersMetabolism and nutrition disorders | 0/3 | 1/8 | 1/7 | 5/59 | 0/3 | 2/11 | 4/29 | 4/42 | 2/22 |
| Injury, Poisoning, and Procedural ComplicationsInjury, poisoning and procedural complications | 0/3 | 0/8 | 1/7 | 1/59 | 0/3 | 0/11 | 1/29 | 0/42 | 1/22 |
| Cardiac DisordersCardiac disorders | 0/3 | 0/8 | 1/7 | 0/59 | 0/3 | 0/11 | 0/29 | 0/42 | 0/22 |
| Age, Categorical(Participants) | Part A: Toripalimab 80 mg Every 14 Days | Part A: Toripalimab 240 mg Every 14 Days | Part A: Toripalimab 480 mg Every 14 Days | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophogeal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 2 | 6 | 5 | 37 | 2 | 6 | 11 | 25 | 9 | 103 |
| >=65 years | 1 | 2 | 2 | 22 | 1 | 5 | 18 | 17 | 13 | 81 |
| Age, Continuous(years) | Part A: Toripalimab 80 mg Every 14 Days | Part A: Toripalimab 240 mg Every 14 Days | Part A: Toripalimab 480 mg Every 14 Days | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophogeal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Median | 62 (62 to 69) | 60.5 (29 to 66) | 55 (46 to 73) | 60 (21 to 85) | 40 (38 to 75) | 63 (53 to 78) | 66 (29 to 83) | 60.5 (35 to 85) | 65.5 (34 to 84) | 62 (21 to 85) |
| Sex: Female, Male(Participants) | Part A: Toripalimab 80 mg Every 14 Days | Part A: Toripalimab 240 mg Every 14 Days | Part A: Toripalimab 480 mg Every 14 Days | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophogeal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Female | 1 | 5 | 5 | 26 | 1 | 2 | 8 | 28 | 4 | 80 |
| Male | 2 | 3 | 2 | 33 | 2 | 9 | 21 | 14 | 18 | 104 |
| Ethnicity (NIH/OMB)(Participants) | Part A: Toripalimab 80 mg Every 14 Days | Part A: Toripalimab 240 mg Every 14 Days | Part A: Toripalimab 480 mg Every 14 Days | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophogeal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 0 | 1 | 0 | 7 | 2 | 1 | 1 | 1 | 1 | 14 |
| Not Hispanic or Latino | 3 | 7 | 7 | 49 | 1 | 9 | 28 | 40 | 20 | 164 |
| Unknown or Not Reported | 0 | 0 | 0 | 3 | 0 | 1 | 0 | 1 | 1 | 6 |
| Region of Enrollment(Participants) | Part A: Toripalimab 80 mg Every 14 Days | Part A: Toripalimab 240 mg Every 14 Days | Part A: Toripalimab 480 mg Every 14 Days | Part B: Sarcoma | Part B: Other Tumors | Part B: Esophogeal | Part B: Gastric/GEJ | Part B: Biliary Tract | Part B: Neuroendocrine | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| United States | 3 | 8 | 7 | 59 | 3 | 11 | 29 | 42 | 22 | 184 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Feb 2018 to primary completion in Jun 2022, 51 months.
Results were first posted in Nov 2024, 2 years 5 months after primary completion.
Of 77 industry-sponsored phase 1 interventional studies under Esophageal Neoplasms, half reached primary completion within 51 months of starting (the median), counting those still running; a quarter within 34 and three quarters within 70. This study's 51 months is close to that.
Counting every sponsor, half of 125 studies reached it within 48 months; a quarter within 29 and three quarters within 76.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.
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