Advanced Solid TumorPhase 1NCT04906473
Safety,Tolerability, Pharmacokinetic,Pharmacodynamics and Efficacy of KY100001 in Patients With Advanced Solid Tumors
A Phase 1 study testing KY100001 in people with Advanced Solid Tumor, run by Kunming Pharmaceuticals, Inc..
Trial summary
to assess the safety,tolerability, pharmacokinetic,pharmacodynamics and efficacy of KY100001 in patients with advanced solid tumors Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Advanced Solid Tumor
- What is being tested?
- KY100001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 109 participants
- Where
- No sites listed yet
- Study dates
- 31 Jul 2021to 31 Oct 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1) Participate in the study voluntarily and sign the informed consent;
- 2) 18≤ age ≤75 years old, gender is not limited;
- 3) Dose escalation stage: patients with advanced solid tumor proven histologically or cytologically without standard treatment regimen, or with recurrence or progression after standard treatment, WHO are ineffective or intolerant to standard treatment regimen (for patients with glioma, WHO grade II, III and IV can be included);
- 4) Dose expansion phase IDH1 mutation positivity in part A/B: Non-cholangiocarcinoma/glioma: Any of the following conditions are allowed: A) Subjects provide genetic test report to prove IDH1 mutation positivity (NGS or qPCR);B) Subjects who voluntarily undergo tumor biopsy and/or blood sample collection for genetic testing and the test results are positive;C) Subjects can provide the last FFPE sample or pathological slides (at least 10 consecutive white slides) and voluntarily accept blood sample collection for genetic testing, and the test result is positive;Patients with cholangiocarcinoma/glioma: Any of the following conditions are allowed: a) Subjects who provide prior genetic testing report demonstrating IDH1 mutation positivity (NGS or qPCR);B) Subjects can provide the most recent FFPE sample or pathological section (at least 10 consecutive white films) or tissue biopsy sample (patients with cholangiocarcinoma should also voluntarily accept blood sample collection) for genetic testing, and the test result is positive;
- 5) Dose Expansion Phase A: Patients with advanced solid tumors with IDH1 gene mutation positive, proven histologically or cytologically, without standard treatment regimens, or relapse or progression after standard treatment, ineffective or intolerant to standard treatment regimens;
- 6) Dose expansion phase B: patients with cholangiocarcinoma/high-grade glioma (WHO grade III \&IV) with IDH1 gene mutation positive, histologically or cytologically proven, WHO do not have a standard treatment regimen, or WHO relapse or progress after standard treatment, are ineffective or intolerant to the standard treatment regimen.Cohort 1: a) Histologically proven stage II-IV cholangiocarcinoma with IDH1 gene mutation positive, not eligible for radical resection, transplantation, or ablation;B) disease progression after treatment with a fluorouracil or gemcitabine basal regimen;C) at least one measurable lesion that has not previously undergone radiotherapy, chemoembolization, radioembolization or other local ablation procedures;Cohort 2: a) high-grade gliomas with IDH1 gene mutation positivity (WHO grade III \&IV);B) The number of disease progression ≤2 times during screening;C) At least one previous CEMRI + perfusion MRI;D) at least one measurable lesion ≥1cm (according to RANO criteria);
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- 7) At least one measurable lesion (non-glioma according to RECIST v1.1 criteria;Brain glioma according to RANO criteria);
- 8) Dose increasing stage: non-glioma patients (Eastern Cooperative Cancer Group (ECOG) score: 0-2) glioma patients (KPS score ≥50);Dose extension stage: non-glioma patients (ECOG score: 0-1 points);Glioma patients (KPS score ≥50);
- 9) Expected survival ≥3 months;
- 10) Within 7 days prior to the administration of the study drug, laboratory tests have met the following criteria.Absolute neutrophils count (ANC) ≥ 1.5×109 /L;Platelet count ≥ 80×109 /L;Hemoglobin ≥ 90 g/L;Serum creatinine ≤ 1.5x upper normal range (ULN) or creatinine clearance rate (CRCL) ≥ 60mL/min (estimated according to Cockcroft-Gault formula);Total bilirubin ≤ 1.5 X ULN or ≤ 2 X ULN (for patients with liver metastasis);AST and ALT≤ 2.5 X ULN or ≤ 5 X ULN (for patients with liver metastasis);Alkaline phosphatase ≤ 2.5 X ULN or ≤ 5 X ULN (for patients with bone metastases and/or suspected disease related liver or bile duct involvement);International Normalized Ratio or Prothrombin Time≤ 1.3x ULN;Partial activated thromboplastin time (APTT) ≤ 1.5 X ULN;
- 11) Fertile men and women of reproductive age must agree to use reliable contraception from the time of signing the informed consent until 180 days after the last dose of the study drug.Women of childbearing age include pre-menopausal women and women within 2 years after menopause.Blood pregnancy test results of women of reproductive age must be negative no more than 7 days before the first study drug administration.
Who cannot join
- 1) Allergic constitution, or previous history of severe allergy, or known allergy to the active ingredients and excipients of the study drug;
- 2) Surgical therapy, chemotherapy, radiation therapy, immunotherapy, molecular targeted therapy, or any other anti-tumor therapy within 4 weeks prior to the first administration of the study drug;
- 3) Non-glioma patients: untreated patients with brain metastases who have symptoms or require treatment to control their symptoms;Or had used any radiation, surgical or other treatment, including treatment to control symptoms, within 2 months prior to the first administration of the study drug;Patients with glioma: Patients who received an unsteady dose of dexamethasone exceeding the equivalent dose of 5mg/ day 5 days before MRI screening;
- 4) Had any of the following heart conditions: a) Had grade III-IV heart failure according to the New York Heart Association's cardiac function rating within 28 days prior to the first administration of the study drug, or had left ventricular ejection fraction (LVEF) ≤50% in Echo within 7 days prior to the first administration of the study drug;B) The history of myocardial infarction in the previous 6 months was screened;C) known unstable angina;D) known severe or uncontrolled ventricular arrhythmias;E) Screening-phase Fridericia calibrated QT interval (QTCF) ≥450 ms (male), ≥470 ms (female), or other factors that increase the risk of prolonged QTC or arrhythmic events (such as heart failure, low potassium, and a family history of long QT syndrome);F) In the screening period, there was hypertension that was still poorly controlled after drug treatment (systolic blood pressure ≥160mmHg, diastolic blood pressure ≥ 100mmHg);
- 5) Difficulty in swallowing or suffering from gastrointestinal diseases or other malabsorption conditions that affect drug absorption, such as intestinal obstruction, Crohn's disease, ulcerative colitis, short bowel syndrome, gastric emptydisturbance;Or severe gastrointestinal related toxicity that does not recover below grade 2 prior to initial administration;Or clinically significant or acute gastrointestinal disease;
- 6) Previous interstitial pulmonary disease, pulmonary fibrosis, drug-induced interstitial pulmonary disease, or radiation pneumonia disease or history;
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- 7) Evidence of severe or uncontrolled liver or kidney disease;
- 8) Hepatitis B virus infection (HBcAb positive and HBV DNA lower than the detection limit could be included);Or people infected with hepatitis C virus (defined as HCV antibody positive);Or persons infected with the human immunodeficiency virus (defined as HIV-positive);
- 9) Active severe infection requiring anti-infective treatment or unexplained fever over 38℃ within 28 days prior to the first administration of the study drug;
- 10) Prior to the initial administration of the study drug, any toxicity from prior antitumor therapy had not returned to CTCAE 5.0 grade ≤1 (except for grade 2 alopecia, which could be included in secondary peripheral neuropathy determined by the investigator and medical examiner to be primary residual toxicity or stable from prior chemotherapy);
- 11) Participated in other clinical trials within 28 days prior to the first administration of the study drug;
- 12) Use of CYP2C8, CYP3A strong inhibitor or inducer within 2 weeks before the first administration of the drug in this study;
- 13) Use of drugs known to extend the Qt /QTc interval within 2 weeks before the first administration of the study drug;
- 14) Pregnant or lactating women;
- 15) The investigator believes that there is any abnormal clinical or laboratory examination or other reasons and he/she is not suitable to participate in the clinical investigator.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: A Phase Ⅰ Clinical Study of Single-arm, Open, Single/Multiple Dose Escalation and Dose Extension to Assess the Safety, Tolerability, Pharmacokinetic, Pharmacodynamics and Efficacy of KY100001 in Patients With Advanced Solid Tumors
to assess the safety,tolerability, pharmacokinetic,pharmacodynamics and efficacy of KY100001 in patients with advanced solid tumors
- Conditions
- Advanced Solid Tumor
- Being tested
- KY100001
- Keywords
- biliary duct cancer, glioma
- Medical subject headings
- Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KY100001Drug
KY100001; Tablet; Oral route; Dose escalation and dose extension
| Study group | Role | What they receive |
|---|---|---|
| KY100001 | Experimental | KY100001; Tablet; Oral route; Dose escalation and dose extension Receives: Drug: KY100001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with abnormal vital signs
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of abnormal vital signs
Measured: through study completion, an average of 12 weeks
Number of subjects with abnormal physical examination
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of abnormal physical examination
Measured: through study completion, an average of 12 weeks
Number of subjects with abnormal laboratory examination
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of abnormal laboratory examination
Measured: through study completion, an average of 12 weeks
Number of subjects with abnormal electrocardiogram
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of abnormal electrocardiogram
Measured: through study completion, an average of 12 weeks
Change from baseline in ECOG score
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of ECOG score
Measured: through study completion, an average of 12 weeks
Secondary outcomes (8)
Pharmacokinetics of KY100001: Cmax
Pharacokinetic evaluation in terms of Cmax
Measured: 4 weeks
Pharmacokinetics of KY100001: Tmax
Pharacokinetic evaluation in terms of Tmax
Measured: 4 weeks
Pharmacokinetics of KY100001: T1/2
Pharacokinetic evaluation in terms of T1/2
Measured: 4 weeks
Pharmacokinetics of KY100001: AUC
Pharacokinetic evaluation in terms of AUC
Measured: 4 weeks
The relationship between 2-hydroxyglutaric acid (2-HG) level and curative effect
biomarker evaluation in terms of 2-hydroxyglutaric acid (2-HG)
Measured: 4 weeks
Objective Response Rate (ORR)
effectiveness evaluation in terms of Objective Response Rate (ORR)
Measured: through study completion, an average of 12 weeks
Overall Survival (OS)
effectiveness evaluation in terms of Overall Survival (OS)
Measured: through study completion, an average of 12 weeks
Progression-Free Survival (PFS)
effectiveness evaluation in terms of Progression-Free Survival (PFS)
Measured: through study completion, an average of 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2021
- Study start31 Jul 2021 (estimated)
- Primary completion31 Dec 2022 (estimated)
- Study completion31 Oct 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Solid TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Single-arm, open, single/multiple dose escalation (100mg/d, 200mg/d, 350mg/d, 550mg/d, 750mg/d) and dose Extension
- Masking
- None (open label)
- Enrolment
- 109 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kunming Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lin Shen, prof., Principal investigator, Peking University Cancer Hospital & Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04906473
- Sponsor's protocol ID
- KPC/KY100001/C101
- Status last verified
- May 2021
- First submitted
- 18 May 2021
- Last updated
- 28 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ⅰ Clinical Study of Single-arm, Open, Single/Multiple Dose Escalation and Dose Extension to Assess the Safety, Tolerability, Pharmacokinetic, Pharmacodynamics and Efficacy of KY100001 in Patients With Advanced Solid Tumors
- ClinicalTrials.gov ID
- NCT04906473
- Acronym / sponsor ID
- KPC/KY100001/C101
- Conditions
- Advanced Solid Tumor
- Keywords
- biliary duct cancer, glioma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 109 participants (estimated)
- Lead sponsor
- Kunming Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Jul 2021 (estimated)
- Primary completion
- 31 Dec 2022 (estimated)
- Study completion
- 31 Oct 2023 (estimated)
- First posted
- 28 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
to assess the safety,tolerability, pharmacokinetic,pharmacodynamics and efficacy of KY100001 in patients with advanced solid tumors
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single-arm, open, single/multiple dose escalation (100mg/d, 200mg/d, 350mg/d, 550mg/d, 750mg/d) and dose Extension
| Arm | Type | Receives |
|---|---|---|
| KY100001 | Experimental | Drug: KY100001KY100001; Tablet; Oral route; Dose escalation and dose extension |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 1) Participate in the study voluntarily and sign the informed consent;
- 2) 18≤ age ≤75 years old, gender is not limited;
- 3) Dose escalation stage: patients with advanced solid tumor proven histologically or cytologically without standard treatment regimen, or with recurrence or progression after standard treatment, WHO are ineffective or intolerant to standard treatment regimen (for patients with glioma, WHO grade II, III and IV can be included);
- 4) Dose expansion phase IDH1 mutation positivity in part A/B: Non-cholangiocarcinoma/glioma: Any of the following conditions are allowed: A) Subjects provide genetic test report to prove IDH1 mutation positivity (NGS or qPCR);B) Subjects who voluntarily undergo tumor biopsy and/or blood sample collection for genetic testing and the test results are positive;C) Subjects can provide the last FFPE sample or pathological slides (at least 10 consecutive white slides) and voluntarily accept blood sample collection for genetic testing, and the test result is positive;Patients with cholangiocarcinoma/glioma: Any of the following conditions are allowed: a) Subjects who provide prior genetic testing report demonstrating IDH1 mutation positivity (NGS or qPCR);B) Subjects can provide the most recent FFPE sample or pathological section (at least 10 consecutive white films) or tissue biopsy sample (patients with cholangiocarcinoma should also voluntarily accept blood sample collection) for genetic testing, and the test result is positive;
- 5) Dose Expansion Phase A: Patients with advanced solid tumors with IDH1 gene mutation positive, proven histologically or cytologically, without standard treatment regimens, or relapse or progression after standard treatment, ineffective or intolerant to standard treatment regimens;
Key exclusion criteria (15 listed)
- 1) Allergic constitution, or previous history of severe allergy, or known allergy to the active ingredients and excipients of the study drug;
- 2) Surgical therapy, chemotherapy, radiation therapy, immunotherapy, molecular targeted therapy, or any other anti-tumor therapy within 4 weeks prior to the first administration of the study drug;
- 3) Non-glioma patients: untreated patients with brain metastases who have symptoms or require treatment to control their symptoms;Or had used any radiation, surgical or other treatment, including treatment to control symptoms, within 2 months prior to the first administration of the study drug;Patients with glioma: Patients who received an unsteady dose of dexamethasone exceeding the equivalent dose of 5mg/ day 5 days before MRI screening;
- 4) Had any of the following heart conditions: a) Had grade III-IV heart failure according to the New York Heart Association's cardiac function rating within 28 days prior to the first administration of the study drug, or had left ventricular ejection fraction (LVEF) ≤50% in Echo within 7 days prior to the first administration of the study drug;B) The history of myocardial infarction in the previous 6 months was screened;C) known unstable angina;D) known severe or uncontrolled ventricular arrhythmias;E) Screening-phase Fridericia calibrated QT interval (QTCF) ≥450 ms (male), ≥470 ms (female), or other factors that increase the risk of prolonged QTC or arrhythmic events (such as heart failure, low potassium, and a family history of long QT syndrome);F) In the screening period, there was hypertension that was still poorly controlled after drug treatment (systolic blood pressure ≥160mmHg, diastolic blood pressure ≥ 100mmHg);
- 5) Difficulty in swallowing or suffering from gastrointestinal diseases or other malabsorption conditions that affect drug absorption, such as intestinal obstruction, Crohn's disease, ulcerative colitis, short bowel syndrome, gastric emptydisturbance;Or severe gastrointestinal related toxicity that does not recover below grade 2 prior to initial administration;Or clinically significant or acute gastrointestinal disease;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KY100001KY100001; Tablet; Oral route; Dose escalation and dose extension | Drug | KY100001 |
Primary outcome
Number of subjects with abnormal vital signs
Time frame: through study completion, an average of 12 weeks
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of abnormal vital signs
Number of subjects with abnormal physical examination
Time frame: through study completion, an average of 12 weeks
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of abnormal physical examination
Number of subjects with abnormal laboratory examination
Time frame: through study completion, an average of 12 weeks
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of abnormal laboratory examination
Number of subjects with abnormal electrocardiogram
Time frame: through study completion, an average of 12 weeks
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of abnormal electrocardiogram
Change from baseline in ECOG score
Time frame: through study completion, an average of 12 weeks
To evaluate the safety of KY100001 in subjects with advanced solid cancer in terms of ECOG score
8 secondary outcomes
Pharmacokinetics of KY100001: Cmax
Time frame: 4 weeks
Pharmacokinetics of KY100001: Tmax
Time frame: 4 weeks
Pharmacokinetics of KY100001: T1/2
Time frame: 4 weeks
Pharmacokinetics of KY100001: AUC
Time frame: 4 weeks
The relationship between 2-hydroxyglutaric acid (2-HG) level and curative effect
Time frame: 4 weeks
Objective Response Rate (ORR)
Time frame: through study completion, an average of 12 weeks
Overall Survival (OS)
Time frame: through study completion, an average of 12 weeks
Progression-Free Survival (PFS)
Time frame: through study completion, an average of 12 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lin Shen, prof. principal investigator
Peking University Cancer Hospital & Institute
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing KY100001 in people with Advanced Solid Tumor, run by Kunming Pharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 28 May 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Qiang Zhang, bachelor (0871-68319868-3052, qiang.zhang@kpc.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of subjects with abnormal vital signs (time frame: through study completion, an average of 12 weeks). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2021; AzTrials last refreshed its copy on 8 Nov 2026.