Acute Myeloid Leukemia (AML)Phase 1NCT03125876
A Phase Ib Study of CT053PTSA in Relapsed / Refractory Acute Myeloid Leukemia (AML)
A Phase 1 study testing CT053PTSA in people with Acute Myeloid Leukemia (AML), run by Sunshine Lake Pharma Co., Ltd. at 1 site in China.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of CT053PTSA in Relapsed/refractory AML patients with FLT3 gene mutation. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- What is being tested?
- CT053PTSA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 13 Nov 2017to 19 Aug 2019
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- study population
- documented acute myeloid leukemia according to World Health Organization（WHO） criteria(excluding acute promyelocytic leukemia), with Fms-like Tyrosine Kinase 3 (FLT3) gene mutation,refractory/relapsed after common or enhanced chemotherapy c) Recovered from toxicity of previous treatment d) Eastern Cooperative Oncology Group (ECOG) performance status of 0-1 e) Life expectation ≥ 12 weeks
- Subjects must have adequate organ function and meeting all of the following laboratory review before enrollment a)blood routine examination: WBC≤2000/mm3 b)liver function: Alanine aminotransferase (ALT) and Aspartate transaminase (AST) ≤2.5×upper limit of normal(ULN); serum bilirubin ≤ 1.5 × ULN c)renal function: Serum creatinine ≤ 1.5×ULN, or the creatinine clearance (CrCl)≥ 60 mL / min calculated by the Cockcroft-Gault formula d) electrolyte: serum potassium≥3.0mmol/L; serum calcium≥2.0 mmol/L e) abnormal serum amylase without symptom≤1.5 × ULN; serum Lipase ≤1.5× ULN f) coagulation function:fibrinogen≥1.0g/L; activated partial thromboplastin time( APTT)≦ULN+10s; prothrombin time(PT)≤ULN+3s g) no obvious organ dysfunction 3)subjects must volunteer to provide evidence of effective diagnosis prior to entry or to accept bone marrow puncture or biopsy for diagnosis, and accept bone marrow puncture or biopsy after treatment to evaluate the efficacy 4) Sign the informed consent
Who cannot join
- Subject meeting any of the following criteria will be excluded.
- treatment history
- chemotherapy, immunotherapy, radiotherapy, or major surgery within 4 weeks prior to administration;
- nitrosourea and mitomycin chemotherapy within 6 weeks prior to the administration;
- have taken live vaccines within 4 weeks prior to /or concurrent with the administration;
- received FLT3 or Axl inhibitors within 6 weeks prior to the administration;
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- have received a trial investigational product, or participated in other clinical trials within 4 weeks prior to administration
- disease history and surgery history
- documented promyelocytic leukemia (t (15; 17) (q22; q11) and / or promyelocytic leukemia(PML)/retinoic acid receptor alpha (RARa) positivity found in the chromosome, variant acute promyelocytic leukemia) b) with myeloid sarcoma or invasion of central nervous system; c) high blood pressure and not well controlled by drug (blood pressure ≥ 140/90 mmHg). Note: Blood pressure before the first administration (mean blood pressure of two measures that 24 hours interval or above ) should be controlled within 140/90 mmHg.
- Doppler ultrasound evaluation: left ventricular ejection fraction (LVEF) <50%; e) NCI CTCAE 4.03 ≥ 2 grade of arrhythmia, or corrected QT interval(QTc )> 450 ms ; patients with a history of torsion or congenital QT prolonged syndrome; f) any of the illness bellowed within 12 months prior to administration: myocardial infarction, severe or unstable angina, coronary artery bypass graft or peripheral artery bypass surgery, congestive heart failure, cerebrovascular events (including Transient ischemic attack); g) multiple factors that affect oral medication (eg, can not swallow, chronic diarrhea and intestinal obstruction, etc.); h)obvious tendency of gastrointestinal bleeding, including the following cases: local active ulcer lesion, and fecal occult blood test(≥++); melena or hematemesis within 2 months; gastrointestinal bleeding may occurs considered by investigator.
- i)history of immunodeficiency, other acquired or congenital immunodeficiency,history of organ transplantation; j) previous thyroid dysfunction,thyroid function can not be maintained at the normal range even have drug taken.
- k)Human immunodeficiency virus(HIV) positivity l) Hepatitis B surface antigen (HBsAg)positivity, and in the active phase(hepatitis B nucleic acid quantity≥ 1.00 × 103copies / ml); m) Hepatitis C antibody(Anti-HCV) positivity and in the active phase (hepatitis C nucleic acid quantity ≥1.00 × 102copies / ml) n) severe retinopathy or exfoliation judged by the Investigator; o) severe electrolyte imbalance judged by the investigator; p) active infectious disease judged by the investigator; q) other acute or chronic medical or psychological illnesses that are not suitable for clinical trials considered by the investigator or sponsor;
- Pregnant or lactating women or female and male with fertility plan. 4)the therapy and/or drug forbidden
- taking anticoagulant or vitamin K antagonist such as warfarin, heparin or other similar drugs;
- taking other anti-leukemia drugs at the same time, including traditional Chinese medicine (some Chinese medicine can not be used listed in Annex 4);
- taking drugs that will prolong QT interval(including Ia and III antiarrhythmic drugs);
- patients who need oxygen therapy every day;
- long-term use of corticosteroids (local inhalation excluded);
- 5)others
- history of Psychotropic drugs abuse and can not drop or the mentally disordered; b) be used to drink grapefruit juice or too much tea, coffee and / or caffeine-containing drink, can not stop during the trial(including Cycle 1 and subsequent treatment period) c) in the vegistrator's judgment, there is a serious accompany disease that jeopardizes patient's safety or interfere with the completion of the study.
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Trial locations and contacts
1 site in 1 country.
China1 site
Hospital of Blood Disease, Chinese Academy of Medical Sciences
Tianjin
About this study
Official title: A Phase Ib, Multi-center, Open, Dose-escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Ningetinib (CT053PTSA) in Relapsed / Refractory Acute Myeloid Leukemia (AML)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of CT053PTSA in Relapsed/refractory AML patients with FLT3 gene mutation.
Read the detailed description
It is a multi-center , open-label, dose escalation study conducted in 2 parts. Dose-escalation part: Subjects will receive oral CT053PTSA once daily repeatedly until disease progression or unacceptable toxicity occurs, each cycle is defined as 28 days. At least 3 subjects will receive CT053PTSA at each dose (60, 80, 100mg) for determination of Maximum Tolerated Dose(MTD) and Dose Limiting Toxicity (DLT) Dose reduction of CT053PTSA will be considered if study drug-related toxicities are observed in a subject after Cycle 1.
Expansion part:Expansion cohort might be set to further investigate the safety and efficacy of CT053PTSA at or lower MTD dose recommended by dose-escalation part.
- Conditions
- Acute Myeloid Leukemia (AML)
- Being tested
- CT053PTSA
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CT053PTSADrug
Also called Ningetinib
receive oral CT053PTSA once daily until disease progression or unacceptable toxicity occurs, each cycle is defined as 28 days
| Study group | Role | What they receive |
|---|---|---|
| CT053PTSA | Experimental | 60mg-100mg Receives: Drug: CT053PTSA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose (MTD)
Safety and Tolerability assessed through adverse events to determine maximum tolerated dose
Measured: day 1-28
Secondary outcomes (4)
Maximum observed plasma concentration (Cmax)
to assess the pharmacokinetic profile in patients with AML
Measured: On day 1,8,15,22,29
Time of maximum observed plasma concentration (Tmax)
to assess the pharmacokinetic profile in patients with AML
Measured: On day 1,8,15,22,29
Area under the plasma concentration time curve
to assess the pharmacokinetic profile in patients with AML
Measured: On day 1,8,15,22,29
Objective response rate (ORR)
based on investigator review of radiographic images
Measured: Through study completion, an expected average of 1.5 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2017
- Study start13 Nov 2017
- Primary completion19 Aug 2019
- Study completion19 Aug 2019
Why it stopped: Company strategy adjustment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sunshine Lake Pharma Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jianxiang Wang, Doctor, Study chair, Hospital of Blood Disease, Chinese Academy of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03125876
- Sponsor's protocol ID
- PCD-DCT053-16-002
- Status last verified
- Mar 2021
- First submitted
- 9 Apr 2017
- Last updated
- 24 Mar 2021
- Results posted
- No
- Data monitoring committee
- No
- 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 24 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ib, Multi-center, Open, Dose-escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Ningetinib (CT053PTSA) in Relapsed / Refractory Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT03125876
- Acronym / sponsor ID
- PCD-DCT053-16-002
- Conditions
- Acute Myeloid Leukemia (AML)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Company strategy adjustment
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Sunshine Lake Pharma Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 13 Nov 2017 (actual)
- Primary completion
- 19 Aug 2019 (actual)
- Study completion
- 19 Aug 2019 (actual)
- First posted
- 24 Apr 2017 (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.
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of CT053PTSA in Relapsed/refractory AML patients with FLT3 gene mutation.
Detailed description
It is a multi-center , open-label, dose escalation study conducted in 2 parts. Dose-escalation part: Subjects will receive oral CT053PTSA once daily repeatedly until disease progression or unacceptable toxicity occurs, each cycle is defined as 28 days. At least 3 subjects will receive CT053PTSA at each dose (60, 80, 100mg) for determination of Maximum Tolerated Dose(MTD) and Dose Limiting Toxicity (DLT) Dose reduction of CT053PTSA will be considered if study drug-related toxicities are observed in a subject after Cycle 1.
Expansion part:Expansion cohort might be set to further investigate the safety and efficacy of CT053PTSA at or lower MTD dose recommended by dose-escalation part.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CT053PTSA | Experimental | Drug: CT053PTSA60mg-100mg |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- study population
- Subjects must have adequate organ function and meeting all of the following laboratory review before enrollment a)blood routine examination: WBC≤2000/mm3 b)liver function: Alanine aminotransferase (ALT) and Aspartate transaminase (AST) ≤2.5×upper limit of normal(ULN); serum bilirubin ≤ 1.5 × ULN c)renal function: Serum creatinine ≤ 1.5×ULN, or the creatinine clearance (CrCl)≥ 60 mL / min calculated by the Cockcroft-Gault formula d) electrolyte: serum potassium≥3.0mmol/L; serum calcium≥2.0 mmol/L e) abnormal serum amylase without symptom≤1.5 × ULN; serum Lipase ≤1.5× ULN f) coagulation function:fibrinogen≥1.0g/L; activated partial thromboplastin time( APTT)≦ULN+10s; prothrombin time(PT)≤ULN+3s g) no obvious organ dysfunction 3)subjects must volunteer to provide evidence of effective diagnosis prior to entry or to accept bone marrow puncture or biopsy for diagnosis, and accept bone marrow puncture or biopsy after treatment to evaluate the efficacy 4) Sign the informed consent
Key exclusion criteria (21 listed)
- Subject meeting any of the following criteria will be excluded.
- treatment history
- disease history and surgery history
- i)history of immunodeficiency, other acquired or congenital immunodeficiency,history of organ transplantation; j) previous thyroid dysfunction,thyroid function can not be maintained at the normal range even have drug taken.
- k)Human immunodeficiency virus(HIV) positivity l) Hepatitis B surface antigen (HBsAg)positivity, and in the active phase(hepatitis B nucleic acid quantity≥ 1.00 × 103copies / ml); m) Hepatitis C antibody(Anti-HCV) positivity and in the active phase (hepatitis C nucleic acid quantity ≥1.00 × 102copies / ml) n) severe retinopathy or exfoliation judged by the Investigator; o) severe electrolyte imbalance judged by the investigator; p) active infectious disease judged by the investigator; q) other acute or chronic medical or psychological illnesses that are not suitable for clinical trials considered by the investigator or sponsor;
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 |
|---|---|---|
| CT053PTSAAlso: Ningetinibreceive oral CT053PTSA once daily until disease progression or unacceptable toxicity occurs, each cycle is defined as 28 days | Drug | CT053PTSA |
Primary outcome
Maximum tolerated dose (MTD)
Time frame: day 1-28
Safety and Tolerability assessed through adverse events to determine maximum tolerated dose
4 secondary outcomes
Maximum observed plasma concentration (Cmax)
Time frame: On day 1,8,15,22,29
Time of maximum observed plasma concentration (Tmax)
Time frame: On day 1,8,15,22,29
Area under the plasma concentration time curve
Time frame: On day 1,8,15,22,29
Objective response rate (ORR)
Time frame: Through study completion, an expected average of 1.5 year
Results and safety
The study was stopped early (reason given: Company strategy adjustment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Mar 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.
Jianxiang Wang, Doctor study chair
Hospital of Blood Disease, Chinese Academy of Medical Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CT053PTSA in people with Acute Myeloid Leukemia (AML), run by Sunshine Lake Pharma Co., Ltd. at 1 site in China.
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 3 inclusion and 21 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: stopped early, as of the sponsor's last update on 24 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
The study was stopped early (reason given: Company strategy adjustment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose (MTD) (time frame: day 1-28). 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 24 Mar 2021.