Myelodysplastic SyndromePhase 1/2NCT02737462
Phase 1/2 Study of CG200745 PPA for Myelodysplastic Syndrome
A Phase 1/2 study testing CG200745 PPA in people with Myelodysplastic Syndrome, run by CrystalGenomics, Inc. at 1 site in South Korea.
Trial summary
<Part I - Phase I trial> The phase I clinical trial is to identify the MTD (Maximum Tolerated Dose) and DLT (Dose Limiting Toxicity) of CG200745 PPA. Initial dose of CG200745 PPA is 150 mg/m^2, and it will be extended to 225 mg/m^2, 300 mg/m^2 or it will be reduced to 75 mg/m^2 based on the results of the cohort of 3… Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- CG200745 PPA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteSouth Korea
- Study dates
- Jun 2016to Dec 2023
- Phase
- Phase 1/2
- 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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Ages: 20 years and above
- Patient with MDS according to French-American-British (FAB) classification
- Patients who failed to respond to prior hypomethylating agents (5-azacytidine, decitabine)
- Eastern Cooperative Oncology Group (ECOG) performance status: 0-2
- Adequate renal and hepatic function
- Total serum bilirubin ≤ 3 x Upper Limit Normal (ULN) (except for the case of increased unconjugated bilirubin)
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- Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT), and Alkaline phosphatase (ALP) < 3 x ULN
- Calculated Glomerular Filtration Rate (GFR) ≥ 50
- Fertile patients, except post-menopausal patients (no menstruation for at least 2 years) or proof of surgical sterility, must use effective contraception up to 3 months after the completion or withdrawal of the study.
- Negative pregnancy test
- Patients who understand the overall procedures and requirements of the study
Who cannot join
- Peripheral or bone marrow blasts: > 30%
- Less than 4 weeks since major surgery or radiotherapy
- Patient with clinically meaningful and relevant, active Central Nerve System (CNS) disorder
- Patient with active liver disease
- Patient with HIV positive
- Hyper-sensitivity to study drug or similar substances of the drugs
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- Prior Histone Deacetylase (HDAC) inhibitor therapy
- Less than 4 weeks since hypomethylating agent or cytotoxic drug therapy
- Less than 4 weeks since immunosuppressive drug therapy
- Patient who participated in another clinical trial within past 4 weeks
- Patient who have severe diseases:
- Severe cardiovascular diseases (severe or unstable angina, congestive heart failure, myocardial infarction within past 1 year, uncontrolled hypertension and uncontrolled arrhythmia)
- Neurological or psychiatric disorder
- Active uncontrolled infection
- Any other diseases that may interfere with the interpretation of study result (according to the judgment of investigator)
- Pregnancy or lactating
- Patient who is not considered to be appropriate for the study according to the judgment of investigator
Trial locations and contacts
1 site in 1 country.
South Korea1 site
Asan Medical Center, Samsung Medical Center, Seoul National University Hospital
Seoul
About this study
Official title: A Phase I/II Study of CG200745 PPA to Determine the Maximum Tolerated Dose and Evaluate the Safety and Efficacy in Patients With Myelodysplastic Syndrome (MDS) Who Failed to Respond to Prior Hypomethylating Therapy
<Part I - Phase I trial> The phase I clinical trial is to identify the MTD (Maximum Tolerated Dose) and DLT (Dose Limiting Toxicity) of CG200745 PPA. Initial dose of CG200745 PPA is 150 mg/m^2, and it will be extended to 225 mg/m^2, 300 mg/m^2 or it will be reduced to 75 mg/m^2 based on the results of the cohort of 3 to 6 subjects per dose level.
Based on the 3+3 dose escalation study design, CG200745 PPA is to be administered according to the dose level. Each cohort consists of 3 or 6 subjects.
<Part II - Phase II trial> In the phase II clinical trial, the subjects will be administered with the dose which is to be identified as a recommended dose based on the results of Phase I study. Each cycle consisted of 28 days, same as the phase I. The entire treatment period is 6 cycles and tumor assessment is to be evaluated at the end of every 2 cycles.
Read the detailed description
<Part I - Phase I trial> The phase I clinical trial is to identify the MTD and DLT of CG200745 PPA. Initial dose of CG200745 PPA is 150 mg/m^2, and it will be extended to 225 mg/m^2, 300 mg/m^2 or it will be reduced to 75 mg/m^2 based on the results of the cohort of 3 - 6 subjects per dose level.
Based on the 3+3 dose escalation study design, CG200745 PPA is to be administered as in four different cohorts according to the dose level. Each cohort consists of 3 or 6 subjects.
- Dose Level -1: CG200745 PPA 75 mg/m^2 x 5 (375 mg/m^2/cycle) / -50%
- Dose Level 1: CG200745 PPA 150 mg/m^2 x 5 (750 mg/m^2/cycle) / initial base dose
- Dose Level 2: CG200745 PPA 225 mg/m^2 x 5 (1,125 mg/m^2/cycle) / 50%
- Dose Level 3: CG200745 PPA 300 mg/m^2 x 5 (1,500 mg/m^2/cycle) / 33%
<Part II - Phase II trial> In the phase II clinical trial, the subjects will be administered with the dose which is to be identified as a recommended dose based on the results of Phase I study. Each cycle consisted of 28 days, same as the phase I. The entire treatment period is 6 cycles and tumor assessment is to be evaluated at the end of every 2 cycles.
- Conditions
- Myelodysplastic Syndrome
- Being tested
- CG200745 PPA
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CG200745 PPADrug
Also called CG200745 PPA (phosphoric acid)
CG200745 PPA intravenously daily for first 5 consecutive days per cycle (4 weeks)
| Study group | Role | What they receive |
|---|---|---|
| CG200745 PPA | Experimental | CG200745 PPA intravenously daily for first 5 consecutive days per cycle (4 weeks) Receives: Drug: CG200745 PPA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
ORR is the proportion of the subjects with Complete Response (CR), Partial Response (PR), marrow CR (mCR), and hematological improvement (HI) in comparison to the total subjects
Measured: up to 6 cycles (each cycle is 28 days)
Secondary outcomes (4)
Area Under the Curve [AUC]
Pharmacokinetics (PK) parameter
Measured: Part I, Cycle 1, Day 1, up to 6 days
Maximum Plasma Concentration [Cmax]
Pharmacokinetics (PK) parameter
Measured: Part I, Cycle 1, Day 1, up to 6 days
Adverse Event
Safety parameter
Measured: up to 6 cycles
Clinical laboratory tests
Safety parameter
Measured: up to 6 cycles
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2016 (estimated)
- Study startJun 2016
- Primary completionDec 2022
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CrystalGenomics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Je-Hwan Lee, M.D., PhD., Principal investigator, Asan Medical CenterJun Ho Jang, M,D., Ph.D., Principal investigator, Samsung Medical CenterSung-soo Yoon, M,D., Ph.D., Principal investigator, Seoul National University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02737462
- Sponsor's protocol ID
- CG200745-2-02
- Status last verified
- Sep 2023
- First submitted
- 22 Mar 2016
- Last updated
- 13 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of CG200745 PPA to Determine the Maximum Tolerated Dose and Evaluate the Safety and Efficacy in Patients With Myelodysplastic Syndrome (MDS) Who Failed to Respond to Prior Hypomethylating Therapy
- ClinicalTrials.gov ID
- NCT02737462
- Acronym / sponsor ID
- CG200745-2-02
- Conditions
- Myelodysplastic Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- CrystalGenomics, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2016 (actual)
- Primary completion
- Dec 2022 (actual)
- Study completion
- Dec 2023 (estimated)
- First posted
- 14 Apr 2016 (estimated)
- 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.
<Part I - Phase I trial> The phase I clinical trial is to identify the MTD (Maximum Tolerated Dose) and DLT (Dose Limiting Toxicity) of CG200745 PPA. Initial dose of CG200745 PPA is 150 mg/m^2, and it will be extended to 225 mg/m^2, 300 mg/m^2 or it will be reduced to 75 mg/m^2 based on the results of the cohort of 3 to 6 subjects per dose level.
Based on the 3+3 dose escalation study design, CG200745 PPA is to be administered according to the dose level. Each cohort consists of 3 or 6 subjects.
<Part II - Phase II trial> In the phase II clinical trial, the subjects will be administered with the dose which is to be identified as a recommended dose based on the results of Phase I study. Each cycle consisted of 28 days, same as the phase I. The entire treatment period is 6 cycles and tumor assessment is to be evaluated at the end of every 2 cycles.
Detailed description
<Part I - Phase I trial> The phase I clinical trial is to identify the MTD and DLT of CG200745 PPA. Initial dose of CG200745 PPA is 150 mg/m^2, and it will be extended to 225 mg/m^2, 300 mg/m^2 or it will be reduced to 75 mg/m^2 based on the results of the cohort of 3 - 6 subjects per dose level.
Based on the 3+3 dose escalation study design, CG200745 PPA is to be administered as in four different cohorts according to the dose level. Each cohort consists of 3 or 6 subjects.
- Dose Level -1: CG200745 PPA 75 mg/m^2 x 5 (375 mg/m^2/cycle) / -50%
- Dose Level 1: CG200745 PPA 150 mg/m^2 x 5 (750 mg/m^2/cycle) / initial base dose
- Dose Level 2: CG200745 PPA 225 mg/m^2 x 5 (1,125 mg/m^2/cycle) / 50%
- Dose Level 3: CG200745 PPA 300 mg/m^2 x 5 (1,500 mg/m^2/cycle) / 33%
<Part II - Phase II trial> In the phase II clinical trial, the subjects will be administered with the dose which is to be identified as a recommended dose based on the results of Phase I study. Each cycle consisted of 28 days, same as the phase I. The entire treatment period is 6 cycles and tumor assessment is to be evaluated at the end of every 2 cycles.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CG200745 PPA | Experimental | Drug: CG200745 PPACG200745 PPA intravenously daily for first 5 consecutive days per cycle (4 weeks) |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Ages: 20 years and above
- Patient with MDS according to French-American-British (FAB) classification
- Patients who failed to respond to prior hypomethylating agents (5-azacytidine, decitabine)
- Eastern Cooperative Oncology Group (ECOG) performance status: 0-2
- Adequate renal and hepatic function
Key exclusion criteria (17 listed)
- Peripheral or bone marrow blasts: > 30%
- Less than 4 weeks since major surgery or radiotherapy
- Patient with clinically meaningful and relevant, active Central Nerve System (CNS) disorder
- Patient with active liver disease
- Patient with HIV positive
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 |
|---|---|---|
| CG200745 PPAAlso: CG200745 PPA (phosphoric acid)CG200745 PPA intravenously daily for first 5 consecutive days per cycle (4 weeks) | Drug | CG200745 PPA |
Primary outcome
Overall Response Rate (ORR)
Time frame: up to 6 cycles (each cycle is 28 days)
ORR is the proportion of the subjects with Complete Response (CR), Partial Response (PR), marrow CR (mCR), and hematological improvement (HI) in comparison to the total subjects
4 secondary outcomes
Area Under the Curve [AUC]
Time frame: Part I, Cycle 1, Day 1, up to 6 days
Maximum Plasma Concentration [Cmax]
Time frame: Part I, Cycle 1, Day 1, up to 6 days
Adverse Event
Time frame: up to 6 cycles
Clinical laboratory tests
Time frame: up to 6 cycles
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2023.
- Individual participant dataSharing planned: Undecided
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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.
Je-Hwan Lee, M.D., PhD. principal investigator
Asan Medical Center
Jun Ho Jang, M,D., Ph.D. principal investigator
Samsung Medical Center
Sung-soo Yoon, M,D., Ph.D. principal investigator
Seoul National University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CG200745 PPA in people with Myelodysplastic Syndrome, run by CrystalGenomics, Inc. at 1 site in South Korea.
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 17 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 13 Sep 2023. 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 South Korea. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
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?
Overall Response Rate (ORR) (time frame: up to 6 cycles (each cycle is 28 days)). 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 13 Sep 2023.