Description:
To assess tolerability of SyB C-1101 when administered orally BID for 21 days followed by a
7-day observation period in patients with recurrent/relapsed or refractory myelodysplastic
syndrome in order to determine a recommended dose (RD). To assess safety, efficacy and
pharmacokinetics.
Title
- Brief Title: Study of SyB C-1101 in Patients With Myelodysplastic Syndrome
- Official Title: Multi-center, Open-label, Phase I Study of SyB C-1101 in Patients With Myelodysplastic Syndrome
Clinical Trial IDs
- ORG STUDY ID:
2017001
- NCT ID:
NCT03495167
Conditions
Interventions
Drug | Synonyms | Arms |
---|
SyB C-1101 | | SyB C-1101 |
Purpose
To assess tolerability of SyB C-1101 when administered orally BID for 21 days followed by a
7-day observation period in patients with recurrent/relapsed or refractory myelodysplastic
syndrome in order to determine a recommended dose (RD). To assess safety, efficacy and
pharmacokinetics.
Trial Arms
Name | Type | Description | Interventions |
---|
SyB C-1101 | Experimental | | |
Eligibility Criteria
Patients who meet all of the following criteria are eligible for enrollment in the study:
1. Histologically or cytologically diagnosed as myelodysplastic syndrome (MDS) according
to WHO criteria or FAB classification. For patients with RAEB in transformation
(RAEB-t), peripheral WBC is ≦25,000 /mm3 and the disease is stable for at least 4
weeks.
2. Classified as Intermediate-1, Intermediate-2 or High-risk, according to IPSS
classification.
3. Patients with a history of previous treatment of the target disease (e.g.,
immunosuppressive therapy, protein anabolic steroids, and chemotherapy including
azacitidine and lenalidomide) and meet one of the followings:
- Patients who failed to achieve complete remission, partial remission, or
hematologic improvement*
- Patients experienced with recurrence/relapse after achieving complete remission,
partial remission, or hematologic improvement*
- Patients who were intolerable to the previous therapy *: The most recent
assessment of the therapeutic effect based on "Clinical application and proposal
for modification of the International Working Group (IWG) response criteria in
myelodysplasia" (IWG2006 criteria)
4. Off all other treatment (including erythropoiesis stimulating agents) for MDS, for at
least 4 weeks prior to enrollment and no carry-over (of antitumor effect) from
previous treatment is expected as judged by Investigator. Transfusion is allowed, as
clinically indicated.
5. Patients with expected survival of ≥3 months.
6. Patients aged 20 years or older (at the time of informed consent).
7. ECOG Performance Status (PS) of 0, 1 or 2
8. Patients with adequate major organ functions (including the heart, lungs, liver, and
kidneys).
- AST (GOT): ≤2.5 -fold the upper limit of normal range at each institution
- ALT (GPT) : ≤2.5 -fold the upper limit of normal range at each institution
- Total bilirubin: <2.0 mg/dL (except patients with Gilbert's disease or hemolysis)
- Serum creatinine: <2.0 mg/dL
- ECG: Absence of abnormal findings that require treatment
- Echocardiography: Absence of abnormal findings that require treatment
9. The patient must sign an informed consent form indicating that s/he understands the
purpose of and procedure required for the study and is willing to participate in the
study.
Maximum Eligible Age: | N/A |
Minimum Eligible Age: | 20 Years |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | Identification of Dose-Limiting Toxicity (DLT) and Number of Patients with DLT in Each Cohort |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Based on the number of patients with DLT and administration dose in each cohort, recommended dosage will be defined for the following clinical phase.
A DLT is defined as an adverse event that occurred during the Cycle 1, for which a causality with the investigational products (IP) cannot be ruled out and meets the following criteria.
Criteria: ≥ Grade3 non-hematological toxicity (except pyrexia). However nausea, vomiting, diarrhea, stomatitis and esophagitis/dysphagia are excluded (≥ Grade 3 nausea, vomiting, and diarrhea persist for ≥ 48 hours and uncontrolled by antiemetic or antidiarrheal agents, and ≥ Grade 3 stomatitis and esophagitis/dysphagia lasting for ≥ 4 days are regarded as DLTs). ≥ Grade 2 pyrexia uncontrolled by antipyretic agents. However, in case pyrexia of ˃ 39°C occurred within 24 hours after administration of SyB C-1101 and its cause is unclear, it is deemed that the causality to the IP cannot be ruled out. |
Secondary Outcome Measures
Measure: | Incidence of adverse events |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Calculate from the rate between number of patients occurred AE and number of patients received SyB C-1101. |
Measure: | Severity of adverse events |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Score as grade 1 to 5 according to criteria by CTCAE v4.0-JCOG. |
Measure: | Relationship of adverse events to SyB C-1101 |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Score as "related " or "not related". |
Measure: | Change of laboratory test values |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Number of patients with changes in laboratory values OR list each lab value separately (e.g.Hgb, Fe, Hct, etc.) |
Measure: | Overall hematologic response rate |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Calculate from the rate of patients scored as CR, PR or marrow CR according to IWG 2006 criteria. |
Measure: | Overall hematologic improvement rate |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Calculate from the rate of patients with hematologic improvement according to IWG 2006 criteria. |
Measure: | Overall cytogenetic response rate |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Calculate from the rate of patients scored as complete cytogeneic response or partial cytogenetic response according to IWG 2006 criteria. |
Measure: | Cmax |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Maximum plasma concentration |
Measure: | tmax |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Time to maximum plasma concentration |
Measure: | AUC |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Area under the plasma concentration curve |
Measure: | t 1/2 |
Time Frame: | Up to 2 years |
Safety Issue: | |
Description: | Half-life time |
Details
Phase: | Phase 1 |
Primary Purpose: | Interventional |
Overall Status: | Unknown status |
Lead Sponsor: | SymBio Pharmaceuticals |
Last Updated
April 11, 2018