Clinical Trials /

Study of SyB C-1101 in Patients With Myelodysplastic Syndrome

NCT03495167

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.

Related Conditions:
  • Myelodysplastic Syndromes
Recruiting Status:

Recruiting

Phase:

Phase 1

Trial Eligibility

Document

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

  • Myelodysplastic Syndrome

Interventions

DrugSynonymsArms
SyB C-1101SyB 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

NameTypeDescriptionInterventions
SyB C-1101Experimental
  • SyB C-1101

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:Recruiting
Lead Sponsor:SymBio Pharmaceuticals

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