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Completed

NCT Number: NCT03495167

Study of SyB C-1101 in Patients With Myelodysplastic Syndrome

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.

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Key information

Conditions

Myelodysplastic Syndrome Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myelodysplastic Syndromes

Age range

20 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Research Site, Nagoya, Aichi-ken, Japan

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Patients who meet all of the following criteria are eligible for enrollment in the study:

  • 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.
  • Classified as Intermediate-1, Intermediate-2 or High-risk, according to IPSS classification.
  • 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)
  • 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.
  • Patients with expected survival of ≥3 months.
  • Patients aged 20 years or older (at the time of informed consent).
  • ECOG Performance Status (PS) of 0, 1 or 2
  • 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
  • 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.

Treatment and study plan

SyB C-1101

Drug

SyB C-1101 (rigosertib sodium) will be administered to two cohorts of patients; each receives either twice daily (560 mg before breakfast and 560 mg before dinner) or twice daily (840 mg before breakfast and 280 mg before dinner. SyB C-1101 will be administered orally twice daily for 21 consecutive days, followed by a 7-day observation period. The treatment period of 28 days (21 days of administration + 7 days of observation) constitutes 1 cycle.

Primary outcomes

  1. Identification of Dose-Limiting Toxicity (DLT) and Number of Patients with DLT in Each Cohort

    Time frame: Up to 2 years

    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 outcomes

  1. Incidence of adverse events

    Time frame: Up to 2 years

    Calculate from the rate between number of patients occurred AE and number of patients received SyB C-1101.

  2. Severity of adverse events

    Time frame: Up to 2 years

    Score as grade 1 to 5 according to criteria by CTCAE v4.0-JCOG.

  3. Relationship of adverse events to SyB C-1101

    Time frame: Up to 2 years

    Score as "related " or "not related".

  4. Change of laboratory test values

    Time frame: Up to 2 years

    Number of patients with changes in laboratory values OR list each lab value separately (e.g.Hgb, Fe, Hct, etc.)

  5. Overall hematologic response rate

    Time frame: Up to 2 years

    Calculate from the rate of patients scored as CR, PR or marrow CR according to IWG 2006 criteria.

  6. Overall hematologic improvement rate

    Time frame: Up to 2 years

    Calculate from the rate of patients with hematologic improvement according to IWG 2006 criteria.

  7. Overall cytogenetic response rate

    Time frame: Up to 2 years

    Calculate from the rate of patients scored as complete cytogeneic response or partial cytogenetic response according to IWG 2006 criteria.

  8. Cmax

    Time frame: Up to 2 years

    Maximum plasma concentration

  9. tmax

    Time frame: Up to 2 years

    Time to maximum plasma concentration

  10. AUC

    Time frame: Up to 2 years

    Area under the plasma concentration curve

  11. t 1/2

    Time frame: Up to 2 years

    Half-life time

Sponsors and collaborators

Lead sponsor

SymBio Pharmaceuticals

Industry

Registry information

Official study title

Multi-center, Open-label, Phase I Study of SyB C-1101 in Patients With Myelodysplastic Syndrome

Important dates

Study start
2017
Primary completion
2019
Study completion
2019
First posted
Apr 11, 2018
Registry last updated
Nov 16, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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