Study of SyB C-1101 in Patients With Myelodysplastic Syndrome

NCT03495167 | Completed Phase 1

• 1 other identifier: 2017001
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 9

Brief Summary

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.

Conditions

Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (1)

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

  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.

  Up to 2 years

Secondary Outcomes (11)

• Incidence of adverse events

  Up to 2 years

• Severity of adverse events

  Up to 2 years

• Relationship of adverse events to SyB C-1101

  Up to 2 years

• Change of laboratory test values

  Up to 2 years

• Overall hematologic response rate

  Up to 2 years

Study Arms (1)

SyB C-1101

EXPERIMENTAL

Drug: SyB C-1101

Interventions

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.

SyB C-1101

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• SymBio Pharmaceuticals: lead

Study Sites (9)

Research Site

Nagoya, Aichi-ken, Japan

Location

Research Site

Maebashi, Gunma, Japan

Location

Research Site

Sapporo, Hokkaido, Japan

Location

Research Site

Kobe, Hyōgo, Japan

Location

Research Site

Kurashiki, Okayama-ken, Japan

Location

Research Site

Shinagawa, Tokyo, Japan

Location

Research Site

Fukuoka, Japan

Location

Research Site

Kumamoto, Japan

Location

Research Site

Kyoto, Japan

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Katsuhisa Goto

  SymBio Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2017
• First Posted: April 11, 2018
• Study Start: October 6, 2017
• Primary Completion: May 28, 2019
• Study Completion: May 28, 2019
• Last Updated: November 16, 2022

Record last verified: 2022-11

Locations

JP (9)