Clinical trial NCT03495167: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Myelodysplastic Syndrome
Lead sponsor: SymBio Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2018-04-11
Enrollment: 10
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Identification of Dose-Limiting Toxicity (DLT) and Number of Patients with DLT in Each Cohort
timeFrame: Up to 2 years
Secondary outcomes:
measure: Incidence of adverse events
timeFrame: Up to 2 years
measure: Severity of adverse events
timeFrame: Up to 2 years
measure: Relationship of adverse events to SyB C-1101
timeFrame: Up to 2 years
measure: Change of laboratory test values
timeFrame: Up to 2 years
measure: Overall hematologic response rate
timeFrame: Up to 2 years
measure: Overall hematologic improvement rate
timeFrame: Up to 2 years
measure: Overall cytogenetic response rate
timeFrame: Up to 2 years
measure: Cmax
timeFrame: Up to 2 years
measure: tmax
timeFrame: Up to 2 years
measure: AUC
timeFrame: Up to 2 years
measure: t 1/2
timeFrame: Up to 2 years
Other outcomes:
Locations:
city: Nagoya
country: Japan
facility: Research Site
state: Aichi
city: Maebashi
country: Japan
facility: Research Site
state: Gunma
city: Sapporo
country: Japan
facility: Research Site
state: Hokkaido
city: Kobe
country: Japan
facility: Research Site
state: Hyogo
city: Kurashiki
country: Japan
facility: Research Site
state: Okayama
city: Shinagawa
country: Japan
facility: Research Site
state: Tokyo
city: Fukuoka
country: Japan
facility: Research Site
city: Kumamoto
country: Japan
facility: Research Site
city: Kyoto
country: Japan
facility: Research Site
Study officials:
affiliation: SymBio Pharmaceuticals
name: Katsuhisa Goto
role: STUDY_DIRECTOR
References: