Clinical trial NCT02000154: Long Term Safety Study of SyB L-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) - Extension Study
Official title: Phase I Clinical Trial of SyB L-1101 in Patients With Myelodysplastic Syndrome - Extension Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Myelodysplastic Syndrome
Lead sponsor: SymBio Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2013-12-03
Enrollment: 1
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: This is an extension study study to investigate long term safety of SyB L-1101 when administered intravenously every 4 weeks to the patients who have completed 8 cycles in the study 2011005 whose purpose is to investigate tolerability of SyB L-1101 when administered intravenously in patients with recurrent/relapsed or refractory myelodysplastic syndrome. Antitumor effects will also be investigated in this study.


Eligibility criteria: Inclusion Criteria:

Patients must satisfy the following conditions listed below.

1. Patients enrolled in the study 2011005 of SyB L-1101 in Patients With Myelodysplastic Syndrome.
2. Patients who was not judged as disease progression\* nor progressive disease/relapsed\*\* at the end of the cycle 8 in the study 2011005. \* hematologic remission according to IWG 2006 criteria \*\* hematologic improvement according to IWG 2006 criteria
3. Patients who met the continuation criteria\*\*\* after Cycle 8 week 2 (Day 15±3) in the study 2011005.\*\*\*defined in the study 2011005 protocol
4. Patients who can be expected to survive at least three months or longer.
5. Patients who have score of 0 to 2 in Eastern Cooperative Oncology Grou (ECOG) Performance Status (P.S.).
6. Patients with adequate function in major organs (heart, lungs, liver, kidneys, etc.).

   * Aspartate aminotransferase (AST): no more than 3.0 times the upper boundary of the reference range at each institution
   * Alanine aminotransferase (ALT): no more than 3.0 times the upper boundary of the reference range at each institution
   * Total bilirubin: no more than 1.5 times the upper boundary of the reference range at each institution
   * Serum creatinine: no more than 1.5 times the upper boundary of the reference range at each institution
   * ECG: no abnormal findings requiring treatment
   * Echocardiography: no abnormal findings requiring treatment
7. Patients who personally signed an informed consent document for participation in this study.

Exclusion Criteria:

Patients who satisfy any of the following conditions will not be enrolled in the study.

1. Patients with anemia (haemolytic anaemia, gastrointestinal haemorrhage, etc.) caused by factors other than MDS.
2. Patients with obvious infectious diseases (including viral infections).
3. Patients with serious complications (liver failure, renal failure, etc.).
4. Patients with a complication of serious heart disease (myocardial infarction, ischemic heart disease, etc.)
5. Patients with a serious gastrointestinal condition (severe or significant nausea/vomiting, diarrhea, etc.)
6. Patients with serious bleeding tendencies (disseminated intravascular coagulation (DIC), internal hemorrhage, etc.).
7. Ascites requiring active medical management including paracentesis, or hyponatremia (defined as serum sodium value of \<130 milliequivalent/L).
8. Patients with an addiction to a legal or illegal drug, or with alcohol dependency.
9. Patients who are nursing, pregnant or may become pregnant.
10. Patients who have not consented to the following contraceptive measures. Patients will avoid sexual intercourse with sexual partners or should use the following contraceptive methods in these time periods: for male patients during the administration period of the trial and for six months after the end of administration; female patients during the administration period of the trial, and until a second menstrual period is confirmed after the end of administration (or in the case of female patients with no menstrual period, for two months after the end of administration). (1) Male patients:The patient will always use a condom. For effective contraception, it is recommended that the female partner also use the contraceptive methods for female patients. (2) Female patients: Female patients who may become pregnant should use one or more types of the following contraceptive methods. In addition, the male partner will always use a condom.

    * Oral contraceptive (birth control pills)
    * Intrauterine device (IUD)
    * Tubal ligation
11. Other patients judged to be unsuitable by an investigator or sub-investigator.

Primary outcomes:
measure: Adverse Events
timeFrame: Up to 20 weeks
Secondary outcomes:
measure: Disease Response Assessment
timeFrame: Up to 20 weeks
measure: Serious Adverse Events
timeFrame: Up to 20 weeks
measure: Hematologic Improvement
timeFrame: Up to 20 weeks
measure: Cytogenetic Response
timeFrame: Up to 20 weeks
Other outcomes:
Locations:
city: Nagoya
country: Japan
facility: Research Site
state: Aichi
city: Isesaki
country: Japan
facility: Research site
state: Kanagawa
city: Sendai
country: Japan
facility: Research Site
state: Miyagi
city: Kurashiki
country: Japan
facility: Research Site
state: Okayama
city: Kawagoe
country: Japan
facility: Research Site
state: Saitama
city: Fukuoka
country: Japan
facility: Research Site
city: Kagoshima
country: Japan
facility: Research Site
city: Kumamoto
country: Japan
facility: Research Site
city: Tokyo
country: Japan
facility: Research Site
Study officials:
affiliation: SymBio Pharmaceuticals
name: Katsuhisa Goto
role: STUDY_DIRECTOR
References: