Myelodysplastic SyndromePhase 1NCT02000154

Long Term Safety Study of SyB L-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS) - Extension Study

A Phase 1 study testing SyB L-1101 in people with Myelodysplastic Syndrome, run by SymBio Pharmaceuticals at 9 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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… Read more about this study

Who is it for?: Ages 20 years and older
Conditions the study is for: Myelodysplastic Syndrome
What is being tested?: SyB L-1101
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 1 participants
Where: 9 sitesJapan
Study dates: Dec 2014to May 2015
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 20 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients must satisfy the following conditions listed below.
Patients enrolled in the study 2011005 of SyB L-1101 in Patients With Myelodysplastic Syndrome.
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
Patients who met the continuation criteria* after Cycle 8 week 2 (Day 15±3) in the study 2011005.*defined in the study 2011005 protocol
Patients who can be expected to survive at least three months or longer.
Patients who have score of 0 to 2 in Eastern Cooperative Oncology Grou (ECOG) Performance Status (P.S.).

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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
Patients who personally signed an informed consent document for participation in this study.

Who cannot join

Patients who satisfy any of the following conditions will not be enrolled in the study.
Patients with anemia (haemolytic anaemia, gastrointestinal haemorrhage, etc.) caused by factors other than MDS.
Patients with obvious infectious diseases (including viral infections).
Patients with serious complications (liver failure, renal failure, etc.).
Patients with a complication of serious heart disease (myocardial infarction, ischemic heart disease, etc.)
Patients with a serious gastrointestinal condition (severe or significant nausea/vomiting, diarrhea, etc.)

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Patients with serious bleeding tendencies (disseminated intravascular coagulation (DIC), internal hemorrhage, etc.).
Ascites requiring active medical management including paracentesis, or hyponatremia (defined as serum sodium value of <130 milliequivalent/L).
Patients with an addiction to a legal or illegal drug, or with alcohol dependency.
Patients who are nursing, pregnant or may become pregnant.
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
Other patients judged to be unsuitable by an investigator or sub-investigator.

Trial locations and contacts

9 sites in 1 country.

City, postcode or hospital

9 sites in 1 country

Japan9 sites

Research Site
Fukuoka, Fukuoka
Research site
Isesaki, Kanagawa
Research Site
Kagoshima, Kagoshima-ken
Research Site
Kawagoe, Saitama
Research Site
Kumamoto, Kumamoto
Research Site
Kurashiki, Okayama-ken
Research Site
Nagoya, Aichi-ken
Research Site
Sendai, Miyagi
Research Site
Tokyo, Tokyo

Other trials in Japan

About this study

Official title: Phase I Clinical Trial of SyB L-1101 in Patients With Myelodysplastic Syndrome - Extension Study

Conditions: Myelodysplastic Syndrome
Being tested: SyB L-1101
Medical subject headings: Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

SyB L-1101Drug
SyB L-1101 (rigosertib sodium) will be administered intravenously 72 continuous hours (3 days), followed by 25-day observation period. The treatment period of 28 days (3 days of administration + 25 days of observation) constitutes 1 cycle. The dose at cycle 8 in the study 2011005 will be the dose (if needed, the dose can be reduced) at the first cycle in this study (cycle 9). From cycle 10 on, the dose of SyB L-1101 will be reduced, delayed, or discontinued according to adverse events and results of observation at the previous cycle.

Study groups in NCT02000154
Study group	Role	What they receive
SyB L-1101	Experimental	Not described Receives: Drug: SyB L-1101

What is being measured

The primary outcome is the main question the study is designed to answer.

Adverse Events
Total number affected by any adverse events (details are presented in adverse event section)
Measured: Up to 20 weeks

Secondary outcomes (4)

Disease Response Assessment
Disease progression
According to the International Working Group 2006 response criteria for Myelodysplastic Syndrome, "disease progression" is defined as no evidence of complete remission (CR), partial remission, marrow CR, stable disease, or failure, and as meeting one of the following conditions.
1. when pretreatment percentage of bone marrow blasts < 5%: ≥ 50% increase to > 5%.
2. when pretreatment percentage of bone marrow blasts 5 to 10%: ≥ 50% increase to > 10%.
3. when pretreatment percentage of bone marrow blasts 10 to 20%: ≥ 50% increase to > 20%.
4. when pretreatment percentage of bone marrow blasts 20 to 30%: ≥ 50% increase to > 30%.
5. other: at least one of the following: decrease to ≤ 50% of neutrophil or platelet count at maximum response, ≥ 2 g/dL decrease in Hgb or transfusion dependence (in the absence of other factors, such as infection, gastrointestinal bleeding, or hemolysis).
Measured: Up to 20 weeks
Serious Adverse Events
Total number affected any serious adverse events
Measured: Up to 20 weeks
Hematologic Improvement
NCA (not considered assessable)
no evidence of hematologic improvement -erythroid, -platelet, -neutrophil, progressive disease, or relapse, defined in the International Working Group 2006 response criteria for myelodysplastic syndrome.
Measured: Up to 20 weeks
Cytogenetic Response
NCA (not considered assessable)
no evidence of cytogenetic response, defined in the International Working Group 2006 response criteria for myelodysplastic syndrome.
Measured: Up to 20 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2014May 2015

First posted3 Dec 2013 (estimated)
Study startDec 2014
Primary completionMay 2015
Study completionMay 2015

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 1 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02000154
Sponsor's protocol ID: 2012001
Status last verified: Feb 2017
First submitted: 26 Nov 2013
Last updated: 31 Mar 2017
Results posted: Yes, on ClinicalTrials.gov

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome

All Myelodysplastic Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 31 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Phase I Clinical Trial of SyB L-1101 in Patients With Myelodysplastic Syndrome - Extension Study
ClinicalTrials.gov ID: NCT02000154
Acronym / sponsor ID: 2012001
Conditions: Myelodysplastic Syndrome
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 1 participants (actual)
Lead sponsor: SymBio PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Dec 2014
Primary completion: May 2015 (actual)
Study completion: May 2015 (actual)
First posted: 3 Dec 2013 (estimated)
Results: Posted 31 Mar 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
SyB L-1101	Experimental	Drug: SyB L-1101

Who may qualify

Ages: 20 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (14 listed)

Patients must satisfy the following conditions listed below.
Patients enrolled in the study 2011005 of SyB L-1101 in Patients With Myelodysplastic Syndrome.
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
Patients who met the continuation criteria* after Cycle 8 week 2 (Day 15±3) in the study 2011005.*defined in the study 2011005 protocol
Patients who can be expected to survive at least three months or longer.

Key exclusion criteria (15 listed)

Patients who satisfy any of the following conditions will not be enrolled in the study.
Patients with anemia (haemolytic anaemia, gastrointestinal haemorrhage, etc.) caused by factors other than MDS.
Patients with obvious infectious diseases (including viral infections).
Patients with serious complications (liver failure, renal failure, etc.).
Patients with a complication of serious heart disease (myocardial infarction, ischemic heart disease, etc.)

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
SyB L-1101SyB L-1101 (rigosertib sodium) will be administered intravenously 72 continuous hours (3 days), followed by 25-day observation period. The treatment period of 28 days (3 days of administration + 25 days of observation) constitutes 1 cycle. The dose at cycle 8 in the study 2011005 will be the dose (if needed, the dose can be reduced) at the first cycle in this study (cycle 9). From cycle 10 on, the dose of SyB L-1101 will be reduced, delayed, or discontinued according to adverse events and results of observation at the previous cycle.	Drug	SyB L-1101

Primary outcome

Adverse Events
Time frame: Up to 20 weeks
Total number affected by any adverse events (details are presented in adverse event section)

4 secondary outcomes

Disease Response Assessment
Time frame: Up to 20 weeks
Serious Adverse Events
Time frame: Up to 20 weeks
Hematologic Improvement
Time frame: Up to 20 weeks
Cytogenetic Response
Time frame: Up to 20 weeks

Results and safety

Results posted to the registry on 31 Mar 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
SyB L-1101	1	1

Primary outcome: Adverse Events

Time frame: Up to 20 weeks. Number. Unit: participants

Measure	SyB L-1101 n = 1
Value	1

4 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
SyB L-1101	0 of 1 (0%)	1 of 1 (100%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Myelodysplastic Syndrome

RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Katsuhisa Goto study director

SymBio Pharmaceuticals

9 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing SyB L-1101 in people with Myelodysplastic Syndrome, run by SymBio Pharmaceuticals at 9 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 31 Mar 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Events (time frame: Up to 20 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02000154.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Mar 2017.