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High Risk Myelodysplastic SyndromePhase 2NCT02131597

Guadecitabine in Treating Patients With Higher-Risk Myelodysplastic Syndromes

A Phase 2 study testing Guadecitabine in people with High Risk Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site in United States.

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 phase II trial studies how well guadecitabine works in treating patients with myelodysplastic syndromes that are at higher risk for becoming acute myeloid leukemia. Guadecitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
High Risk Myelodysplastic Syndrome
What is being tested?
Guadecitabine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
100 participants
Where
1 siteUnited States
Study dates
10 Nov 2014to 25 Jul 2024
Phase
Phase 2
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients with higher risk MDS (International Prognostic Scoring System [IPSS] int-2 or high; or >= 10% blasts as defined by World Health Organization [WHO])
  • No prior intensive chemotherapy or high-dose cytarabine (>= 1 g/m^2)
  • Prior biologic therapies (=< 1 cycle of prior decitabine or azacitidine), targeted therapies, or single agent chemotherapy is allowed
  • Off chemotherapy for 2 weeks prior to entering this study with no toxic effects of that therapy, unless there is evidence of rapidly progressive disease
  • Hydroxyurea is permitted for control of counts prior to treatment
  • Hematopoietic growth factors are allowed
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  • Eastern Cooperative Oncology Group (ECOG) performance status =< 2
  • Serum creatinine =< 1.5 mg/dL
  • Serum bilirubin =< 1.5 x upper limit of normal (ULN)
  • Aspartate transaminase (AST) or alanine transaminase (ALT) =< 2.5 x ULN
  • Alkaline phosphatase =< 2.5 x ULN
  • Provide signed written informed consent
  • Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent
  • Female patients of childbearing potential must have a negative pregnancy test within 2 weeks prior to entering this study
  • Women who are able to become pregnant and men who can father a child must use birth control while on study and for at least 8 weeks after your last dose of study drug(s); acceptable birth control includes a condom or a diaphragm with spermicidal jelly; and birth control methods that are taken by mouth, injected, or implanted; if you are already using birth control, you must check with the study staff to make sure that it is considered one of the acceptable forms to use in this study

Who cannot join

  • Current concomitant chemotherapy, radiation therapy, or immunotherapy other than as specified in the protocol
  • Use of investigational agents within 30 days or any anticancer therapy within 2 weeks prior to entering this study with the exception of hydroxyurea; the patient must have recovered from all acute toxicities from any previous therapy
  • Have any other severe concurrent disease, or have a history of serious organ dysfunction or disease involving the heart, kidney, liver, or other organ system that may place the patient at undue risk to undergo treatment
  • Patients with a systemic fungal, bacterial, viral, or other infection not controlled (defined as exhibiting ongoing signs/symptoms related to the infection and without improvement, despite appropriate antibiotics or other treatment)
  • Pregnant or lactating patients
  • Any significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results
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  • Any concurrent malignancy
  • Exceptions
  • Patients with treated non-melanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia, regardless of the disease-free duration, are eligible for this study if definitive treatment for the condition has been completed
  • Patients with organ-confined prostate cancer with no evidence of recurrent or progressive disease based on prostate-specific antigen (PSA) values are also eligible for this study if hormonal therapy has been initiated or a radical prostatectomy has been performed

Trial locations and contacts

1 site in 1 country.

United States1 site

  • M D Anderson Cancer Center

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: Phase 2 Study of SGI-110 in Patients With Higher Risk MDS

This phase II trial studies how well guadecitabine works in treating patients with myelodysplastic syndromes that are at higher risk for becoming acute myeloid leukemia. Guadecitabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To evaluate the complete response (CR) rate with SGI-110 (guadecitabine) in patients with higher risk myelodysplastic syndrome (MDS).

SECONDARY OBJECTIVES:

  1. Overall response rate, survival, transformation to acute myeloid leukemia (AML), transfusion independence.

II. Safety and toxicity.

OUTLINE:

Patients receive guadecitabine subcutaneously (SC) on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.

After completion of study treatment, patients are followed up at 30 days, and then every 2 months.

Being tested
Guadecitabine

What participants receive

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

  • GuadecitabineDrug

    Also called DNMT inhibitor SGI-110, S110, SGI-110

    Given SC

Study groups in NCT02131597
Study groupRoleWhat they receive
Treatment (guadecitabine)ExperimentalPatients receive guadecitabine SC on days 1-5. Treatment repeats every 4-8 weeks for up to 24 courses in the absence of disease progression or unacceptable toxicity. Patients with stable disease after 3 courses are taken off therapy after 6 courses. Patients may continue to receive treatment after 24 courses if the investigator determines it is in the patient's best interest.
Receives: Drug: Guadecitabine

What is being measured

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

  1. Number of Participants With a Complete Response (CR)

    Complete Response is Normalization of the peripheral blood and bone marrow with </= 5% bone marrow blasts, a peripheral blood granulocyte count >/= 1.0 x 10^9/L, and a platelet count >/= 100 x 10^9/L. Hemoglobin >/= 11 g/dL at any point while on treatment after the first cycle of therapy.

    Measured: At cycle 3 and cycle 6, cycles are up every 4 to 8 weeks. Up to 9 years, 8 months and 15 days.

Secondary outcomes (2)

  1. Overall Survival

    Time from date of treatment start until date of death due to any cause or last Follow-up.

    Measured: Up to 9 years, 8 months and 15 days

  2. Time to Acute Myeloid Leukemia (AML) Transformation

    Measured: Up to 9 years, 8 months and 15 days

Timeline

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

  1. First posted6 May 2014 (estimated)
  2. Study start10 Nov 2014
  3. Primary completion25 Jul 2024
  4. Study completion25 Jul 2024

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
100 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02131597
Sponsor's protocol ID
2013-0901
Other IDs
NCI-2014-02377, 2013-0901
Status last verified
Jun 2025
First submitted
2 May 2014
Last updated
17 Jul 2025
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Urrutia S, Bose P, Alvarado Y, Borthakur G, Ravandi F, Daver N, Pemmaraju N, Jabbour E, Takahashi K, Kadia T, DiNardo C, Kornblau S, Kanagal-Shamanna R, Huang X, Bodden K, Kantarjian H, Garcia-Manero G. Prospective performance of the IWG-2023 criteria and IPSS-M in a phase 2 trial of guadecitabine for higher-risk MDS or CMML. Blood Neoplasia. 2024 Mar 29;1(2):100008. doi: 10.1016/j.bneo.2024.100008. eCollection 2024 Jun.

    PubMed 40454402

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Guadecitabine in people with High Risk Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site in United States.

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 15 inclusion and 10 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 17 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 17 Jul 2025). 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?

Number of Participants With a Complete Response (CR) (time frame: At cycle 3 and cycle 6, cycles are up every 4 to 8 weeks. Up to 9 years, 8 months and 15 days.). 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 NCT02131597, which also lists 1 linked publication.

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 17 Jul 2025.

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