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Myelodysplastic SyndromesPhase 2NCT02779777

Tipifarnib in Subjects With Myelodysplastic Syndromes

A Phase 2 study testing Tipifarnib in people with Myelodysplastic Syndromes, run by Kura Oncology, Inc. at 2 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This is a Phase 2 randomized, open-label, two-stage study designed to investigate the antitumor activity of tipifarnib in approximately 36 eligible subjects with MDS who have no known curative treatment. Subjects will be randomized to receive tipifarnib orally with food according to one of 2 treatment regimens. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
Tipifarnib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
2 sitesUnited States
Study dates
1 Jun 2016to 28 Aug 2018
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

  • Subject is at least 18 years of age.
  • Documented pathological evidence of MDS as defined by the World Health Organization (WHO) criteria.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
  • Subjects have no known curative treatment.
  • Subject is willing and able to comply with scheduled visits, treatment plans, laboratory tests and other procedures (including bone marrow assessments).
  • At least 1 week since the last systemic therapy regimen prior to Cycle 1 Day 1. Subjects must have recovered to NCI CTCAE v. 4.03 < Grade 2 from all acute toxicities (excluding Grade 2 toxicities that are not considered a safety risk by the Sponsor and Investigator) or toxicity must be deemed irreversible by the Investigator.
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  • Acceptable hematological function:
  • Absolute neutrophil count < 1000/mm3
  • Platelet count > 20,000/mm3
  • Acceptable liver function:
  • Total or direct bilirubin ≤ 1.5 times upper limit of normal (x ULN); does not apply to subjects with Gilbert's syndrome diagnosed as per institutional guidelines.
  • AST (SGOT) and ALT (SGPT) ≤ 2.5 x ULN.
  • Acceptable renal function with serum creatinine ≤ 1.5 x ULN or a calculated creatinine clearance ≥ 60 mL/min using the Cockcroft-Gault or Modification of Diet in Renal Disease formulas.
  • Female subjects must be:
  • Of non-child-bearing potential (surgically sterilized or at least 2 years post-menopausal); or
  • If of child-bearing potential, subject must use a highly effective method of contraception, such as combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner or sexual abstinence. Both females and male subjects with female partners of child-bearing potential must agree to use a highly effective method of contraception for 2 weeks prior to screening, during, and at least 4 weeks after last dose of trial medication. Female subjects must have a negative serum or urine pregnancy test within 72 hours prior to start of trial medication.
  • Not breast feeding at any time during the study.
  • Written and voluntary informed consent understood, signed and dated.

Who cannot join

  • Known prior progression to acute myeloid leukemia (AML), defined by at least 20% blasts in the blood or bone marrow.
  • Myelodysplastic or myeloproliferative syndrome other than MDS.
  • More than two prior systemic regimens for MDS. Prior systemic regimens are those that are considered standard of care for the treatment of MDS, have been received at standard doses for at least one full treatment cycle and exclude ESA.
  • Prior cytoreductive therapy for blast reduction.
  • Participation in any interventional study within 1 week or 5 half lives (whichever is longer) of Cycle 1 Day 1.
  • Ongoing treatment with an anticancer agent for MDS not contemplated in this protocol.
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  • Prior treatment (at least 1 full treatment cycle) with a farnesyltransferase inhibitor.
  • Clinically significant anemia due to iron, B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding. If marrow stain for iron is not available, the transferrin saturation (iron/total iron binding capacity Fe/TIBC) must be >20% or serum ferritin must be >100 ng/dL.
  • Active coronary artery disease requiring treatment, myocardial infarction within the prior year, New York Heart Association grade III or greater congestive heart failure, cerebro-vascular attack within the prior year, or current serious cardiac arrhythmia requiring medication except atrial fibrillation.
  • Major surgery, other than diagnostic surgery, within 2 weeks prior to Cycle 1 Day 1, without complete recovery.
  • Active, concurrent malignancy requiring radiation, chemotherapy, or immunotherapy (excluding non-melanoma skin cancer, adjuvant hormonal therapy for breast cancer and hormonal treatment for castration sensitive prostate cancer).
  • Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic therapy. Known infection with human immunodeficiency virus (HIV), or an active infection with hepatitis B or hepatitis C.
  • Subjects who have exhibited allergic reactions to tipifarnib, or structural compounds similar to tipifarnib or to its excipients.
  • Concomitant disease or condition that could interfere with the conduct of the study, or that would, in the opinion of the investigator, pose an unacceptable risk to the subject in this study.
  • The subject has legal incapacity or limited legal capacity.
  • Significantly altered mental status that would limit the understanding or rendering of informed consent and compliance with the requirements of this protocol. Unwillingness or inability to comply with the study protocol for any reason.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • Columbia University Medical Center

    New York, New York, 10032

Other trials in United States

About this study

Official title: An Adaptive Phase 2 Study of Tipifarnib in Subjects With Myelodysplastic Syndromes

This is a Phase 2 randomized, open-label, two-stage study designed to investigate the antitumor activity of tipifarnib in approximately 36 eligible subjects with MDS who have no known curative treatment. Subjects will be randomized to receive tipifarnib orally with food according to one of 2 treatment regimens.

Read the detailed description

This Phase 2 study will investigate the antitumor activity in terms of ORR of tipifarnib in approximately 36 eligible subjects with MDS who have no known curative treatment. Eligible subjects may have received no more than 2 prior systemic regimens. Subjects will be randomized to receive tipifarnib orally with food according to one of 2 dose regimens. In the absence of unmanageable toxicities, subjects may continue to receive tipifarnib treatment until disease progression.

Disease assessments will be performed at screening and at least once every approximately 12 weeks starting at the end of cycle 3. Determination of ORR will be assessed by the Investigator according to the MDS International Working Group (IWG) criteria (Cheson 2006). Upon disease progression, all subjects in the study will be followed approximately every 12 weeks for survival and the use of subsequent therapy until either death or 12 months after accrual of the study has been completed, whichever occurs first.

Being tested
Tipifarnib
Medical subject headings
Myelodysplastic Syndromes

What participants receive

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

  • TipifarnibDrug

    Also called Zarnesta

    Oral tablet

Study groups in NCT02779777
Study groupRoleWhat they receive
Regimen 1: TipifarnibExperimental600 mg twice daily (bid) for 7 days on Days 1-7 in 28 day cycles.
Receives: Drug: Tipifarnib
Regimen 2: TipifarnibExperimental300 mg bid for 21 days on Days 1-21 in 28 day cycles.
Receives: Drug: Tipifarnib

What is being measured

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

  1. Objective Response Rate (ORR)

    ORR was assessed by the investigator and included complete response (CR), partial response (PR), marrow complete remission (MR), and hematologic improvement (HI) according to the MDS International Working Group (IWG) criteria.

    Measured: 1 year

Secondary outcomes (7)

  1. Rate of Transfusion Independence

    Rate of transfusion independence is defined as number of participants with transfusion independence at any response assessment over 1 year.

    Measured: 1 year

  2. Duration of Transfusion Independence

    Duration of transfusion independence is defined as the period of time where participants achieved transfusion independence.

    Measured: 1 year

  3. Hematologic Improvement

    Number of subjects who experienced hematologic improvements (erythroid response, platelet response, or neutrophil response).

    Measured: 1 year

  4. Duration of Response

    Duration of response is defined as the number of days from the start date of response (whichever response is achieved first) to the first date that progressive disease is objectively documented.

    Measured: 1 year

  5. Progression-free Survival (PFS) at 1 Year

    PFS is defined as the time (in months) from enrollment to either first observation of progressive disease or occurrence of death due to any cause within 1 year (approximately 4 time intervals for disease assessments) of either first administration of tipifarnib or the last disease assessment.

    Measured: 1 year

  6. Overall Survival (OS) at 1 Year

    OS is defined as the time (in months) from enrollment to occurrence of death due to any cause within 1 year (approximately 4 time intervals for disease assessments) of either first administration of tipifarnib or the last disease assessment.

    Measured: 1 year

  7. Number of Participants Who Experienced Treatment-emergent Adverse Events (TEAEs)

    TEAEs are defined as AEs that started on or after the first dose of study drug and within approximately 30 days of the last administration of study drug. AEs were summarized by the number and percentage of subjects who experienced the event, according to system organ class and preferred term. A subject reporting multiple cases of the same AE will be counted once within each system organ class and similarly counted once within each preferred term.

    Measured: 1 year

Timeline

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

  1. First posted20 May 2016 (estimated)
  2. Study start1 Jun 2016
  3. Primary completion28 Aug 2018
  4. Study completion28 Aug 2018

Why it stopped: Lack of efficacy

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
Randomised
Intervention model
Single group
Masking
Single blind
Who is masked
Participant
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02779777
Sponsor's protocol ID
KO-TIP-003
Status last verified
Jul 2024
First submitted
10 May 2016
Last updated
31 Jul 2024
Data monitoring committee
Yes
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Tipifarnib in people with Myelodysplastic Syndromes, run by Kura Oncology, Inc. at 2 sites 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 18 inclusion and 16 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: stopped early, as of the sponsor's last update on 31 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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 31 Jul 2024). 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?

Objective Response Rate (ORR) (time frame: 1 year). 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 NCT02779777.

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 Jul 2024.

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