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Advanced Malignant Solid TumorsPhase 1NCT02045095

A Dose Escalation Study of MLN7243 (TAK-243) in Adult Participants With Advanced Solid Tumors

A Phase 1 study testing MLN7243 in people with Advanced Malignant Solid Tumors, run by Millennium Pharmaceuticals, Inc. at 7 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

The purpose of this study is to evaluate safety and tolerability (establish maximum tolerated dose [MTD], inform the recommended phase 2 dose [RP2D], and identify the dose-limiting toxicities [DLTs]) of MLN7243. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Advanced Malignant Solid Tumors
What is being tested?
MLN7243
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
29 participants
Where
7 sitesUnited States
Study dates
31 Jan 2014to 9 Nov 2016
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Each participant must meet all of the following inclusion criteria to be enrolled in the study:
  • Male or female participants 18 years or older.
  • Participants must have a histologically confirmed diagnosis of an advanced, metastatic malignant solid tumor and must have failed or exhausted standard therapies or for which no standard therapy is available.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
  • Participants with adequate hematologic and organ function.
  • All participants must have radiographically detectable tumors with measurable disease as defined by RECIST (version 1.1).
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  • Participants undergoing a biopsy procedure must have accessible lesions which are safe to biopsy.
  • Recovered (that is, less than or equal to (<=) Grade 1 toxicity) from the reversible effects of prior antineoplastic therapy, except alopecia.
  • Female participants who are postmenopausal for at least 1 year before the screening visit, surgically sterile, or agree to practice 2 effective methods of contraception, at the same time, from the time of signing the informed consent form through 4 months after the last dose of study drug, or agree to practice true abstinence.
  • Male participants who agree to practice effective barrier contraception during the entire study treatment period through 4 months after the last dose of study drug or agree to practice true abstinence.
  • Suitable venous access for the study-required blood sampling including PK sampling.

Who cannot join

  • Participants meeting any of the following exclusion criteria are not to be enrolled in the study:
  • Participants with clinically significant pre-existing cardiac impairment.
  • Participants homozygous for the ABCG2 (BCRP) c.421C greater than (>) 1 A polymorphism will be excluded from the study until the safety of a minimum of the first 3 dose levels has been characterized and the sponsor confirms that such participants can be enrolled at doses that are at least 3-fold lower than the most recently determined safe and tolerable dose among at least 3 dose limiting toxicities (DLT)-evaluable non-homozygous participants.
  • Participants with known active central nervous system (CNS) lesions are excluded. Systemic antineoplastic therapy or investigational agents within 21 days before the first dose of study drug.
  • Radiotherapy within 14 days before the first dose of study drug is not allowed except for limited field radiotherapy for palliative bone pain.
  • For participants where tumor biopsies are required or requested:
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  • Any known coagulation abnormalities that would contraindicate the tumor biopsy procedure.
  • Ongoing therapy with any anticoagulant or antiplatelet agents (example, aspirin, clopidogrel [Plavix®], heparin, or warfarin).
  • Major surgery within 28 days before the first dose of MLN7243.
  • Life-threatening illness unrelated to cancer.
  • Any evidence of active infection or antibiotic therapy within 14 days before the first dose of MLN7243.
  • Known human immunodeficiency virus (HIV) positivity or AIDS-related illness, hepatitis B virus, and hepatitis C virus.
  • Participants whose weight is less than (<) 40 kilogram (kg).
  • History of uncontrolled sleep apnea syndrome and other conditions that could result in excessive daytime sleepiness, such as severe chronic obstructive pulmonary disease.
  • Female participants who are lactating and breastfeeding or have a positive serum pregnancy test during the Screening period or a positive urine pregnancy test on Day 1 before first dose of study drug.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United States7 sites
  • Study site

    Boston, Massachusetts

  • Study site

    Charleston, South Carolina

  • Study site

    Cleveland, Ohio

  • Study site

    Nashville, Tennessee

  • Study site

    Philadelphia, Pennsylvania

  • Study site

    San Antonio, Texas

  • Study site

    St Louis, Missouri

Other trials in United States

About this study

Official title: A Phase 1, Open-Label, Multicenter, Dose Escalation Study to Assess the Safety and Tolerability of MLN7243, an Inhibitor of Ubiquitin-Activating Enzyme (UAE), in Adult Patients With Advanced Solid Tumors

The purpose of this study is to evaluate safety and tolerability (establish maximum tolerated dose [MTD], inform the recommended phase 2 dose [RP2D], and identify the dose-limiting toxicities [DLTs]) of MLN7243.

Read the detailed description

This is a single arm Phase I study with multiple dosing cohorts as noted below:

  • Schedule A: MLN7243 1 mg
  • Schedule A: MLN7243 2 mg
  • Schedule A: MLN7243 4 mg
  • Schedule A: MLN7243 8 mg
  • Schedule A: MLN7243 12 mg
  • Schedule A: MLN7243 18 mg
  • Schedule A: MLN7243 Homozygous Mutant 4 mg
Being tested
MLN7243
Keywords
Drug therapy

What participants receive

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

  • MLN7243Drug

    Also called TAK-243

    Dose escalation stage Schedule A: Intravenous infusion on Days 1, 4, 8, 11 for a 21-day treatment cycle.

    Schedule B: Intravenous infusion on Days 1, 8, 15 for a 28-day treatment cycle.

    Dose expansion stage: MLN7243 will be administered following schedule A (twice-weekly, 21-day dosing) and/or B (once-weekly, 28-day dosing).

Study groups in NCT02045095
Study groupRoleWhat they receive
Schedule A: MLN7243 1 mgExperimentalMLN7243 1 milligram (mg), infusion, intravenously over 10-minutes, on Days 1, 4, 8, and 11 followed by 10 days of rest in a 21-day treatment cycle for a maximum of 12 months, or until symptomatic deterioration or disease progression (PD) or discontinuation of study for another reason, or until study is stopped.
Receives: Drug: MLN7243
Schedule A: MLN7243 2 mgExperimentalMLN7243 2 mg, infusion, intravenously over 10-minutes, on Days 1, 4, 8, and 11 followed by 10 days of rest in a 21-day treatment cycle for a maximum of 12 months, or until symptomatic deterioration or PD, or discontinuation of study for another reason, or until study is stopped.
Receives: Drug: MLN7243
Schedule A: MLN7243 4 mgExperimentalMLN7243 4 mg, infusion, intravenously over 10-minutes, on Days 1, 4, 8, and 11 followed by 10 days of rest in a 21-day treatment cycle for a maximum of 12 months, or until symptomatic deterioration or PD or discontinuation of study for another reason, or until study is stopped.
Receives: Drug: MLN7243
Schedule A: MLN7243 8 mgExperimentalMLN7243 8 mg, infusion, intravenously over 10-minutes, on Days 1, 4, 8, and 11 followed by 10 days of rest in a 21-day treatment cycle for a maximum of 12 months, or until symptomatic deterioration or PD or discontinuation of study for another reason, or until study is stopped.
Receives: Drug: MLN7243
Schedule A: MLN7243 12 mgExperimentalMLN7243 12 mg, infusion, intravenously over 10-minutes, on Days 1, 4, 8, and 11 followed by 10 days of rest in a 21-day treatment cycle for a maximum of 12 months, or until symptomatic deterioration or PD or discontinuation of study for another reason, or until study is stopped.
Receives: Drug: MLN7243
Schedule A: MLN7243 18 mgExperimentalMLN7243 18 mg, infusion, intravenously over 10-minutes, on Days 1, 4, 8, and 11 followed by 10 days of rest in a 21-day treatment cycle for a maximum of 12 months, or until symptomatic deterioration or PD or discontinuation of study for another reason, or until study is stopped.
Receives: Drug: MLN7243
Schedule A: MLN7243 Homozygous Mutant 4 mgExperimentalMLN7243 homozygous mutant 4 mg, infusion, intravenously over 10-minutes, on Days 1, 4, 8, and 11 followed by 10 days of rest in a 21-day treatment cycle for a maximum of 12 months, or until symptomatic deterioration or PD or discontinuation of study for another reason, or until study is stopped.
Receives: Drug: MLN7243

What is being measured

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

  1. Number of Participants Reporting One or More Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Measured: Baseline up to 30 days after last dose of study drug (Cycle 10 Day 41)

  2. Number of Participants With Laboratory Related TEAEs by System Organ Class (SOC)

    Measured: Baseline up to 30 days after last dose of study drug (Cycle 10 Day 41)

  3. Number of Participants With Vital Sign Related TEAEs by Preferred Term (PT)

    Measured: Baseline up to 30 days after last dose of study drug (Cycle 10 Day 41)

  4. Number of Participants With Clinically Significant Electrocardiogram (ECG) Abnormalities

    Measured: Cycle 1 Day 1 up to Cycle 1 Day 11

  5. Number of Participants With Clinically Significant Echocardiogram Abnormalities

    Measured: Cycle 1 Day 2 up to 30 days after last dose of study drug (Cycle 10 Day 41)

  6. Number of Participants With TEAEs Related to Tropinin I and T

    Measured: Baseline up to 30 days after last dose of study drug (Cycle 10 Day 41)

Secondary outcomes (13)
  1. Ceoi: Plasma Concentration at the End of Infusion for TAK-243

    Measured: Cycle 1 Day 1 and 11: pre-infusion to end of infusion (up to 10 minutes)

  2. AUClast: Area Under the Plasma Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration for TAK-243

    Measured: Cycle 1 Day 1 pre-dose and at multiple time points (up to 48 hours) post-dose; Cycle 1 Day 11 pre-dose and at multiple time points (up to 72 hours) post-dose

  3. AUCτ: Area Under the Plasma Concentration-time Curve Over the Dosing Interval for TAK-243

    Measured: Cycle 1 Day 1 pre-dose and at multiple time points (up to 48 hours) post-dose; Cycle 1 Day 11 pre-dose and at multiple time points (up to 72 hours) post-dose

  4. AUC∞: Area Under the Plasma Concentration-time Curve From Time 0 to Infinity for TAK-243

    Measured: Cycle 1 Day 1 pre-dose and at multiple time points (up to 48 hours) post-dose; Cycle 1 Day 11 pre-dose and at multiple time points (up to 72 hours) post-dose

  5. CL: Total Clearance After Intravenous Administration for TAK-243

    Measured: Cycle 1 Day 1 pre-dose and at multiple time points (up to 48 hours) post-dose; Cycle 1 Day 11 pre-dose and at multiple time points (up to 72 hours) post-dose

  6. Vss: Volume of Distribution at Steady State for TAK-243

    Measured: Cycle 1 Day 1 pre-dose and at multiple time points (up to 48 hours) post-dose; Cycle 1 Day 11 pre-dose and at multiple time points (up to 72 hours) post-dose

  7. Aet: Amount of TAK-243 Excreted Unchanged in Urine

    Measured: Cycle 1 Day 1; Cycle 1 Day 11

  8. Fet: Percentage of TAK-243 Excreted Unchanged in Urine

    Measured: Cycle 1 Day 1; Cycle 1 Day 11

  9. Terminal Phase Elimination Half-life (T1/2) for TAK-243

    Measured: Cycle 1 Day 1 pre-dose and at multiple time points (up to 48 hours) post-dose; Cycle 1 Day 11 pre-dose and at multiple time points (up to 72 hours) post-dose

  10. Change From Baseline in Immunohistochemistry (IHC) Biomarkers in Tumor Biopsies at Cycle 1 Day 12 (C1D12) as Assessed by Histological Score (H-score)

    The pharmacodynamics IHC biomarkers included polyubiquitin marker and ubquityl (Ub)-histone H2B marker. H-score was a composite score that comprised of intensity and percentage of staining and was used for assessing the amount of protein or phospho-protein present in a biopsy sample. The composite score obtained by H-score is derived by summing the percentages of cell staining at each intensity multiplied by the weighted intensity of staining (0, 1+, 2+, 3+; where 3+ indicates the strongest staining, 2+ indicates medium staining, 1+ indicates weak staining, and 0 indicates no staining). The composite H-score ranges from 0 to 300, with a score of 0 representing the absence of any of the target protein and an H-score of 300 representing maximum staining and intensity of the target protein.

    Measured: Baseline and Cycle 1 Day 12

  11. Change From Baseline in IHC Biomarkers in Tumor Biopsies at C1D12 as Assessed by Positive Index

    The pharmacodynamics IHC biomarkers included polyubiquitin marker and Ub-histone H2B marker. Positive index was calculated by taking the number of positive cells over the total number of cells.

    Measured: Baseline and Cycle 1 Day 12

  12. Percentage of Participants With Best Overall Response

    Best overall response for participant is best observed post-baseline disease response as per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria. Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes (whether target and non target) must have reduction in short axis to less than (<) 10 millimeter (mm). Partial Response (PR): at least 30 percent (%) decrease in sum of diameter of target lesions, taking as reference baseline sum of diameter. Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference smallest sum of diameter; PD: at least 20% increase in sum of diameter of target lesions, taking as reference, smallest sum on study (this includes baseline sum if that is smallest on study). In addition to relative increase of 20%, sum must also demonstrate an absolute increase of at least mm. The appearance of 1 or more new lesions is also considered progression.

    Measured: Baseline up to end of study (approximately 7 months)

  13. Duration of Response

    Duration of any response (CR or PR) was defined as the time (in both days and months) from the date of first documented response per the investigator response assessment to the date of first progressive disease after the first documented response or, if the participant discontinues treatment, the date of last disease assessment as per RECIST version 1.1. CR: Disappearance of all target lesions. Any pathological lymph nodes (whether target and non target) must have reduction in short axis to <10 mm. PR: at least 30% decrease in sum of diameter of target lesions, taking as reference baseline sum of diameter.

    Measured: Baseline up to end of study (approximately 7 months)

Timeline

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

  1. First posted24 Jan 2014 (estimated)
  2. Study start31 Jan 2014
  3. Primary completion9 Nov 2016
  4. Study completion9 Nov 2016

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 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
29 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02045095
Sponsor's protocol ID
C33001
Other IDs
U1111-1203-6359
Status last verified
Nov 2018
First submitted
18 Dec 2013
Last updated
14 Mar 2019
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing MLN7243 in people with Advanced Malignant Solid Tumors, run by Millennium Pharmaceuticals, Inc. at 7 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 11 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: stopped early, as of the sponsor's last update on 14 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 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 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 14 Mar 2019). 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 Reporting One or More Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: Baseline up to 30 days after last dose of study drug (Cycle 10 Day 41)). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
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 14 Mar 2019.

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