NCT05012397

Brief Summary

Phase 2, multicenter, single-arm, open-label basket study designed to evaluate the safety and efficacy of milademetan in patients with advanced or metastatic solid tumors refractory or intolerant to standard-of-care therapy that exhibit wild-type (WT) TP53 and MDM2 copy number (CN) ≥ 8 using prespecified biomarker criteria.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Nov 2021

Geographic Reach
1 country

14 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 13, 2021

Completed
6 days until next milestone

First Posted

Study publicly available on registry

August 19, 2021

Completed
2 months until next milestone

Study Start

First participant enrolled

November 1, 2021

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 15, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 15, 2023

Completed
1 year until next milestone

Results Posted

Study results publicly available

October 17, 2024

Completed
Last Updated

October 17, 2024

Status Verified

October 1, 2024

Enrollment Period

2 years

First QC Date

August 13, 2021

Results QC Date

August 16, 2024

Last Update Submit

October 14, 2024

Conditions

Keywords

MDM2MilademetanBasket Study

Outcome Measures

Primary Outcomes (1)

  • To Determine the ORR of Treatment With Milademetan in Patients With Advanced/Metastatic Solid Tumors With MDM2 Gene Amplification.

    Overall Response Rate (ORR) of treatment with milademetan, as defined as the percentage of patients who have achieved confirmed complete response (CR) or Partial Response (PR) according to RECIST v1.1

    From first dose date to first confirmed complete or partial response or study completion date; up to 23.5 months.

Secondary Outcomes (4)

  • Duration of Response (DOR)

    From start date of response to first PD or study completion date; up to 23.5 months

  • Progression-free Survival (PFS)

    From the first dose date to the earliest date of recurrence, progression, death, or study completion; up to 23.5 months

  • Growth Modulation Index (GMI)

    From the start date of the most recent prior line of therapy to the PD date on the study; up to 23.5 months

  • Disease Control Rate (DCR)

    From first dose date to first CR, PR, or stable disease (SD) >= 16 weeks, or study completion date; up to 23.5 months

Study Arms (1)

Milademetan (RAIN-32)

EXPERIMENTAL

260 mg once dailly orally on Days 1 to 3 and Days 15 to 17 of each 28-day cycle

Drug: RAIN-32

Interventions

260 mg once daily orally on Days 1 to 3 and Days 15 to 17 of each 28-day cycle

Also known as: Milademetan
Milademetan (RAIN-32)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Histologically and/or cytologically confirmed diagnosis of a cancer that is a locally advanced or metastatic solid tumor
  • Measurable tumor lesion(s) in accordance with RECIST v1.1
  • Received all standard therapy appropriate for their tumor type and stage of disease or, in the opinion of the Investigator, would be unlikely to tolerate or derive clinically meaningful benefit from appropriate standard-of-care therapy
  • Resolution of any clinically relevant toxic effects of prior chemotherapy, surgery, radiotherapy, or hormonal therapy
  • Presence of WT TP53 and MDM2 gene amplification by tumor tissue/blood testing, defined as ≥ 8 copies in tumor tissue by central laboratory or ≥ 8 copies or 4-fold increase in tumor tissue or blood by local testing
  • Prescreening for TP53 and MDM2 at a Central Laboratory:
  • MDM2 amplification: CN unknown and where CN cannot be derived for documentation by interpretation of reported results
  • MDM2 amplification: CN 6 to 7.9
  • MDM2 amplification: 3-3.9-fold increase
  • MDM2 amplification with CN ≥ 8 and with equivocal TP53 mutation upon discussion with Sponsor's Medical Monitor
  • ECOG performance status of 0 or 1
  • Adequate bone marrow function:
  • Platelet count ≥ 100 × 10\^9/L
  • Hemoglobin ≥ 9.0 g/dL
  • Absolute neutrophil count ≥ 1.5 × 10\^9/L
  • +5 more criteria

You may not qualify if:

  • Prior treatment with a murine double minute 2 (MDM2) inhibitor
  • Well-differentiated/dedifferentiated liposarcoma or intimal sarcoma/cardiac sarcoma
  • Primary malignancies that required systemic antineoplastic treatment within the previous 2 years, except for localized cancers that have apparently been cured
  • Has a primary malignant brain tumor of any grade or histology
  • Untreated brain metastases
  • Gastrointestinal conditions that could affect the absorption of milademetan, in the opinion of the Investigator
  • Known HIV infection or active hepatitis B or C infection
  • Major surgery ≤ 3 weeks of the first dose of milademetan
  • Curative-intent radiation therapy ≤ 4 weeks or palliative radiation therapy
  • Uncontrolled or significant cardiovascular disease
  • QTcF at rest, where the mean QTcF interval is \> 480 milliseconds
  • Myocardial infarction within 6 months
  • Uncontrolled angina pectoris within 6 months
  • New York Heart Association Class 3 or 4 congestive heart failure
  • Uncontrolled hypertension

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

Stanford University Medical Center

Palo Alto, California, 94305, United States

Location

Florida Cancer Specialists

Fort Myers, Florida, 33901, United States

Location

Florida Cancer Specialists

St. Petersburg, Florida, 33705, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02214, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

Hematology Oncology Associates of Central NY

Syracuse, New York, 13057, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

University of Cincinnati Medical Center

Cincinnati, Ohio, 45267, United States

Location

Sanford Health

Sioux Falls, South Dakota, 57104, United States

Location

Tennessee Oncology, PLLC

Nashville, Tennessee, 37203, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Northwest Medical Specialities

Tacoma, Washington, 77030, United States

Location

Related Publications (1)

  • Gounder MM, Bauer TM, Schwartz GK, Weise AM, LoRusso P, Kumar P, Tao B, Hong Y, Patel P, Lu Y, Lesegretain A, Tirunagaru VG, Xu F, Doebele RC, Hong DS. A First-in-Human Phase I Study of Milademetan, an MDM2 Inhibitor, in Patients With Advanced Liposarcoma, Solid Tumors, or Lymphomas. J Clin Oncol. 2023 Mar 20;41(9):1714-1724. doi: 10.1200/JCO.22.01285. Epub 2023 Jan 20.

    PMID: 36669146BACKGROUND

MeSH Terms

Conditions

CholangiocarcinomaSarcomaAdenocarcinoma of LungOvarian NeoplasmsUterine Cervical NeoplasmsCarcinoma, Non-Small-Cell LungStomach NeoplasmsBiliary Tract NeoplasmsMelanomaPancreatic NeoplasmsTesticular Germ Cell TumorAdrenocortical Carcinoma

Interventions

milademetan

Condition Hierarchy (Ancestors)

AdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplasmsNeoplasms, Connective and Soft TissueLung NeoplasmsRespiratory Tract NeoplasmsThoracic NeoplasmsNeoplasms by SiteEndocrine Gland NeoplasmsOvarian DiseasesAdnexal DiseasesGenital Diseases, FemaleFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesGenital Neoplasms, FemaleUrogenital NeoplasmsGenital DiseasesEndocrine System DiseasesGonadal DisordersUterine NeoplasmsUterine Cervical DiseasesUterine DiseasesCarcinoma, BronchogenicBronchial NeoplasmsLung DiseasesRespiratory Tract DiseasesGastrointestinal NeoplasmsDigestive System NeoplasmsDigestive System DiseasesGastrointestinal DiseasesStomach DiseasesBiliary Tract DiseasesNeuroendocrine TumorsNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms, Nerve TissueNevi and MelanomasSkin NeoplasmsSkin DiseasesSkin and Connective Tissue DiseasesPancreatic DiseasesAdrenal Cortex NeoplasmsAdrenal Gland NeoplasmsAdrenal Cortex DiseasesAdrenal Gland Diseases

Results Point of Contact

Title
Clinical Research
Organization
Rain Oncology

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 13, 2021

First Posted

August 19, 2021

Study Start

November 1, 2021

Primary Completion

October 15, 2023

Study Completion

October 15, 2023

Last Updated

October 17, 2024

Results First Posted

October 17, 2024

Record last verified: 2024-10

Data Sharing

IPD Sharing
Will not share

Locations