A Study to Investigate the Safety and Efficacy of NST-628 Oral Tablets in Subjects With Solid Tumors

NCT ID: NCT06326411

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

230 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-09

Study Completion Date

2029-11-30

Brief Summary

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This is a two-part Phase 1, open label, multi-center, single arm, non-randomized, multiple dose, safety, pharmacokinetic (PK) and preliminary efficacy study of single agent NST-628 in adult patients with MAPK pathway mutated/dependent advanced solid tumors who have exhausted standard treatment options.

Detailed Description

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The study includes two parts, a dose escalation part (Part A) followed by a dose expansion part (Part B). Part A will estimate the maximum tolerated dose (MTD) in dose escalation cohorts in patients with advanced solid tumors for whom no standard therapy is available in order to establish the recommended dose for expansion (RDE). Successive cohorts of subjects will receive escalating doses of NST-628 orally once daily in 28-day cycles.

Bayesian Optimal Interval (BOIN) method will be used for dose escalation.

Once MTD is reached or dose escalation is stopped prior to reaching MTD and provisional RDE selected, the provisional RDE level will be expanded. If warranted by dose/toxicity/anti-tumor activity observations, additional, lower dose level(s) may also be expanded.

Part B of the study will include up to 6 cohorts of approximately up to 30 subjects each with select MAPK pathway mutant solid tumors enrolled at the RDE in order to explore benefit from treatment as suggested by preclinical findings and will better define the safety profile of NST-628 at the RDE. Additional safety information gathered in Part B may be used to modify the dose recommended for future studies.

The end of the study is the last visit of the last subject.

Conditions

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Oncology MEK Mutation RAF Gene Mutation Ras (KRAS or NRAS) Gene Mutation Melanoma NSCLC Glioma Solid Tumor, Adult MAPK Pathway Gene Mutation

Keywords

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RAS MEK RAF solid tumor

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part A Dose Escalation and Part B Dose Expansion

Part A will evaluate the safety of NST-628 with advanced solid tumors and determine the recommended dose for expansion of NST-628.

Part B will evaluate the objective tumor response rate in subjects with advanced solid tumors harboring specified genetic alterations receiving NST-628 and evaluate the safety of NST-628 in subjects with advanced solid tumors in cohorts defined below:

i. Melanoma Cohorts

1. Activating NRAS mutations
2. Select BRAF alterations

ii. Non-Melanoma Cohorts:

1. Solid tumors with NRAS activating mutations
2. Solid tumors with KRAS activating mutations
3. Solid tumors with select BRAF alterations
4. Glioma with BRAF alterations

Group Type EXPERIMENTAL

NST-628

Intervention Type DRUG

NST-628 is a small molecule non-covalent pan-RAF/MEK dual molecular glue targeting RAF and MEK nodes of MAPK pathway.

Interventions

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NST-628

NST-628 is a small molecule non-covalent pan-RAF/MEK dual molecular glue targeting RAF and MEK nodes of MAPK pathway.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Subjects are eligible to be included in the study only if all of the following criteria apply:

1. Subjects must be ≥18 years old (or of legal age of consent in the country in which the study is taking place) at the time of signing the informed consent.
2. Subjects who have a histologically or cytologically documented metastatic or locally advanced solid tumor, for which standard of care (SoC) therapy does not exist, no longer provides benefit, or is not tolerated by the subject, or the subject has been assessed by the Investigator as not being suitable for SoC therapy.

1. Part A: Subjects with any solid tumor with genetic alteration of or evidence of tumor dependence upon the RAS/MAPK pathway (subject to additional restrictions specified in the study protocol)
2. Part B: Subjects must be diagnosed with one of the following solid tumors harboring specified genetic alterations based on a validated local test:

i. Melanoma Cohorts:
1. Activating NRAS mutations
2. Select BRAF alterations

ii. Non-Melanoma Cohorts:
1. Solid tumors with NRAS activating mutations
2. Solid tumors with KRAS activating mutations
3. Solid tumors with select BRAF alterations
4. Glioma with BRAF alterations
3. Newly obtained or archived tumor tissue is required
4. Part B: measurable disease as defined by RECIST Version 1.1 or by other disease assessment tool standard for a given tumor type (if RECIST v. 1.1 is not standard)
5. Performance status

1. Solid tumors other than glioma: ECOG 0 or 1
2. Glioma: Karnofsky ≥ 70 and ECOG 0 or 1
6. Have adequate organ function
7. Understand and voluntarily sign an Institutional Review Board/Independent Ethics Committee-approved informed consent form prior to any study-specific evaluation.
8. Life expectancy ≥ 12 weeks

Exclusion Criteria

Subjects are excluded from the study if any of the following criteria apply:

1. Conditions interfering with oral intake of NST-628
2. Conditions interfering with intestinal absorption of an orally administered drug
3. A history or current evidence of significant retinal pathology leading to increased risk of RVO
4. A history or evidence of cardiovascular risk
5. Current or history within 6 months of planned Cycle 1 Day 1 of pneumonitis or interstitial lung disease (ILD)
6. Part B: prior treatment with any MEK or BRAF inhibitor
7. Untreated or symptomatic central nervous system (CNS) metastases
8. Chemotherapy, radiation, gene therapy, vaccine therapy, or anti-cancer antibodies / ADCs within 28 days of Cycle 1 Day 1
9. Targeted small molecule agents within 14 days or 5 half-lives of Cycle 1 Day 1
10. Females who are pregnant or breastfeeding.
11. For fertile patients (female able to become pregnant or male able to father a child), refusal to use effective contraception during the period of the trial and for 6 months after the last dose of NST-628
12. Presence of any serious or unstable concomitant systemic disorder incompatible with the clinical study
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Nested Therapeutics, Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCSF Helen Diller Family Comprehensive Cancer Center

San Francisco, California, United States

Site Status RECRUITING

UCLA Hematology/Oncology

Westwood, Los Angeles, California, United States

Site Status RECRUITING

Sarah Cannon Research Institute at Health ONE

Denver, Colorado, United States

Site Status RECRUITING

Yale Cancer Center

New Haven, Connecticut, United States

Site Status RECRUITING

Moffitt Cancer Center

Tampa, Florida, United States

Site Status RECRUITING

Roswell Park

Buffalo, New York, United States

Site Status RECRUITING

Laura & Isaac Perlmutter Cancer Center at NYU Langone Health

New York, New York, United States

Site Status RECRUITING

Columbia University Medical Center

New York, New York, United States

Site Status RECRUITING

Memorial Slone Kettering Cancer Center

New York, New York, United States

Site Status RECRUITING

UPMC Hillman Cancer Center

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

SCRI Oncology Partners

Nashville, Tennessee, United States

Site Status RECRUITING

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, United States

Site Status RECRUITING

NEXT Oncology - Austin

Austin, Texas, United States

Site Status RECRUITING

NEXT Oncology - Dallas

Dallas, Texas, United States

Site Status RECRUITING

MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

START Moutain Region

West Valley City, Utah, United States

Site Status RECRUITING

NEXT Oncology - Virginia

Fairfax, Virginia, United States

Site Status RECRUITING

The Kinghorn Cancer Center, St. Vincent's Health Network

Darlinghurst, New South Wales, Australia

Site Status RECRUITING

Scientia Clinical Research, Ltd

Randwick, New South Wales, Australia

Site Status RECRUITING

Gallipoli Medical Research Centre- Greenslopes Private Hospital

Greenslopes, Queensland, Australia

Site Status RECRUITING

Southern Oncology Research Unit

Adelaide, South Australia, Australia

Site Status RECRUITING

Cabrini Health Limited

Malvern, Victoria, Australia

Site Status RECRUITING

Cabrini Hospital

Malvern, Victoria, Australia

Site Status RECRUITING

Countries

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United States Australia

Central Contacts

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CMO

Role: CONTACT

Phone: 617-468-4292

Email: [email protected]

Ann Marie Kennedy

Role: CONTACT

Phone: 919-427-4225

Email: [email protected]

Facility Contacts

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Phu Lam Clinical Research Manager

Role: primary

Bartosz Chmielowski, MD

Role: primary

Role: primary

Ingrid Palma

Role: primary

Justin Martin

Role: primary

Role: backup

Study Coordinator

Role: primary

Nurse Navigation Team

Role: primary

Role: primary

Role: primary

Angie Boeing

Role: primary

Brianna Flores

Role: primary

Erica Torres

Role: primary

Rabia Khan

Role: primary

Marie Asay

Role: primary

Blake Patterson

Role: primary

Jia (Jenny) Liu, MD

Role: primary

Shiena Lemin

Role: primary

Sarah McLennan

Role: primary

Meggan O'Riley

Role: primary

Study Coordinator

Role: primary

Study Coordinator

Role: primary

Other Identifiers

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NST-628-001

Identifier Type: -

Identifier Source: org_study_id