Actively Recruiting

Phase 1
Age: 18Years +
All Genders
ID06326411

A Phase I, Open Label Single-arm Two-part Study to Investigate Safety, Pharmacokinetics, and Preliminary Efficacy of Pan-RAF/MEK Glue NST-628 Oral Tablets in Subjects With Solid Tumors Harboring Genetic Alterations in the MAPK Pathway and Other Solid Tumors

Led by Nested Therapeutics, Inc · Updated on 2026-04-20

230

Participants Needed

23

Research Sites

52 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are evaluating the safety, pharmacokinetics, and preliminary effectiveness of NST-628, an oral drug targeting the MAPK pathway, in adults with advanced solid tumors that have specific genetic mutations and have exhausted standard treatments. This Phase 1, open-label, multi-center study includes patients with tumors dependent on the MAPK pathway, such as melanoma and glioma, aiming to find a suitable dose and observe tumor responses. The study has two parts: Part A involves dose escalation where increasing doses of NST-628 are given once daily in 28-day cycles to determine the maximum tolerated dose and the recommended dose for expansion. Part B involves dose expansion with several cohorts of patients harboring specific MAPK pathway mutations receiving the recommended dose to further assess safety and tumor response. Dose adjustments may be made based on observed effects. Participants will undergo regular assessments including safety evaluations, tumor response measurements using standardized criteria, and pharmacokinetic analyses throughout the study, which lasts about one year on average for primary outcomes and up to two years for survival monitoring. Tumor tissue samples are required, and patients will be followed until the last visit of the final participant. Safety, tumor response, progression-free survival, overall survival, and drug behavior in the body are key outcomes measured.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 years or older at the time of informed consent
  • Histologically or cytologically confirmed metastatic or locally advanced solid tumor with no effective standard therapy or not suitable for standard treatment
  • Part A: Any solid tumor with RAS/MAPK pathway genetic alteration or tumor dependence
  • Part B: Diagnosis of specific solid tumors with activating NRAS, KRAS, or select BRAF mutations, including melanoma and glioma
  • Newly obtained or archived tumor tissue available
  • Measurable disease per RECIST Version 1.1 or standard assessment for tumor type (Part B)
  • Performance status: ECOG 0 or 1 for solid tumors other than glioma; Karnofsky ≥ 70 and ECOG 0 or 1 for glioma
  • Adequate organ function
  • Ability to understand and voluntarily sign informed consent
  • Life expectancy of at least 12 weeks
Not Eligible

You will not qualify if you...

  • Conditions interfering with oral intake or intestinal absorption of NST-628
  • History or current significant retinal pathology increasing risk of retinal vein occlusion (RVO)
  • History or evidence of cardiovascular risk
  • Pneumonitis or interstitial lung disease within 6 months before starting treatment
  • Prior treatment with MEK or BRAF inhibitors (Part B)
  • Untreated or symptomatic central nervous system metastases
  • Chemotherapy, radiation, gene therapy, vaccine therapy, or anti-cancer antibodies within 28 days before treatment start
  • Targeted small molecule agents within 14 days or five half-lives before treatment start
  • Pregnant or breastfeeding females
  • Refusal to use effective contraception during and for 6 months after the trial (fertile patients)
  • Serious or unstable systemic disorders incompatible with the study

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Repeated 28-day cycles until disease progression or discontinuation

Participants receive escalating doses of NST-628 oral tablets once daily in 28-day cycles to determine the maximum tolerated dose and recommended dose for expansion. Following dose escalation, participants receive the recommended dose to evaluate safety and tumor response.

Visits occur regularly during each 28-day treatment cycle

Follow-up

Duration - Up to 2 years after treatment completion

Participants are monitored for safety, tumor response, progression-free survival, overall survival, and pharmacokinetics of NST-628 after treatment ends.

Periodic visits over the follow-up period

Trial Site Locations

Total: 23 locations

1

UCSF Helen Diller Family Comprehensive Cancer Center

San Francisco, California, United States, 94158

Actively Recruiting

2

UCLA Hematology/Oncology

Westwood, Los Angeles, California, United States, 90024

Actively Recruiting

3

Sarah Cannon Research Institute at Health ONE

Denver, Colorado, United States, 80218

Actively Recruiting

4

Yale Cancer Center

New Haven, Connecticut, United States, 06511

Completed

5

Moffitt Cancer Center

Tampa, Florida, United States, 33612

Actively Recruiting

6

Roswell Park

Buffalo, New York, United States, 14263

Actively Recruiting

7

Laura & Isaac Perlmutter Cancer Center at NYU Langone Health

New York, New York, United States, 10016

Actively Recruiting

8

Columbia University Medical Center

New York, New York, United States, 10032

Actively Recruiting

9

Memorial Slone Kettering Cancer Center

New York, New York, United States, 10065

Actively Recruiting

10

UPMC Hillman Cancer Center

Pittsburgh, Pennsylvania, United States, 15232

Actively Recruiting

11

SCRI Oncology Partners

Nashville, Tennessee, United States, 37203

Actively Recruiting

12

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, United States, 37232

Actively Recruiting

13

NEXT Oncology - Austin

Austin, Texas, United States, 78758

Active, Not Recruiting

14

NEXT Oncology - Dallas

Dallas, Texas, United States, 75039

Active, Not Recruiting

15

MD Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

16

START Moutain Region

West Valley City, Utah, United States, 84119

Completed

17

NEXT Oncology - Virginia

Fairfax, Virginia, United States, 22031

Actively Recruiting

18

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

Darlinghurst, New South Wales, Australia, 2010

Actively Recruiting

19

Scientia Clinical Research, Ltd

Randwick, New South Wales, Australia, 2031

Completed

20

Gallipoli Medical Research Centre- Greenslopes Private Hospital

Greenslopes, Queensland, Australia, 120

Actively Recruiting

21

Southern Oncology Research Unit

Adelaide, South Australia, Australia, 5042

Actively Recruiting

22

Cabrini Health Limited

Malvern, Victoria, Australia, 3144

Actively Recruiting

23

Cabrini Hospital

Malvern, Victoria, Australia, 3144

Actively Recruiting

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Research Team

C

CMO

A

Ann Marie Kennedy

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

1

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