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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05186753
Registration number
NCT05186753
Ethics application status
Date submitted
19/11/2021
Date registered
11/01/2022
Date last updated
8/04/2024
Titles & IDs
Public title
(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
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Scientific title
A Multi-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of The Safety and Efficacy of CGT9486 in Subjects With Nonadvanced Systemic Mastocytosis
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Secondary ID [1]
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CGT9486-21-202
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
SSM
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Mastocytosis, Indolent
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Mastocytosis, Systemic
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Mastocytosis
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Condition category
Condition code
Skin
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Other skin conditions
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Blood
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Other blood disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Bezuclastinib Tablets (Formulation A)
Treatment: Drugs - Bezuclastinib Tablets (Formulation B)
Treatment: Drugs - Placebo Tablets
Experimental: (Part 1a) Bezuclastinib Dose 1 + BSC -
Experimental: (Part 1a) Bezuclastinib Dose 2 + BSC -
Placebo Comparator: (Part 1a) Placebo + BSC -
Experimental: (Part 1b) Bezuclastinib Dose 1 + BSC -
Experimental: (Part 1b) Bezuclastinib Dose 2 + BSC -
Placebo Comparator: (Part 1b) Placebo + BSC -
Experimental: (Part 2) Bezuclastinib Selected Dose + BSC -
Placebo Comparator: (Part 2) Placebo + BSC -
Experimental: (Part 3) Bezuclastinib + BSC -
Treatment: Drugs: Bezuclastinib Tablets (Formulation A)
Bezuclastinib will be administered orally, once daily continuously for 28-day cycles
Treatment: Drugs: Bezuclastinib Tablets (Formulation B)
Bezuclastinib will be administered orally, once daily continuously for 28-day cycles
Treatment: Drugs: Placebo Tablets
Placebo will be administered orally, once daily continuously for 28-day cycles
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Part 1: Determine recommended dose of bezuclastinib (CGT9486) in subjects with NonAdvSM
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Assessment method [1]
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Selection of the recommended dose to be used in subsequent parts of the study.
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Timepoint [1]
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3 months
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Primary outcome [2]
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Part 2: Efficacy of bezuclastinib at the selected dose versus placebo
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Assessment method [2]
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Mean absolute change in a disease-specific patient reported outcome (PRO)
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Timepoint [2]
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6 months
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Primary outcome [3]
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Part 3: Safety and tolerability of bezuclastinib as assessed by number of adverse events
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Assessment method [3]
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CTCAE v5
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Timepoint [3]
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Up to 24 months
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Secondary outcome [1]
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Safety and tolerability of bezuclastinib as assessed by number of adverse events
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Assessment method [1]
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CTCAE v5
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Timepoint [1]
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Up to 24 months
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Secondary outcome [2]
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Proportion of subjects who had at least 50% reduction in serum tryptase
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Assessment method [2]
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Timepoint [2]
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Up to 24 months
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Secondary outcome [3]
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Proportion of subjects who had at least 50% reduction in mast cell burden
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Assessment method [3]
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Timepoint [3]
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Up to 24 months
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Secondary outcome [4]
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Proportion of subjects who had at least a 50% reduction in peripheral blood D816V allele fraction
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Assessment method [4]
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Timepoint [4]
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Up to 24 months
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Secondary outcome [5]
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Change and percent change in patient reported outcome (PRO) measures
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Assessment method [5]
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Timepoint [5]
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Up to 24 months
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Secondary outcome [6]
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Change and percent change in serum tryptase
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Assessment method [6]
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Timepoint [6]
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Up to 24 months
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Secondary outcome [7]
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Change and percent change in bone marrow mast cells
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Assessment method [7]
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Timepoint [7]
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Up to 24 months
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Secondary outcome [8]
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Change and percent change in the levels of KIT D816V mutation allele burden
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Assessment method [8]
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Timepoint [8]
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Up to 24 months
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Secondary outcome [9]
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Assess the pharmacokinetics (PK) of bezuclastinib in subjects with NonAdvSM
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Assessment method [9]
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Plasma concentrations of CGT9846
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Timepoint [9]
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Up to 24 months
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Secondary outcome [10]
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Change and percent change in the Mast Cell Quality of Life (MC-QOL) Score
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Assessment method [10]
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Scale of 0-100, higher numbers represent more severe impairment to quality of life.
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Timepoint [10]
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up to 24 months
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Secondary outcome [11]
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Change and percent change in 12-item Short Form Health Survey (SF-12)
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Assessment method [11]
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Scale of 0-100, higher numbers represent better symptom outcomes
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Timepoint [11]
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up to 24 months
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Secondary outcome [12]
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Change and percent change in EuroQol 5 Dimensions 5 Levels (EQ 5D-5L)
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Assessment method [12]
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Scale of 0-100, higher numbers represent better symptom outcomes
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Timepoint [12]
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up to 24 months
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Secondary outcome [13]
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Determine responder rates of subjects treated with bezuclastinib at the selected dose
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Assessment method [13]
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Response rate based on reduction in disease specific PRO
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Timepoint [13]
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6 months
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Eligibility
Key inclusion criteria
Key
1. Diagnosed with 1 of the following diagnoses according to the 2016 World Health
Organization (WHO) classification for systemic mastocytosis (SM):
- Indolent systemic mastocytosis (ISM), including the bone marrow mastocytosis
subvariant
- Smoldering systemic mastocytosis (SSM)
2. Moderate-to-severe symptoms based on a disease-specific PRO and after establishing a
stable regimen of at least 2 antimediator therapies over a 14-day eligibility period
3. Eastern Cooperative Oncology Group (ECOG) Performance Status 0 to 2
4. For patients receiving corticosteroids, the dose must be =10 mg/day of prednisone or
equivalent
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Diagnosed with any of the following WHO SM classifications: bone marrow mastocytosis,
advanced systemic mastocytosis including SM with associated hematologic neoplasm,
aggressive SM, mast cell leukemia; or mast cell sarcoma
2. Diagnosed with mastocytosis of the skin without systemic involvement
3. Received prior treatment with any targeted KIT inhibitor with the exception of
approved agents for the treatment of SM
4. Received prior cytoreductive therapy or investigational agent for <14 days or 5 half-
lives of the drug and for cladribine, interferon alpha, pegylated interferon, or
antibody therapy <28 days or 5 half-lives of the drug (whichever is longer), before
starting screening assessments
5. Received radiotherapy or psoralen and ultraviolet A therapy <14 days before starting
screening assessments
6. Received any hematopoietic growth factor support <14 days before starting screening
assessments
7. History of clinically significant bleeding event within 30 days before the first dose
of study drug or need for therapeutic anticoagulation on study
8. Need for treatment of corticosteroids at >10 mg/day of prednisone or equivalent
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
27/06/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/11/2026
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Actual
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Sample size
Target
138
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,SA
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Recruitment hospital [1]
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Royal Prince Alfred Hospital - Camperdown
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Recruitment hospital [2]
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Royal Adelaide Hospital - Adelaide
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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5000 - Adelaide
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Arizona
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Country [2]
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United States of America
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State/province [2]
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California
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Country [3]
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United States of America
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State/province [3]
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Florida
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Country [4]
0
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United States of America
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State/province [4]
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Georgia
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Country [5]
0
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United States of America
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State/province [5]
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Illinois
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Country [6]
0
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United States of America
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State/province [6]
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Maryland
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Country [7]
0
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United States of America
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State/province [7]
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Massachusetts
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Country [8]
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United States of America
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State/province [8]
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Michigan
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Country [9]
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United States of America
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State/province [9]
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Minnesota
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Country [10]
0
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United States of America
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State/province [10]
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Missouri
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Country [11]
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United States of America
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State/province [11]
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New Hampshire
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Country [12]
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United States of America
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State/province [12]
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New York
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Country [13]
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United States of America
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State/province [13]
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North Carolina
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Country [14]
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United States of America
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State/province [14]
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Ohio
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Country [15]
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United States of America
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State/province [15]
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Tennessee
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Country [16]
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United States of America
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State/province [16]
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Texas
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Country [17]
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Belgium
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State/province [17]
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Edegem
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Country [18]
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Canada
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State/province [18]
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Alberta
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Country [19]
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Canada
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State/province [19]
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Ontario
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Country [20]
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Czechia
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State/province [20]
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Praha 10
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Country [21]
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France
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State/province [21]
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Paris
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Country [22]
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France
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State/province [22]
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Toulouse
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Country [23]
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Germany
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State/province [23]
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Aachen
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Country [24]
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Germany
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State/province [24]
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Berlin
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Country [25]
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Germany
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State/province [25]
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Mannheim
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Country [26]
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Greece
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State/province [26]
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Athens
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Country [27]
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Ireland
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State/province [27]
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Dublin
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Country [28]
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Italy
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State/province [28]
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Bologna
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Country [29]
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Italy
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State/province [29]
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Firenze
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Country [30]
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Italy
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State/province [30]
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Pavia
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Country [31]
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Italy
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State/province [31]
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Rome
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Country [32]
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Netherlands
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State/province [32]
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Groningen
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Country [33]
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Norway
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State/province [33]
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Oslo
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Country [34]
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Poland
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State/province [34]
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Gdansk
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Country [35]
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Spain
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State/province [35]
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Barcelona
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Country [36]
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Spain
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State/province [36]
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Madrid
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Country [37]
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Spain
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State/province [37]
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Toledo
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Country [38]
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Switzerland
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State/province [38]
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Basel
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Country [39]
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United Kingdom
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State/province [39]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Cogent Biosciences, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a multi-part, randomized, double-blind, placebo-controlled Phase 2 clinical study
comparing the safety and efficacy of bezuclastinib (CGT9486) plus best supportive care (BSC)
with placebo plus BSC in patients with nonadvanced systemic mastocytosis (NonAdvSM),
including indolent systemic mastocytosis and smoldering systemic mastocytosis, whose symptoms
are not adequately controlled by BSC. This study will be conducted in three parts. Patients
in Parts 1a, 1b and 2 will receive bezuclastinib or placebo, and may roll over onto Part 3 to
receive treatment with bezuclastinib.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05186753
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Rachael Easton, MD, PhD
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Address
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Cogent Biosciences
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Hina Jolin, PharmD
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Address
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Country
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Phone
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+1 (617) 945-5576
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05186753
Download to PDF