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JOURNAL ONKOLOGIE – STUDIE

(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis

Rekrutierend

NCT-Nummer:
NCT05186753

Studienbeginn:
Juni 2022

Letztes Update:
08.04.2024

Wirkstoff:
Bezuclastinib Tablets (Formulation A), Bezuclastinib Tablets (Formulation B), Placebo Tablets

Indikation (Clinical Trials):
Mastocytosis, Mastocytosis, Systemic

Geschlecht:
Alle

Altersgruppe:
Erwachsene (18+)

Phase:
Phase 2

Sponsor:
Cogent Biosciences, Inc.

Collaborator:
-

Studienleiter

Rachael Easton, MD, PhD
Study Director
Cogent Biosciences

Kontakt

Studienlocations
(3 von 51)

Universitaetsklinikum Aachen, AoeR
52074 Aachen
GermanyRekrutierend» Google-Maps
Charité Universitätsmedizin Berlin
12203 Berlin
(Berlin)
GermanyRekrutierend» Google-Maps
University Medical Centre Mannheim
68167 Mannheim
(Baden-Württemberg)
GermanyRekrutierend» Google-Maps
One of a Kind Clinical Research Center
85258 Scottsdale
United StatesRekrutierend» Google-Maps
Modena Allergy and Asthma Clinical
92037 La Jolla
United StatesRekrutierend» Google-Maps
Innovative Research of West Florida
33756 Clearwater
United StatesRekrutierend» Google-Maps
Mayo Clinic - Jacksonville
32224 Jacksonville
United StatesRekrutierend» Google-Maps
Mid Florida Hematology and Oncology Center
32763 Orange City
United StatesRekrutierend» Google-Maps
Walter Reed National Military Medical Center
20814 Bethesda
United StatesRekrutierend» Google-Maps
Allervie Clinical Research
20769 Glenn Dale
United StatesRekrutierend» Google-Maps
Chesapeake Research Center
21162 White Marsh
United StatesRekrutierend» Google-Maps
Brigham and Women's Hospital
02115 Boston
United StatesRekrutierend» Google-Maps
Dana Farber Cancer Institute
02215 Boston
United StatesRekrutierend» Google-Maps
University of Michigan
48109 Ann Arbor
United StatesRekrutierend» Google-Maps
Mayo Clinic- Rochester
55905 Rochester
United StatesRekrutierend» Google-Maps
Washington University at St. Louis
63110 Saint Louis
United StatesRekrutierend» Google-Maps
Dartmouth Hitchcock Medical Center
03766 Lebanon
United StatesRekrutierend» Google-Maps
Icahn School of Medicine at Mount Sinai
10029 New York
United StatesRekrutierend» Google-Maps
University of Cincinnati
45221 Cincinnati
United StatesRekrutierend» Google-Maps
The Ohio State University
43210 Columbus
United StatesRekrutierend» Google-Maps
Vanderbilt University Medical Center
37235 Nashville
United StatesRekrutierend» Google-Maps
The University of Texas MD Anderson Cancer Center
77030 Houston
United StatesRekrutierend» Google-Maps
Royal Prince Alfred Hospital
2050 Camperdown
AustraliaRekrutierend» Google-Maps
Antwerp University Hospital (UZA)
2650 Edegem
BelgiumRekrutierend» Google-Maps
Toronto Allergy and Asthma Clinic
M5G 1E2 Toronto
CanadaRekrutierend» Google-Maps
Fakultni nemocnice Kralovske Vinohrady
Praha 10
CzechiaRekrutierend» Google-Maps
AP-HP- Hopital Pitie-Salpetriere
75013 Paris
FranceRekrutierend» Google-Maps
CHU de Toulouse - Hopital Larrey
31400 Toulouse
FranceRekrutierend» Google-Maps
Attikon University General Hospital Athens
Athens
GreeceRekrutierend» Google-Maps
IRCCS Azienda Ospedaliero-Universitaria di Bologna - Policlinico di Sant'Orsola
40138 Bologna
ItalyRekrutierend» Google-Maps
Azienda Ospedaliero-Universitaria Careggi
Firenze
ItalyRekrutierend» Google-Maps
Fondazione IRCCS Policlinico San Matteo
27100 Pavia
ItalyRekrutierend» Google-Maps
Policlinico Universitario Agostino Gemelli
168 Rome
ItalyRekrutierend» Google-Maps
University Medical Center Groningen
9713 Groningen
NetherlandsRekrutierend» Google-Maps
Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii
Gdańsk
PolandRekrutierend» Google-Maps
Hospital Universitario Vall d'Hebron
08035 Barcelona
SpainRekrutierend» Google-Maps
Hospital Universitario Ramon y Cajal
28046 Madrid
SpainRekrutierend» Google-Maps
Instituto de Estudios de Mastocitosis de Castilla La Mancha-Hospital Virgen del Valle
Toledo
SpainRekrutierend» Google-Maps
Alle anzeigen

Studien-Informationen

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.

Ein-/Ausschlusskriterien

Key Inclusion Criteria:

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

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

Studien-Rationale

Primary outcome:

1. Part 1: Determine recommended dose of bezuclastinib (CGT9486) in subjects with NonAdvSM (Time Frame - 3 months):
Selection of the recommended dose to be used in subsequent parts of the study.

2. Part 2: Efficacy of bezuclastinib at the selected dose versus placebo (Time Frame - 6 months):
Mean absolute change in a disease-specific patient reported outcome (PRO)

3. Part 3: Safety and tolerability of bezuclastinib as assessed by number of adverse events (Time Frame - Up to 24 months):
CTCAE v5

Secondary outcome:

1. Safety and tolerability of bezuclastinib as assessed by number of adverse events (Time Frame - Up to 24 months):
CTCAE v5

2. Proportion of subjects who had at least 50% reduction in serum tryptase (Time Frame - Up to 24 months)

3. Proportion of subjects who had at least 50% reduction in mast cell burden (Time Frame - Up to 24 months)

4. Proportion of subjects who had at least a 50% reduction in peripheral blood D816V allele fraction (Time Frame - Up to 24 months)

5. Change and percent change in patient reported outcome (PRO) measures (Time Frame - Up to 24 months)

6. Change and percent change in serum tryptase (Time Frame - Up to 24 months)

7. Change and percent change in bone marrow mast cells (Time Frame - Up to 24 months)

8. Change and percent change in the levels of KIT D816V mutation allele burden (Time Frame - Up to 24 months)

9. Assess the pharmacokinetics (PK) of bezuclastinib in subjects with NonAdvSM (Time Frame - Up to 24 months):
Plasma concentrations of CGT9846

10. Change and percent change in the Mast Cell Quality of Life (MC-QOL) Score (Time Frame - up to 24 months):
Scale of 0-100, higher numbers represent more severe impairment to quality of life.

11. Change and percent change in 12-item Short Form Health Survey (SF-12) (Time Frame - up to 24 months):
Scale of 0-100, higher numbers represent better symptom outcomes

12. Change and percent change in EuroQol 5 Dimensions 5 Levels (EQ 5D-5L) (Time Frame - up to 24 months):
Scale of 0-100, higher numbers represent better symptom outcomes

13. Determine responder rates of subjects treated with bezuclastinib at the selected dose (Time Frame - 6 months):
Response rate based on reduction in disease specific PRO

Studien-Arme

  • 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

Geprüfte Regime

  • Bezuclastinib Tablets (Formulation A) (CGT9486 / PLX9486 / ):
    Bezuclastinib will be administered orally, once daily continuously for 28-day cycles
  • Bezuclastinib Tablets (Formulation B) (CGT9486 / PLX9486 / ):
    Bezuclastinib will be administered orally, once daily continuously for 28-day cycles
  • Placebo Tablets:
    Placebo will be administered orally, once daily continuously for 28-day cycles

Quelle: ClinicalTrials.gov


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