Neoplasms

Immune System Diseases

All Conditions

Blood and Lymph Conditions

Skin and Connective Tissue Diseases

Respiratory Tract (Lung and Bronchial) Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Digestive System Diseases

Behaviors and Mental Disorders

Diseases and Abnormalities at or Before Birth

Nutritional and Metabolic Diseases

Rare Diseases

Mastocytosis

Leukemia

Carcinoma

Melanoma

Carcinoma, Non-Small-Cell Lung

Renal Insufficiency

Glioma

Sarcoma

Lung Neoplasms

Gastrointestinal Stromal Tumors

Aggression

Mastocytosis, Systemic

Mast Cell Activation Disorders

Leukemia, Mast-Cell

Ichthyosis, X-Linked

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Gastrointestinal Neoplasms

Digestive System Neoplasms

Gastrointestinal Diseases

Soft Tissue Sarcoma

Germ Cells Tumors

Penile Cancer

Systemic Mastocytosis

Escalation Phase: DCC-2618 tablets in escalating dose cohorts given orally BID (twice daily) every 12 hours or once daily (QD) for repeated 28-day cycles. Participants may continue to receive study drug until discontinuation criteria are met. \[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with GIST who have received 3 prior therapies. \[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with GIST who have received 4 prior therapies. \[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with GIST who have received at least one prior therapy and no more than 2 prior therapies. \[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with systemic mastocytosis and other hematologic malignancies.\[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with malignant gliomas.\[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with other solid tumors. \[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with melanomas. \[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with soft tissue sarcomas.\[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with germ cell, penile cancer and non-small cell lung carcinoma (NSCLC). \[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles in patients with GIST and other solid tumors with renal impairment. \[Closed for Enrollment\]

150 mg DCC-2618 given once daily in repeated 28-day cycles for active patients from the Escalation and Extension Phases.

DCC-2618

Deciphera Pharmaceuticals, LLC

This is a Phase 1, open-label, first-in-human (FIH) dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antitumor activity of DCC-2618, administered orally (PO), in adult patients with advanced malignancies. The study consists of 2 parts, a dose-escalation phase, and an expansion phase. All active patients (from both dose-escalation and expansion phases) will then transition into an extension phase.

A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced Malignancies

A Multicenter Phase 1, Open-Label Study of DCC-2618 to Assess Safety, Tolerability, Efficacy, and Pharmacokinetics in Patients With Advanced Malignancies

Dose limiting toxicities, AEs, SAEs, discontinuation of drug due to toxicity, physical exams and ECOG PS, ophthalmologic examinations, changes from baseline in laboratory parameters, electrocardiograms, LVEF, and vital signs.

Objective response rate (ORR); Disease control rate (DCR)

Deciphera Pharmaceuticals LLC

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Hematologic Diseases

Acute Graft Versus Host Disease

Single interview lasting approximately one hour

Interview

The purpose of this study is to explore the key symptoms of individuals with systemic mastocytosis. Interviews of enrolled individuals will be conducted to learn about the disease symptoms and condition. The interview will last approximately 60 minutes and will be conducted by a trained interviewer, be audio-recorded (with patient consent), and all information provided by the patient will be treated confidentially and made anonymous so that it is non-identifiable. The interview may be conducted face-to-face, over the phone, or virtually via Skype, a free video conferencing program, depending upon the patient's geographic location. Patients will be compensated for their participating time.

This is not a medication-related study, and no medication will be distributed or tested during this study. Participation in this study will not affect any treatment or assistance that a patient currently receives or may receive in the future.

Patient-Reported Outcome Questionnaire for Systemic Mastocytosis

Patient-Reported Outcome Questionnaire Development for Symptoms of Systemic Mastocytosis

Blueprint Medicines Corporation

Adelphi Values LLC

Symptoms and General Pathology

Nervous System Diseases

Syndrome

Preleukemia

Leukemia, Myelomonocytic, Chronic

Leukemia, Myelomonocytic, Juvenile

Myelodysplastic Syndromes

Hypersensitivity

Skin Diseases

Hematologic Neoplasms

Myeloproliferative Disorders

Soft Tissue Neoplasms

Urticaria

Urticaria Pigmentosa

Immune Complex Diseases

Dementia

Aberrant Motor Behavior in Dementia

Behavioral Symptoms

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Chronic Myelomonocytic Leukemia

Juvenile Myelomonocytic Leukemia

Chronic Graft Versus Host Disease

Chronic Myeloproliferative Disorders

All Drugs and Chemicals

Antigen-Antibody Complex

Bezuclastinib is administered as tablets to be taken orally, continuously in 28-day cycles.

bezuclastinib

Hina Jolin, PharmD

Rachael Easton, MD, Ph.D.

This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).

There are two parts to this study, including Part I, Dose Optimization, and Part II Expansion. Part II is separated into two stages.

(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis

A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis

Incidence of AEs according to CTCAE version 5.0 or higher

Percentage change in plasma concentrations of bezuclastinib

Percentage change in mast cell infiltration in the bone marrow and percentage change in eosinophilia and monocytosis in the blood

0 -10 points (higher values represent worse symptom outcomes)

0 - 7 points (higher values represent better symptom outcomes)

0 - 100 (higher values represent better symptom outcomes)

0 - 252 (higher values represent worse symptom outcomes)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced Malignancies	NCT02571036	Gastrointestina... Advanced System... Advanced Cancer...	DCC-2618 DCC-2618	18 Years -	Deciphera Pharmaceuticals LLC
Patient-Reported Outcome Questionnaire for Systemic Mastocytosis	NCT02380222	Aggressive Syst... SM w Assoc Clon... Mast Cell Leuke... Smoldering Syst... Indolent System...	Interview	18 Years -	Adelphi Values LLC
A Safety, Tolerability and PK Study of DCC-2618 in Patients With Advanced Malignancies	NCT02571036	Gastrointestina... Advanced System... Advanced Cancer...	DCC-2618 DCC-2618	18 Years -	Deciphera Pharmaceuticals LLC
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis	NCT04996875	Advanced System... SM With an Asso... Mast Cell Leuke... Aggressive Syst...	bezuclastinib	18 Years -	Cogent Biosciences, Inc.
Patient-Reported Outcome Questionnaire for Systemic Mastocytosis	NCT02380222	Aggressive Syst... SM w Assoc Clon... Mast Cell Leuke... Smoldering Syst... Indolent System...	Interview	18 Years -	Adelphi Values LLC
Patient-Reported Outcome Questionnaire for Systemic Mastocytosis	NCT02380222	Aggressive Syst... SM w Assoc Clon... Mast Cell Leuke... Smoldering Syst... Indolent System...	Interview	18 Years -	Adelphi Values LLC

Spots Global Cancer Trial Database for aggressive systemic mastocytosis (asm)