SNPMiner Trials by Shray Alag


SNPMiner Trials: Clinical Trial Report


Report for Clinical Trial NCT03731260

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy

This is a Phase 2, randomized, double-blind, placebo-controlled study comparing the efficacy and safety of avapritinib + best supportive care (BSC) with placebo + BSC in patients with indolent systemic mastocytosis (ISM) and smoldering systemic mastocytosis (SSM) whose symptoms are not adequately controlled by BSC. The study will be conducted in 3 parts. All patients will receive treatment with avapritinib during Part 3 including those rolling over from the placebo group.

NCT03731260 Indolent Systemic Mastocytosis Smoldering Systemic Mastocytosis
MeSH: Mastocytosis Mastocytosis, Systemic
HPO: Mastocytosis

2 Interventions

Name: Avapritinib

Description: Avapritinib tablet

Type: Drug

(Part 1) Avapritinib Dose 1 + BSC (Part 1) Avapritinib Dose 2 + BSC (Part 1) Avapritinib Dose 3 + BSC (Part 2) Avapritinib RP2D + BSC (Part 3) Avapritinib RP2D + BSC

Name: Placebo

Description: Placebo tablet

Type: Drug

(Part 1) Placebo + BSC (Part 2) Placebo + BSC


Primary Outcomes

Measure: Recommended Phase 2 dose (RP2D) in patients with ISM (Part 1)

Time: 9 months

Description: 0 - 120 points (higher value represents worse symptom outcomes)

Measure: Mean change in ISM Symptom Assessment Form (ISM-SAF) total symptom score (TSS) in patients with ISM and SSM (Part 2)

Time: 12 weeks

Description: 0 - 120 points (higher value represents worse symptom outcomes)

Measure: Mean change in ISM Symptom Assessment Form (ISM-SAF) total symptom score (TSS) in patients with ISM and SSM (long term, Part 3)

Time: Up to 5 years

Description: CTCAE version 5.0

Measure: Safety of avapritinib as assessed by number of adverse events (long term, Part 3)

Time: Up to 5 years

Secondary Outcomes

Measure: Change in serum tryptase

Time: Up to 5 years

Measure: Change in V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) allele burden in blood

Time: Up to 5 years

Measure: Change in bone marrow mast cells

Time: 12 weeks

Measure: Change in skin mast cells in patients with baseline cutaneous mastocytosis (CM)

Time: 12 weeks

Measure: Change in percent body surface area (BSA) involved by CM

Time: Every 12 weeks during treatment through Week 52 of Part 3

Measure: Change in pigmentation of cutaneous lesions in patients with baseline CM as assessed by skin photography

Time: Every 12 weeks during treatment through Week 52 of Part 3

Measure: Change in best supportive care (BSC) concomitant medication usage

Time: Up to 5 years

Description: 0 - 100 points (higher value represents worse symptom outcomes)

Measure: Change in Mastocytosis Quality of Life Questionnaire (MC-QoL)

Time: Each study visit through Part 3 Week 52

Description: 0 - 10 points (higher value represents worse symptom outcomes)

Measure: Change in Patient's Global Impression of Symptom Severity (PGIS)

Time: Each study visit through Part 3 Week 52

Description: 0 - 100 points (higher value represents better symptom outcomes)

Measure: Change in 12-item Short Form Health Survey (SF-12)

Time: Each study visit through Part 3 Week 52

Description: 1 - 7 (higher value represents worse symptom outcomes)

Measure: Change in Patients' Global Impression of Change (PGIC)

Time: Each study visit through Part 3 Week 52

Description: 0 - 100 (higher value represents better symptom outcomes)

Measure: Change in EuroQuol 5 Dimensions 5 Levels (EQ 5D-5L)

Time: Each study visit through Part 3 Week 52

Description: CTCAE version 5.0

Measure: Safety of avapritinib as assessed by number of adverse events

Time: Up to 5 years

Description: h•ng/mL

Measure: Area Under Curve (0 to Tau) for avapritinib (Parts 1 and 2 only)

Time: Every cycle (28 days) up to cycle 4

Purpose: Treatment

Allocation: Randomized

Parallel Assignment


There is one SNP

SNPs


1 D816V

Change in V-kit Hardy-Zuckerman 4 feline sarcoma viral oncogene homolog aspartate 816 valine (KIT D816V) allele burden in blood. --- D816V ---



HPO Nodes


HPO:
Mastocytosis
Genes 1
KIT