Chronic Inducible Urticaria, Chronic Spontaneous Urticaria
Conditions
Keywords
Chronic Inducible Urticaria, Chronic Spontaneous Urticaria, BLU-808, CIndU, CSU, Chronic Urticaria, CU, Cold Urticaria, ColdU, Symptomatic Dermographism, SD
Brief summary
This is a 2-part, proof-of-concept study to be conducted globally, designed to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of BLU-808, a wild type KIT inhibitor, in participants with CIndU (Part A) or CSU (Part B).
Interventions
DRUGBLU-808
Oral administration
DRUGPlacebo
Oral administration
Sponsors
Blueprint Medicines Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Masking description
Arms A1 and A3 are non-randomized and open label. Arm B is randomized and double blind.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Part A: Confirmed diagnosis of CIndU for ≥3 months prior to Day 1 that is inadequately controlled with second generation H1-antihistamines. * Part B: Confirmed diagnosis of CSU for ≥3 months prior to Day 1 that is inadequately controlled with second generation H1-antihistamines. Key
Exclusion criteria
* Part A: Any active urticaria that may interfere with study assessments. * Part B: Participant has a clearly defined predominant cause of chronic urticaria or sole trigger such as symptomatic dermographism and cold-induced urticaria. * Part A and Part B: Any other skin disease associated with chronic itching or angioedema that might influence the study evaluations and results, skin diseases associated with only wheals and no itch, or autoinflammatory diseases with urticarial lesions. * Part A and Part B: Significant medical, psychiatric, or surgical conditions, or physical findings that may affect participant safety, study drug metabolism, study participation, or assessment of study results. * Part A and Part B: Abnormal laboratory values that may pose risks or interfere with study participation. * Part A and Part B: Pregnancy or plans for pregnancy; breastfeeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A and Part B: Number of Participants with Treatment-emergent Adverse Events (TEAEs) | Day 1 through Week 16 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part A: Mean Change From Baseline in Critical Temperature Threshold (CTT) at Week 12 | Baseline, Week 12 | CTT is the temperature at which CIndU symptoms appear. |
| Part A: Mean Change From Baseline in Total Fric Score (TFS) at Week 12 | Baseline, Week 12 | TFS is a measure of CIndU trigger thresholds and disease activity. |
| Part A and Part B: Complete Response Rate | Week 12 | — |
| Part A and Part B: Absolute Change From Baseline in Serum Tryptase Concentration at Week 12 | Baseline, Week 12 | — |
| Part A and Part B: Percent Change From Baseline in Serum Tryptase Concentration at Week 12 | Baseline, Week 12 | — |
| Part A and Part B: Area Under the Curve (AUC) of BLU-808 | Day 1 to Day 57 | — |
| Part A and Part B: Maximum Plasma Concentration (Cmax) of BLU-808 | Day 1 to Day 57 | — |
| Part A and Part B: Minimum Plasma Concentration (Cmin) of BLU-808 | Day 1 to Day 57 | — |
| Part A and Part B: Apparent Clearance (CL/F) of BLU-808 | Day 1 to Day 57 | — |
| Part A and Part B: Apparent Volume of Distribution for the Central Compartment (Vc/F) of BLU-808 | Day 1 to Day 57 | — |
| Part A and Part B: Terminal Half-life (t½) of BLU-808 | Day 1 to Day 57 | — |
| Part B: Mean Change From Baseline in Urticaria Activity Score Over 7 Days (UAS7) at Week 12 | Baseline, Week 12 | UAS7 is a patient-reported outcome used to assess symptoms in participants with CSU. |
Countries
Denmark, Germany, Italy, Spain, Taiwan, United States
Contacts
CONTACTBlueprint Medicines
CONTACTBlueprint Medicines, EU Contact
Outcome results
None listed