Muckle-Wells syndrome
Conditions
Interventions
Product Name: None
Product Code: VX-765
Pharmaceutical Form: Tablet
CAS Number: 273404-37-8
Current Sponsor code: VX-765
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration num
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Confirmed diagnosis of Muckle-Wells syndrome, as defined by mutations of the NALP3/CIAS1 gene, associated with laboratory evidence of acute phase response; symptoms of rash, conjunctivitis, fever, fatigue, or arthralgia; SAA greater than 10 mg/L on 2 consecutive occasions, patient agreement to discontinue anti-inflammatory medications. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Pregnancy/lactation, any chronic inflammatory disorder other than Muckle-Wells Syndrome, medication that may alter drug absorption, recent use of other investigational drugs, recent blood/plasma donation, drug allergy/hypersensitivity
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Investigate safety/tolerability, anti-inflammatory effect and pharmacokinetics of VX-765 in patients with Muckle-Wells Syndrome.;Secondary Objective: ;Primary end point(s): Safety and tolerability measurements; anti-inflammatory measurements, pharmacokinetic parameters | — |
Countries
United Kingdom
Outcome results
None listed