Pachyonychia congenita. Enlarged and hypertrophic nails
Conditions
Interventions
Intervention 1: Intervention group: Participants will be treated with simvastatin, given as a single oral dose of 40 mg in the evening. In patients aged 10 to 18 years, the treatment will be initiated
Sponsors
Shahid Beheshti University of Medical Sciences
Eligibility
Sex/Gender
All
Age
10 Years to 65 Years
Inclusion criteria
Inclusion criteria: .Weight equal or more than 32kg Mentally healthy and able to clearly understand, assess, and respond questions.
Exclusion criteria
Exclusion criteria: Cardiovascular disease. Renal impairment. Hepatic impairment or severe gastrointestinal disease. Diabetes. History of myopathy. Active fingernail and cutaneous cyst infection. If there is an infection in skin or fingernail, it must be treated before starting the trial. Pregnant or breastfeeding. History of hypersensitivity to statins. Previous consumption of statins for other reasons. History of drug or alcohol abuse.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Area occupied by heperkeratotic lesions on palms and soles. Timepoint: At the beginning and end of each study phase. Method of measurement: Photography, imagej software.;Plantar lesions maximum thickness. Timepoint: At the beginning and end of each study phase. Method of measurement: 20 MHz B mode Ultrasonography.;Maximum nail thickness. Timepoint: At the beginning and end of each study phase. Method of measurement: 20 MHz B mode Ultrasonography.;Self-report nail grooming need. Timepoint: At the beginning and end of each study phase. Method of measurement: Likert scale.;Self-report hyperkeratotic lesion grooming need. Timepoint: At the beginning and end of each study phase. Method of measurement: Likert scale.;Physicians satisfaction with patients response. Timepoint: At the beginning and end of each study phase. Method of measurement: Likert scale.;Patients mobility,treadmill tolerance test. Timepoint: At the beginning and end of each study phase. Method of measurement: The maximum time a patient could tolerate walking a on a treadmill.;Mcgill pain questionnaire. Timepoint: At the beginning and end of each study phase. Method of measurement: Questionnaire.;Dermatology Life Quality Index (DLQI). Timepoint: At the beginning and end of each study phase. Method of measurement: Questionnaire.;Keratin gene expression. KRT 6a, 6b, 6c, 16, 17. Timepoint: At the beginning and end of each study phase. Method of measurement: Histophatologic evluation. | — |
Secondary
| Measure | Time frame |
|---|---|
| Simvastatin side-effects. Timepoint: Throughout the treatment. Method of measurement: Any exacerbation of clinical signs or laboratory changes.;Patient compliance with medication. Timepoint: Throughout the treatment. Method of measurement: According to physicians estimates, Likert scale. | — |
Countries
Iran (Islamic Republic of), United States of America
Contacts
Public ContactFateme Rajabi
Shahid Beheshti University of Medical Sciences
Outcome results
None listed