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Assessing the effectiveness of simvastatin in the treatment of patients with pachyonychia congenita: A crossover randomized controlled trial.

Protocol summary

Study aim
The efficacy of simvastatin in reducing signs and symptoms in patients with pachyonychia congenita.
Design
A multicenter concealed, randomized, blinded, sham-controlled, crossover clinical trial of 20 patients, enrolled between January 2019 and September 2019 and followed for 14 months.
Settings and conduct
The patients will be randomly assigned into two groups. The participants in the case group will receive a single dose of simvastatin 40 mg per day (20 mg in individuals aged 10-18) for six months followed by a washout period of 2 months. Then they will crossover to the control group. The participants in the control arm will receive placebo for six months. After a washout period of two months, they will crossover to the case group. Patients will be evaluated throughout the study for clearance of palmoplantar and nail lesions, and overall improvement in quality of life. Histologic specimens will be obtained at the beginning and the end of each phase for investigating the molecular effects of simvastatin.
Participants/Inclusion and exclusion criteria
Inclusion Criteria: Age between10 to 65 years. Weight equal or more than 32kg. Mentally able to respond to questions. Exclusion criteria: Comorbidities such as cardiovascular, renal or hepatic impairment or diabetes. Drug-related concerns such as a previous history of myopathy, hypersensitivity to statins, pregnancy, and breastfeeding. Confounding factors such as cutaneous infection, previous consumption of statins, and drug/alcohol abuse.
Intervention groups
Patients with genetically confirmed diagnosis of pachyonychia congenita are randomly assigned in two groups. The control group will receive placebo and the intervention group will receive simvastatin for 6 months. After a washout period, the groups will cross over.
Main outcome variables
Hyperkeratotic lesion thickness, Nail thickness, Skin pathological changes, Pain, Quality of life.

General information

Reason for update
Acronym
IRCT registration information
IRCT registration number: IRCT20160320027109N3
Registration date: 2019-01-09, 1397/10/19
Registration timing: registered_while_recruiting

Last update: 2019-01-09, 1397/10/19
Update count: 0
Registration date
2019-01-09, 1397/10/19
Registrant information
Name
Fahimeh Abdollahimajd
Name of organization / entity
Skin Research Center
Country
Iran (Islamic Republic of)
Phone
+98 21 2274 1507
Email address
fabdollahimajd@sbmu.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2019-01-01, 1397/10/11
Expected recruitment end date
2019-09-02, 1398/06/11
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Assessing the effectiveness of simvastatin in the treatment of patients with pachyonychia congenita: A crossover randomized controlled trial.
Public title
Effect of simvastatin in treatment of pachyonychia congenita
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
.Weight equal or more than 32kg Mentally healthy and able to clearly understand, assess, and respond questions.
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.
Age
From 10 years old to 65 years old
Gender
Both
Phase
2-3
Groups that have been masked
  • Participant
  • Care provider
  • Investigator
  • Outcome assessor
  • Data analyser
Sample size
Target sample size: 20
Randomization (investigator's opinion)
Randomized
Randomization description
Recruited patients are randomly allocated to either treatment or control arm. Both arms are in equal size. Randomization sequence is generated by Random Allocation Software version 1.0 May 2004, using a simple random method. It generates a randomization code for each participant (individual randomization). Randomization is run just one time at the beginning of the study. Then participants crossover based on the first randomized allocation. Sequentially numbered sealed opaque envelops are used to conceal the allocation. Each participant receives one envelope containing the randomization code.
Blinding (investigator's opinion)
Triple blinded
Blinding description
Blinding of the participants is achieved via administration of placebo which has the same package, figure, color, taste, smell, size, dosage, and route of administration as the drug used in the case group (simvastatin and the placebo are provided by the same company). In order to blind the outcome assessors, the researchers, and the data analyzers, simvastatin and placebo are referred to as A and B throughout the investigation.
Placebo
Used
Assignment
Crossover
Other design features

Secondary Ids

empty

Ethics committees

1

Ethics committee
Name of ethics committee
Ethics committee of Skin Research Center Shahid Beheshti University of Medical Sciences
Street address
The SBMU SRC (Skin Research Center),Shohada-E- Tajrish Educational Hospital,Qods Sq. ,Tehran, Iran
City
Tehran
Province
Tehran
Postal code
1989934148
Approval date
2018-12-09, 1397/09/18
Ethics committee reference number
IR.SBMU.SRC.REC.1397.019

Health conditions studied

1

Description of health condition studied
Pachyonychia congenita
ICD-10 code
Q84.5
ICD-10 code description
Enlarged and hypertrophic nails

Primary outcomes

1

Description
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

2

Description
Plantar lesions maximum thickness
Timepoint
At the beginning and end of each study phase
Method of measurement
20 MHz B mode Ultrasonography

3

Description
Maximum nail thickness
Timepoint
At the beginning and end of each study phase
Method of measurement
20 MHz B mode Ultrasonography

4

Description
Self-report nail grooming need
Timepoint
At the beginning and end of each study phase
Method of measurement
Likert scale

5

Description
Self-report hyperkeratotic lesion grooming need
Timepoint
At the beginning and end of each study phase
Method of measurement
Likert scale

6

Description
Physicians satisfaction with patients response
Timepoint
At the beginning and end of each study phase
Method of measurement
Likert scale

7

Description
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

8

Description
Mcgill pain questionnaire
Timepoint
At the beginning and end of each study phase
Method of measurement
Questionnaire

9

Description
Dermatology Life Quality Index (DLQI)
Timepoint
At the beginning and end of each study phase
Method of measurement
Questionnaire

10

Description
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 outcomes

1

Description
Simvastatin side-effects
Timepoint
Throughout the treatment.
Method of measurement
Any exacerbation of clinical signs or laboratory changes

2

Description
Patient compliance with medication
Timepoint
Throughout the treatment.
Method of measurement
According to physicians estimates, Likert scale.

Intervention groups

1

Description
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 with 20 mg of simvastatin. Before the beginning of the treatment and after 6 months on treatment, patients will be evaluated for the change in area and thickness of the lesions and indicators related to pain and quality of life. A biopsy specimen will be obtained both before and after the treatment to evaluate gene expression profiles. After a washout period of two months without treatment, patients will be assigned to the control group.
Category
Treatment - Drugs

2

Description
Control group:Participants will receive a single oral dose in the evening. Before the beginning of the treatment and after 6 months on treatment, patients will be evaluated for the change in area and thickness of the lesions and indicators related to pain and quality of life. A biopsy specimen will be obtained both before and after the treatment to evaluate gene expression profiles. After a washout period of two months without treatment, patients will be assigned to the control group.
Category
Treatment - Drugs

Recruitment centers

1

Recruitment center
Name of recruitment center
Dermatology clinic, Shohada-e Tajrish Hospital
Full name of responsible person
Fahimeh Abdollahimajd
Street address
Shohada-e Tajrish Hospital,Qods Sq. ,Tehran, Iran
City
Tehran
Province
Tehran
Postal code
1989934148
Phone
+98 21 2274 1507
Email
fabdollahimajd@sbmu.ac.ir

2

Recruitment center
Name of recruitment center
Department of Dermatology and Cutaneous Biology, Thomas Jefferson University
Full name of responsible person
Hassan Vahidnezhad
Street address
233 S, 10th Street, Suite 450 BLSB.
City
Philadelphia
Postal code
PA 19107
Phone
+1 215-503-5785
Email
Hassan.Vahidnezhad@jefferson.edu
Web page address

Sponsors / Funding sources

1

Sponsor
Name of organization / entity
Shahid Beheshti University of Medical Sciences
Full name of responsible person
Dr. Hamideh Moravvej
Street address
Skin Research Center, Shohada-e Tajrish Hospital, Qods sq, Tehran, Iran
City
Tehran
Province
Tehran
Postal code
1989934148
Phone
+98 21 2274 1507
Email
fabdollahimajd@sbmu.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Shahid Beheshti University of Medical Sciences
Proportion provided by this source
40
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic

Person responsible for general inquiries

Contact
Name of organization / entity
Shahid Beheshti University of Medical Sciences
Full name of responsible person
Fateme Rajabi
Position
Dermatology resident
Latest degree
Medical doctor
Other areas of specialty/work
Dermatology
Street address
The SBMU SRC (Skin Research Center),Shohada-E- Tajrish Educational Hospital,Qods Sq. ,Tehran, Iran
City
Tehran
Province
Tehran
Postal code
1989934148
Phone
+98 21 2274 1507
Email
fatemarajabi@gmail.com

Person responsible for scientific inquiries

Contact
Name of organization / entity
Shahid Beheshti University of Medical Sciences
Full name of responsible person
Fahimeh Abdollahimajd
Position
Assitant Professor
Latest degree
Specialist
Other areas of specialty/work
Dermatology
Street address
The SBMU SRC (Skin Research Center),Shohada-E- Tajrish Educational Hospital,Qods Sq. ,Tehran, Iran
City
Tehran
Province
Tehran
Postal code
1989934148
Phone
+98 21 2274 1507
Email
fabdollahimajd@sbmu.ac.ir

Person responsible for updating data

Contact
Name of organization / entity
Thomas Jefferson University, Dermatology & cutaneous biology
Full name of responsible person
Masoomeh Faghankhani
Position
Postdoc research fellow
Latest degree
Medical doctor
Other areas of specialty/work
Dermatology
Street address
233 S, 10th Street, Suite 450 BLSB.
City
Philadelphia
Province
Pennsylvania
Postal code
PA 19107
Phone
+1 215-503-5785
Email
Masoomeh.Faghankhani@jefferson.edu

Sharing plan

Deidentified Individual Participant Data Set (IPD)
Yes - There is a plan to make this available
Study Protocol
Undecided - It is not yet known if there will be a plan to make this available
Statistical Analysis Plan
Yes - There is a plan to make this available
Informed Consent Form
Undecided - It is not yet known if there will be a plan to make this available
Clinical Study Report
Undecided - It is not yet known if there will be a plan to make this available
Analytic Code
Undecided - It is not yet known if there will be a plan to make this available
Data Dictionary
Undecided - It is not yet known if there will be a plan to make this available
Title and more details about the data/document
It is not yet known if there will be a plan to make this available
When the data will become available and for how long
It is not yet known if there will be a plan to make this available
To whom data/document is available
It is not yet known if there will be a plan to make this available
Under which criteria data/document could be used
It is not yet known if there will be a plan to make this available
From where data/document is obtainable
It is not yet known if there will be a plan to make this available
What processes are involved for a request to access data/document
It is not yet known if there will be a plan to make this available
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