Investigation of the safety and efficacy of rituximab drug on patients with dermatomyositis in young people
Design
Clinical trial, without control group, single trial, non-blinded, phase 2 on 7 patients
Settings and conduct
The current research will be carried out on children with dermatomyositis referred to Mofid Children's Hospital. Upon entering the study, after receiving the demographic and basic information of the patients, the disease activity status before the intervention (Time 0) will be evaluated by the JDM-DAS tool. Then, patients will be treated with the appropriate dose of rituximab based on their Body Surface Area (BSA).
Participants/Inclusion and exclusion criteria
Conditions for entry: children under the age of 16 with definitive diagnosis of juvenile dermatomyositis based on criteria and have visited Mofid Children's Hospital within the study period.
Exclusion criteria: Non-compliance and non-attendance of patients; contraindication for rituximab administration; presence of concomitant diseases and overlap syndrome.
Intervention groups
Children with juvenile dermatomyositis referred to Mofid Children's Hospital, based on the patient's body surface area, are treated with an appropriate dose of rituximab with a time interval (0 and 1 week), with a dose of 575 mg/m2 for patients with a body surface area less than or equal to with 1.5 and a dose of 750 mg/m2 for patients whose body surface area is greater than 1.5. Patients are visited regularly for a period of six months, and a disease activity assessment form (both doctor's and patient's prescriptions) is completed at each visit. In addition, patients and their caregivers are asked about any side effects and effects of treatment.
Main outcome variables
Disease Activity status (physician's score and patient 's score); Improvement of clinical and laboratory symptoms of the patient; Drug Side Effects
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20231119060108N1
Registration date:2024-01-15, 1402/10/25
Registration timing:prospective
Last update:2024-01-15, 1402/10/25
Update count:0
Registration date
2024-01-15, 1402/10/25
Registrant information
Name
Pooneh Tabibi
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 21 2222 7021
Email address
tabibi_p@yahoo.com
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2024-01-21, 1402/11/01
Expected recruitment end date
2024-05-21, 1403/03/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Safety and Efficacy of Rituximab in patients with juvenile dermatomyositis (JDM) who were admitted at Mofid children hospital
Public title
Safety and Efficacy of Rituximab in patients with juvenile dermatomyositis
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Patient under 16 years old
Patients with the diagnosis of dermatomyositis, which is confirmed based on the diagnosis criteria
Exclusion criteria:
Presence of concomitant diseases and overlap syndrome
Contraindications for rituximab injection
Age
To 16 years old
Gender
Both
Phase
2
Groups that have been masked
No information
Sample size
Target sample size:
7
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Single
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics Committee of Shahid Beheshti University of Medical Sciences
Street address
3rd floor, Faculty of Medicine, next to Taleghani Hospital, Evin, Shahid Chamran Highway
City
Tehran
Province
Tehran
Postal code
1983963113
Approval date
2023-12-26, 1402/10/05
Ethics committee reference number
IR.SBMU.MSP.REC.1402.500
Health conditions studied
1
Description of health condition studied
Juvenile dermatomyositis
ICD-10 code
M33.0
ICD-10 code description
Juvenile dermatomyositis
Primary outcomes
1
Description
Disease activity
Timepoint
At the beginning of the intervention (before the start of the intervention), one week, one month, two months, three months, six months and one year after rituximab injection.
Method of measurement
Juvenile Dermatomyositis-Disease Activity Score questionnaire, questionnaire of characteristics and information of dermatomyositis patients
2
Description
Drug Side Effects
Timepoint
One week, one month, two months, three months, six months and one year after rituximab injection.
Method of measurement
Questioning the patient and parents
Secondary outcomes
1
Description
Daily activity and quality of life
Timepoint
Before starting the intervention, one month, two, three months, six months and one year after drug injection
Method of measurement
Juvenile Dermatomyositis-Disease Activity Score questionnaire, questionnaire of characteristics and information of dermatomyositis
Intervention groups
1
Description
Intervention group: Patients based on Body Surface Area (BSA) treated with the appropriate dose of rituximab (575 mg/m2 in patients with BSA ≤1.5 m2 and 750 mg/m2 in patients with BSA>1.5 m2), Two doses one week apart, with the Zitox brand (company Ariogen Pharmed) will be placed
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Mofid children hospital
Full name of responsible person
Pooneh Tabibi
Street address
Mofid Children's Hospital, Shariati Street, Tehran
City
Tehran
Province
Tehran
Postal code
1546815514
Phone
+98 21 2222 7021
Email
tabibi_p@yahoo.com
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Shahid Beheshti University of Medical Sciences
Full name of responsible person
Dr Afshin Zarghi
Street address
Shahid Beheshti University of Medical Sciences, Research and Technology, student bouleard, Velanjak, Tehran
City
Tehran
Province
Tehran
Postal code
1983963113
Phone
+98 21 2387 2206
Email
urm@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
100
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
Pooneh Tabibi
Position
Assistant specialist in pediatric rheumatology
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Mofid Children's Hospital, Shariati Street, Tehran
City
Tehran
Province
Tehran
Postal code
1546815514
Phone
+98 21 2222 7021
Fax
Email
Tabibi_p@yahoo.com
Person responsible for scientific inquiries
Contact
Name of organization / entity
Shahid Beheshti University of Medical Sciences
Full name of responsible person
Pooneh Tabibi
Position
Assistant specialist in pediatric rheumatology
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Mofid Children's Hospital, Shariati Street, Tehran
City
Tehran
Province
Tehran
Postal code
1546815514
Phone
+98 21 2222 7021
Fax
Email
Tabibi_p@yahoo.com
Person responsible for updating data
Contact
Name of organization / entity
Shahid Beheshti University of Medical Sciences
Full name of responsible person
Pooneh Tabibi
Position
Assistant specialist in pediatric rheumatology
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Mofid Children's Hospital, Shariati Street, Tehran
City
Tehran
Province
Tehran
Postal code
1546815514
Phone
+98 21 2222 7021
Fax
Email
Tabibi_p@yahoo.com
Sharing plan
Deidentified Individual Participant Data Set (IPD)
Yes - There is a plan to make this available
Study Protocol
No - There is not a plan to make this available
Statistical Analysis Plan
No - There is not a plan to make this available
Informed Consent Form
Yes - There is a plan to make this available
Clinical Study Report
Yes - There is a plan to make this available
Analytic Code
No - There is not a plan to make this available
Data Dictionary
No - There is not a plan to make this available
Title and more details about the data/document
In the current study, after completing the study, all potential data will be shared with other researchers through the Research Gate data sharing system after de-identifying the patients.
When the data will become available and for how long
Access will start from the time the results are printed.
To whom data/document is available
The data will be publicly available to all interested parties through the link created on the researcher's Research Gate page.
Under which criteria data/document could be used
The use of research data for future studies by referring to the original published article of the current research will be unimpeded.
From where data/document is obtainable
The data will be transparently accessible through the link included in the final article.
What processes are involved for a request to access data/document
The data will be transparently accessible through the link included in the final article.