| CTRI Number |
CTRI/2024/02/062740 [Registered on: 16/02/2024] Trial Registered Prospectively |
| Last Modified On: |
06/10/2025 |
| Post Graduate Thesis |
Yes |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Single Arm Study |
|
Public Title of Study
|
Tofacitinib in skin calcifications in childhood myositis |
|
Scientific Title of Study
|
Tofacitinib for calcinosis cutis associated with juvenile dermatomyositis: an open label single arm study |
| Trial Acronym |
TOFCAL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| NIL |
NIL |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Dr Narendra Bagri |
| Designation |
Additional Professor ( Chief Guide) |
| Affiliation |
AIIMS New Delhi |
| Address |
Pediatric Rheumatology, Department of Pediatrics
All India Institute of Medical Sciences
New Delhi-110029
New Delhi
DELHI
110029
India |
| Phone |
9013545665 |
| Fax |
|
| Email |
drnarendrabagri@gmail.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Dr Narendra Bagri |
| Designation |
Additional Professor ( Chief Guide) |
| Affiliation |
AIIMS New Delhi |
| Address |
Pediatric Rheumatology, Department of Pediatrics
All India Institute of Medical Sciences
New Delhi-110029
New Delhi
DELHI
110029
India |
| Phone |
9013545665 |
| Fax |
|
| Email |
drnarendrabagri@gmail.com |
|
Details of Contact Person
Public Query
|
| Name |
Dr Pragati Jain |
| Designation |
Junior Resident (Thesis student) |
| Affiliation |
AIIMS New Delhi |
| Address |
Department of Pediatrics
All India Institute of Medical Sciences
New Delhi-110029
New Delhi
DELHI
110029
India |
| Phone |
8839962842 |
| Fax |
|
| Email |
doctorpragati23@gmail.com |
|
|
Source of Monetary or Material Support
|
| All India Institute of Medical Sciences (AIIMS), New Delhi |
|
|
Primary Sponsor
|
| Name |
Department of Pediatrics, AIIMS, New Delhi |
| Address |
Mother Child Block Department of Pediatrics, AIIMS, New Delhi |
| Type of Sponsor |
Research institution and hospital |
|
|
Details of Secondary Sponsor
|
|
|
Countries of Recruitment
|
India |
|
Sites of Study
|
| No of Sites = 1 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Pragati |
AIIMS New Delhi |
Room no.836, 8th floor, Mother Child Block, Division of Rheumatology, Department of Pediatrics
New Delhi
DELHI |
8839962842
doctorpragati23@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| Ethics Committee for Post Graduate Research |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: M330||Juvenile dermatomyositis, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Comparator Agent |
Not Applicable |
Not Applicable |
| Intervention |
Tofacitinib |
Eligible children will be enrolled in the study and will be given oral tofacitinib (intervention) in the recommended doses as per weight band by EMA (European Medicines agency) for 24 ± 2 weeks
10 to 20 kg 3.2 mg BD,
20 to 40 kg 4 mg BD,
above 40 kg 5 mg BD
|
|
|
Inclusion Criteria
|
| Age From |
2.00 Year(s) |
| Age To |
18.00 Year(s) |
| Gender |
Both |
| Details |
1Children age 2 to 18 years with
-Juvenile Dermatomyositis (JDM) OR Juvenile Dermatomyositis Systemic Scleroderma overlap (JDM-SSc) AND
-calcinosis cutis on physical examination or previous imaging. |
|
| ExclusionCriteria |
| Details |
1Patient cyclophosphamide during the preceding 1 month or rituximab in the last 6 months OR
-Topical tacrolimus OR
- Active Tuberculosis OR
- Hemoglobin (Hb) levels less than 9 g per dL OR
- Absolute lymphocyte count less than 500 cells per mm3
-Absolute neutrophil count less than 1000 cells per mm3
|
|
|
Method of Generating Random Sequence
|
Not Applicable |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Not Applicable |
|
Primary Outcome
|
| Outcome |
TimePoints |
| Effect of Tofacitinib on calcinosis cutis in children with JDM as assesed by reduction in Agatston score using ultra low-dose whole body CT scan. |
At baseline and 24 ±2 week follow-up |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
1)Proportion of children demonstrating improvement in clinical scores ( CDASI and CMAS)
2)Number of subjects with adverse events.
3)Change in doses(mean) of steroids
4)Correlation of baseline laboratory parameters ( myositis specific antibody and interferon levels) with outcome. |
1) At baseline and 24 ±2 week follow-up
2)Anytime from initiation of therapy to 24 ±2 weeks after initiation of therapy.
3)Anytime from initiation of therapy to 24 ±2 weeks after initiation of therapy.
4)At baseline and 24 ±2 week follow-up
|
|
|
Target Sample Size
|
Total Sample Size="22"
Sample Size from India="22"
Final Enrollment numbers achieved (Total)= "25"
Final Enrollment numbers achieved (India)="25" |
|
Phase of Trial
|
Phase 3 |
|
Date of First Enrollment (India)
|
20/02/2024 |
| Date of Study Completion (India) |
07/08/2025 |
| Date of First Enrollment (Global) |
Date Missing |
| Date of Study Completion (Global) |
Date Missing |
|
Estimated Duration of Trial
|
Years="2"
Months="0"
Days="0" |
Recruitment Status of Trial (Global)
Modification(s)
|
Not Applicable |
| Recruitment Status of Trial (India) |
Completed |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
Aim - To study the role of tofacitinib in reducing the burden of calcinosis cutis associated with juvenile dermatomyositis over a 6 month follow-up period Primary Objectives -To study the effect of tofacitinib as an adjunct to standard therapy in children 2 to 18 years with JDM/JDM SSc overlap (Annexure 1 and 2) and calcinosis cutis, on the burden of calcinosis cutis as assessed by reduction in Agatston score ( Annexure 5 ) using ultra-low-dose whole-body CT scan at 24 ±2 weeks follow-up compared to baseline. Standard therapy includes Stable doses of steroids (<1mg/kg/day during previous 2 weeks) (if on steroids) and either of Methotrexate (10-15 mg/m2/week) OR MMF (800 to 1000/m2/day) OR HCQ (5mg/kg/day) Secondary Objective- To study the effect of tofacitinib as an adjunct to standard therapy in children (<18 yr) with JDM and calcinosis cutis on disease activity as assessed by proportion of children demonstrating improvement in clinical scores (CMAS and CDASI) at 24±2 weeks follow-up compared to baseline, Assess the number of children experiencing adverse events including infections, anemia, lymphopenia, transaminitis, and renal dysfunction,To assess the ability to taper steroids by analyzing the change in doses (mean) of steroids, To study the association of baseline laboratory parameters with outcomes including Myositis-associated and specific autoantibodies and INF levels (subject to availability of funds) Methodology - Study design - Open label single arm study Study settings -Pediatric Rheumatology Clinic, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi.
Study duration -Feb 2024 – Feb 2025 (enrolment shall start after ethical approval.)
Study population- Children ( 2-18years age ) diagnosed as JDM / JDM SSc overlap and calcinosis cutis coming to Pediatric Rheumatology services in the Department of Pediatrics AIIMS, New Delhi shall be screened for eligibility in the trial.
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