| CTRI Number |
CTRI/2025/12/098353 [Registered on: 03/12/2025] Trial Registered Prospectively |
| Last Modified On: |
02/12/2025 |
| Post Graduate Thesis |
No |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Single Arm Study |
|
Public Title of Study
|
Efficacy and safety of oral sirolimus in vascular malformation |
|
Scientific Title of Study
|
Efficacy and safety of oral sirolimus in vascular malformation: A prospective pilot assessor-blinded interventional study. |
| Trial Acronym |
nil |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| nil |
NIL |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Gomathy Sethuraman |
| Designation |
Professor |
| Affiliation |
MD Dermatology, professor, dept of dermatology, All India Institute of Medical Sciences, New Delhi |
| Address |
Room 4070, Academic block, All India Institute of Medical Sciences, New Delhi, Ansari Nagar, New Delhi, 110029
New Delhi
DELHI
110029
India |
| Phone |
8296529521 |
| Fax |
|
| Email |
aiimsgsr@gmail.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Shreya K |
| Designation |
Senior resident |
| Affiliation |
MD Dermatology, senior resident, dept of dermatology, All India Institute of Medical Sciences, New Delhi |
| Address |
Room 4070, Academic block, All India Institute of Medical Sciences, New Delhi, Ansari Nagar, New Delhi, 110029
New Delhi
DELHI
110029
India |
| Phone |
8296529521 |
| Fax |
|
| Email |
shreyakgowda@gmail.com |
|
Details of Contact Person
Public Query
|
| Name |
Shreya K |
| Designation |
Senior resident |
| Affiliation |
MD Dermatology, senior resident, dept of dermatology, All India Institute of Medical Sciences, New Delhi |
| Address |
Room 4070, Academic block, All India Institute of Medical Sciences, New Delhi, Ansari Nagar, New Delhi, 110029
New Delhi
DELHI
110029
India |
| Phone |
8296529521 |
| Fax |
|
| Email |
shreyakgowda@gmail.com |
|
|
Source of Monetary or Material Support
|
| Monetary support from IADVL academy India
Material support All India Institute of Medical Sciences, New Delhi, Ansari Nagar, New Delhi, 110029 |
|
|
Primary Sponsor
|
| Name |
nil |
| Address |
nil |
| Type of Sponsor |
Other [nil] |
|
|
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 Shreya K |
AIIMS |
Room 308, new rak opd, A wing, All India Institute of Medical Sciences, New Delhi, Ansari Nagar, New Delhi, 110029
New Delhi
DELHI |
8296529521
shreyakgowda@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| AIIMS Delhi |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: L99||Other disorders of skin and subcutaneous tissue in diseases classified elsewhere, (2) ICD-10 Condition: L99||Other disorders of skin and subcutaneous tissue in diseases classified elsewhere, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Comparator Agent |
nil |
nil |
| Intervention |
oral sirolimus |
oral sirolimus 0.8ng/m2 over 1 year |
|
|
Inclusion Criteria
|
| Age From |
1.00 Day(s) |
| Age To |
99.00 Year(s) |
| Gender |
Both |
| Details |
Complex microcystic and macrocystic lymphatic malformations, venolymphatic or mixed malformations and or capillary malformations, vascular malformations associated overgrowth syndrome |
|
| ExclusionCriteria |
| Details |
Concurrent use of drug with cytochrome P450 3A4 inducer such as barbiturates, glucocorticoids, rifampicin, clarithromycin, and erythromycin. Immunosuppressed patient or patients known case of human immunodeficiency virus infection, ateriovenous and capillary malformations |
|
|
Method of Generating Random Sequence
|
Other |
|
Method of Concealment
|
An Open list of random numbers |
|
Blinding/Masking
|
Outcome Assessor Blinded |
|
Primary Outcome
|
| Outcome |
TimePoints |
| to assess the percentage reduction in volume of vascular malformation at the end of 1 year of oral sirolimus |
baseline, 1 month, 3 months, 6 months, 9 months and 12 months |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| To assess time to attain resolution & safety of oral sirolimus in vascular malformation |
baseline, 1 month, 3 months, 6 months, 9 months & 12 months |
|
|
Target Sample Size
|
Total Sample Size="30"
Sample Size from India="30"
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" |
|
Phase of Trial
|
Phase 4 |
|
Date of First Enrollment (India)
|
13/12/2025 |
| Date of Study Completion (India) |
Applicable only for Completed/Terminated trials |
| Date of First Enrollment (Global) |
Date Missing |
| Date of Study Completion (Global) |
Applicable only for Completed/Terminated trials |
|
Estimated Duration of Trial
|
Years="1"
Months="0"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Not Yet Recruiting |
| Recruitment Status of Trial (India) |
Not Yet Recruiting |
|
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
|
Management
of vascular malformation is crucial to prevent the compression of vital organs,
ulceration, and limb length discrepancies. Various complex and interlinking
molecular pathways are involved in the pathogenesis of vascular malformation,
thereby targeted therapy plays a pivotal role in the treatment. Sirolimus also
known as rapamycin is a mTOR inhibitor, blocks the RAS-RAF-MEK and
PIK3CA-AKT-mTOR pathways. Therefore, resulting in apoptosis and inhibition of
vascular pathways. |