| CTRI Number |
CTRI/2024/10/075019 [Registered on: 09/10/2024] Trial Registered Prospectively |
| Last Modified On: |
09/10/2024 |
| Post Graduate Thesis |
Yes |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Single Arm Study |
|
Public Title of Study
|
Effectiveness of the drug Sirolimus in treatment of vascular anomalies. |
|
Scientific Title of Study
|
Efficacy and Safety of Sirolimus in the Management of pediatric Vascular Tumors and Malformations- A Prospective 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 |
Dr Saurav Sharma |
| Designation |
Senior Resident Doctor, DM Pediatric Oncology |
| Affiliation |
AIIMS, New Delhi |
| Address |
Mother and child block, Division of pediatric oncology, Department of Pediatrics, AIIMS Delhi, Sri Aurobindo Marg, Ansari Nagar, New Delhi
New Delhi
DELHI
110029
India |
| Phone |
7726981145 |
| Fax |
|
| Email |
sssauravsharma.787@gmail.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Dr Jagdish Prasad Meena |
| Designation |
Additional Professor |
| Affiliation |
AIIMS, New Delhi |
| Address |
Room no. 832, 8th Floor, Mother and Child Block, Division of pediatric oncology, Department of Pediatrics, AIIMS, New Delhi
New Delhi
DELHI
110029
India |
| Phone |
8890139358 |
| Fax |
|
| Email |
drjpmeena@gmail.com |
|
Details of Contact Person
Public Query
|
| Name |
Dr Jagdish Prasad Meena |
| Designation |
Additional Professor |
| Affiliation |
AIIMS, New Delhi |
| Address |
Room no. 832, 8th Floor, Mother and Child Block, Division of pediatric oncology, Department of Pediatrics, AIIMS, New Delhi
New Delhi
DELHI
110029
India |
| Phone |
8890139358 |
| Fax |
|
| Email |
drjpmeena@gmail.com |
|
|
Source of Monetary or Material Support
|
| Department of Pediatrics, AIIMS NEW DELHI, Ansari Nagar, New Delhi, India. Pin-110029. |
|
|
Primary Sponsor
|
| Name |
Dr Saurav Sharma(self) |
| Address |
Senior Resident(DM), Division of Paediatric Oncology, Department of Paediatrics, Mother and child block, AIIMS, ANSARI NAGAR, NEW DELHI- 110029 |
| Type of Sponsor |
Other [self] |
|
|
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 Saurav Sharma |
AIIMS New Delhi |
Mother and Child block, Division of Pediatric Oncology, Department of Pediatrics, AIIMS New Delhi, Sri Aurobindo Marg, Ansari Nagar East, New Delhi, India. Pin-110029.
New Delhi
DELHI |
7726981145
sssauravsharma.787@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| Institute of Ethics committee, AIIMS New Delhi |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: C00-D49||Neoplasms, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Comparator Agent |
Not applicable |
Not applicable |
| Intervention |
Sirolimus administration |
Sirolimus will be given at a dose of 0.8 mg/m² twice daily to each patient. Maximum dose is set as 5 mg/dose. The first dose of the drug will be supervised by the PI. If dose is not concurrent with 1 tab/ half tab, then tablet will be crushed and disintegrated in 10 ml water immediately prior to administration and the required dose will be administered. If patients vomits within 20 mins of administration, the dose of drug will be repeated. Patient/ guardian will be explained on how to take the subsequent doses. Total duration would be 6 months of administration. |
|
|
Inclusion Criteria
|
| Age From |
1.00 Day(s) |
| Age To |
18.00 Year(s) |
| Gender |
Both |
| Details |
1. Children aged ≤18 years diagnosed with Newly diagnosed vascular malformation.
2.Patients with benign vascular tumors who are refractory to 3 months of conventional therapy.
3.Patients with locally aggressive vascular tumors
4.Written informed consent
|
|
| ExclusionCriteria |
| Details |
1.Children less than 1 year of age with infantile hemangioma.
2.Known hypersensitivity to sirolimus
3.Concurrent nephrotoxic agent use
4.CYP3A4 inducers or inhibitors while receiving sirolimus
5.Baseline cytopenias (ANC less than 500/mm3, Hb less than 8 gm/dl, and platelet less than 50,000/mm3).
6.Baseline SGOT/SGPT more than 5 times ULN or serum bilirubin more than 1.5 times ULN
7.Abnormal lipid profiles: triglycerides (TG) more than 130mg/dl, cholesterol more than 170 mg/dl
|
|
|
Method of Generating Random Sequence
|
Not Applicable |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Not Applicable |
|
Primary Outcome
|
| Outcome |
TimePoints |
| Reduction in size of vascular anomaly |
6 month |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| Improvement in clinical signs and symptoms of vascular mnalformation. |
3 month |
| Improvement in quality of life of the patients. |
3 month |
| Evaluation of side effect of the Sirolimus during the treatment. |
3 month |
| Distribution of types of vascular anomaly. |
at the end of study. |
|
|
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 2 |
|
Date of First Enrollment (India)
|
31/10/2024 |
| 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="2"
Months="0"
Days="0" |
|
Recruitment Status of Trial (Global)
|
Not Applicable |
| 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 - YES
- What data in particular will be shared?
Response - Individual participant data that underlie the results reported in this article, after de-identification (text, tables, figures, and appendices).
- What additional supporting information will be shared?
Response - Study Protocol
Response - Statistical Analysis Plan
Response - Informed Consent Form
Response - Clinical Study Report
- Who will be able to view these files?
Response - Researchers whose proposed use of the data has been approved by an independent review committee identified for this purpose.
- For what types of analyses will this data be available?
Response - To achieve aims in the approved proposal.
- By what mechanism will data be made available?
Response - Proposals should be directed to [drjpmeena@gmail.com].
- For how long will this data be available start date provided 01-07-2027 and end date provided 01-07-2032?
Response - Beginning 3 months and ending 5 years following article publication.
- Any URL or additional information regarding plan/policy for sharing IPD?
Additional Information - NIL
|
|
Brief Summary
|
Vascular anomalies represent a broad spectrum of disorders ranging from a tiny “birthmark†to life threatening entities. They are difficult to treat clinical entities causing significant morbidity and functional impairment. Currently available treatment modalities like sclerotherapy/ embolization/ surgical excision are seldom curative with high recurrence rates. Medical management can be a way forward in this regard. Sirolimus has shown promising results in the past studies as an effective drug in management of vascular anomalies leading to decrease in size and improvement in functional status of the patients. Thus our research hypothesis is that the use of sirolimus will improve clinical symptoms, reduce the size, and improve the quality of life in symptomatic patients with vascular anomalies in 70% of patients after 3 months of use. This will be a single arm trial to be conducted at Department of paediatrics, AIIMS Delhi for a period of 2 years from july 2024 to july 2026. The primary objective will be to assesss the efficacy of Sirolimus in decreasing the size of vascular anomalies. The secondary objectives will be to assess the efficacy of sirolimus in decreasing the signs and symptoms of vascular anomalies, in improving the quality of life of the patients; and to assess its safety during the treatment. Additionally, data regarding the distribution of types of vascular anomalies will be recorded. |