Systematic Approach for Children With Orbital Malformation With Sirolimus Use

NCT07378657 | Recruiting Phase 3 DMC

• 1 other identifier: FMASU R 271/2025
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this interventional study is to Set a systematic management approach for orbital lymphatic malformation and to study the safety and efficacy of sirolimus in children and adolescents with orbital lymphatic malformation\]. The main questions it aims to answer are: \[what is the safety of using sirolimus in patients with orbital lymphatic malformation\]? \[what is the efficacy of sirolimus in patients with orbital lymphatic malformation\]? Participants will be divided into three groups based on their symptoms, extent and type of orbital lymphatic malformation Group 1 ( will receive injection sclerotherapy): All Patients with symptomatic lymphatic malformation except those with complex malformation , Group 2 (will receive sirolimus on 0.8 mg per m2 twice daily for 6 months ):A) Patients with symptomatic complex lymphatic malformation. B) Patients in group 1 if there is progression in lesion size or ineffective local therapy Group 3 ( will receive both injection sclerotherapy and sirolimus):A) Patients in group 1 and patients with complex lymphatic malformation if there is concern for complications like hemorrhage, amblyopia and vision loss since diagnosis.

Conditions

Orbital Lymphatic Malformation

Keywords

sirolimus; orbital lymphatic malformation

Outcome Measures

Primary Outcomes (1)

• safety of sirolimus in patients with orbital lymphatic malformation

  assessment of safety of sirolimus

  6 months

Secondary Outcomes (1)

• efficacy of sirolimus in patients with orbital malformation

  6 months

Study Arms (3)

Group 1: All Patients with symptomatic LM except complex type

ACTIVE COMPARATOR

Group 1: All Patients with symptomatic lymphatic malformation except those with complex lymphatic malformation.They will be assigned to sclerotherpay .Local intralesional trancutaneous injection dose will be 0.6 mg / kg of bleomycin dissolved in the total volume of the lesion calculated from dimensions of orbital lesion in MRI orbit with contrast. Intralesional injection frequency will be guided by clinical response of the patients and imaging studies.If there is response clinically, injection will be continued every one month until there is no further response or if the lesion is reaching 75% of lesion size Or if there is no response from first injection.For lid and conjunctival components local injection will be for small , moderate and large lesions 2 IU, 4 IU, 6 IU respectively and further injection every month guided by lesion response.Less than 25% reduction will not receive further injection, if there is 25%- 50 % reduction injection will continue till reaching 75%

Procedure: Injection Sclerotherapy

Group 2: Patients with complex LM or patients from group 1 if no response

ACTIVE COMPARATOR

Patients assigned to sirolimus arm will be patients with A) Patients with symptomatic complex lymphatic malformation. B) Patients in group 1 if there is progression in lesion size or ineffective local therapy. Dose will be 0.8 mg/m2 twice daily orally for 6 months ,published literature for sirolimus' use in the pediatric population recommends a starting dose of sirolimus 1.6 mg/m2/day divided twice daily (10) with target trough level of 8-15 ng/ml will be done after 2 weeks from starting therapy

Drug: Sirolimus (Rapamune®)

Group 3: A) Patients in group 1 or complex if there is concern for complications like hemorrhage, am

ACTIVE COMPARATOR

Dose will be 0.8 mg/m2 twice daily orally for 6 months ,published literature for sirolimus' use in the pediatric population recommends a starting dose of sirolimus 1.6 mg/m2/day divided twice daily (10) with target trough level of 8-15 ng/ml will be done after 2 weeks from starting therapy

Other: both injection sclerotherapy and sirolimus

Interventions

Injection Sclerotherapy PROCEDURE

For group 1: They will be assigned to sclerotherpay .Local intralesional trancutaneous injection dose will be 0.6 mg / kg of bleomycin dissolved in the total volume of the lesion calculated from dimensions of orbital lesion in MRI orbit with contrast. Intralesional injection frequency will be guided by clinical response of the patients and imaging studies.If there is response clinically, injection will be continued every one month until there is no further response or if the lesion is reaching 75% of lesion size Or if there is no response from first injection.For lid and conjunctival components local injection will be for small , moderate and large lesions 2 IU, 4 IU, 6 IU respectively and further injection every month guided by lesion response.Less than 25% reduction will not receive further injection, if there is 25%- 50 % reduction injection will continue till reaching 75% or more clinical reduction in size

Group 1: All Patients with symptomatic LM except complex type

Sirolimus (Rapamune®) DRUG

Dose will be 0.8 mg/m2 twice daily orally for 6 months ,published literature for sirolimus' use in the pediatric population recommends a starting dose of sirolimus 1.6 mg/m2/day divided twice daily (10) with target trough level of 8-15 ng/ml will be done after 2 weeks from starting therapy

Group 2: Patients with complex LM or patients from group 1 if no response

both injection sclerotherapy and sirolimus OTHER

They will be assigned to sclerotherpay .Local intralesional trancutaneous injection dose will be 0.6 mg / kg of bleomycin dissolved in the total volume of the lesion calculated from dimensions of orbital lesion in MRI orbit with contrast. Intralesional injection frequency will be guided by clinical response of the patients and imaging studies.If there is response clinically, injection will be continued every one month until there is no further response or if the lesion is reaching 75% of lesion size Or if there is no response from first injection.For lid and conjunctival components local injection will be for small , moderate and large lesions 2 IU, 4 IU, 6 IU respectively and further injection every month guided by lesion response.Less than 25% reduction will not receive further injection, if there is 25%- 50 % reduction injection will continue till reaching 75% or more clinical reduction in size sirolimus will be given in a dose of 0.8 mg per m2 twice daily .

Group 3: A) Patients in group 1 or complex if there is concern for complications like hemorrhage, am

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age : 1-18 years Children and adolescents diagnosed with orbital low flow vascular malformation based on clinical , radilogical findings

You may not qualify if:

• Children and adolescents with orbital high flow vascular malformation

Sponsors & Collaborators

• Ain Shams University: lead

Study Sites (1)

Ain Shams University

Cairo, Cairo Governorate, 11321, Egypt

RECRUITING

MeSH Terms

Interventions

Sirolimus

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Central Study Contacts

nihal hussien aly mostafa

CONTACT

+201001053961; nihal.hussien.ali@gmail.com

iman ahmed ragab

CONTACT

+201002550680; hragab68@hotmail.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: associate professor of pediatrics -pediatric hematology and oncology unit

Study Record Dates

• First Submitted: January 23, 2026
• First Posted: January 30, 2026
• Study Start: November 1, 2025
• Primary Completion (Estimated): November 1, 2027
• Study Completion (Estimated): November 1, 2027
• Last Updated: January 30, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

EG (1)