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Efficacy and safety of oral sirolimus in the treatment of pediatric PIK3CA-Related Overgrowth Spectrum (PROS)

Efficacy and safety of oral sirolimus in the treatment of pediatric PIK3CA-Related Overgrowth Spectrum (PROS)

Status
Recruiting
Phases
Phase 4
Study type
Interventional
Source
ChiCTR
Registry ID
ChiCTR2200056185
Enrollment
Unknown
Registered
2022-02-01
Start date
2022-07-01
Completion date
Unknown
Last updated
2024-08-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

PIK3CA-Related Overgrowth Spectrum (PROS)

Interventions

Sponsors

Beijing Children’s Hospital, Capital Medical University (National Center for Children’s Health, China)
Lead Sponsor

Eligibility

Sex/Gender
All
Age
No minimum to 17 Years

Inclusion criteria

Inclusion criteria: 1. Conform to ISSVA (2018) vascular disease diagnostic standard; 2. Gender is not limited; Less than 17 years; 3. Vascular diseases cover a wide range, involving large areas of skin on the body surface and exceeding the subcutaneous fat layer; 4. The risk of tissue and organ dysfunction is high; 5. Stable vital signs; 6. Be able to receive treatment as required; 7. Parents or legal guardians fully understand the test content, voluntarily participate in and sign the informed consent form; 8. The physical examination is qualified, and there are no serious heart, lung, liver, kidney, digestive tract, nervous system, mental disorders, metabolic abnormalities and other diseases in the past.

Exclusion criteria

Exclusion criteria: 1. Blood system diseases; 2. Autoimmune diseases; 3. Liver dysfunction; 4. Decreased renal function; 5. Suffering from serious infection and chronic infectious diseases (such as tuberculosis) in active period; 6. Allergies to macrolides or research drug components or excipients; 7. Patients who participated in any other drug clinical study three months before the start of the trial; 8. Any systemic drugs for vascular malformations were used within 4 weeks before the baseline visit.

Design outcomes

Primary

MeasureTime frame
Effective rate;

Countries

China

Contacts

Public ContactBin Zhang

Beijing Children’s Hospital, Capital Medical University (National Center for Children’s Health, China)

binzhang@163.com+86 13910007494

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 4, 2026