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Sirolimus Treatment in Pediatric Patients With Ataxia-Telangiectasia
Sponsor: Sara Elva Espinosa Padilla, MD
Summary
Ataxia-telangiectasia (A-T) is a progressive disease which is, to date, incurable, affecting motor skills, and a high cancer and recurrent infections risk. Although there has been great progress in recent years in understanding its pathophysiology, we have little information that produces any benefit for its treatment. Thus, Sirolimus has a wide variety of potential mechanisms of action that could be beneficial in A-T, including neuroprotective effects, antiangiogenic, anti-infectious, antineoplastic, and aging slower. With that aim, we conducted a pilot study in our hospital evaluating the safety and potential benefit of sirolimus in the disease.
Official title: Immunological, Dermatological, and Neurological Effects of Sirolimus in Ataxia-Telangiectasia
Key Details
Gender
All
Age Range
Any - 16 Years
Study Type
INTERVENTIONAL
Enrollment
6
Start Date
2024-07-11
Completion Date
2026-08-20
Last Updated
2026-09-15
Healthy Volunteers
No
Interventions
Rapamune (sirolimus/rapamycin)
After baseline evaluation, oral sirolimus was initiated at a dose of 1 mg/m²/day divided into two daily doses. Treatment duration was 6 months, safety was assessed throughout the trial.
Locations (1)
Instituto Nacional de Pediatría IRB
Mexico City, Mexico City, Mexico