{"type":"trials","record":{"id":3145,"source_id":"clinicaltrials-gov","external_id":"NCT07820579","title":"Sirolimus Treatment in Pediatric Patients With Ataxia-Telangiectasia","brief_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.","overall_status":"Completed","phases":["PHASE2"],"study_type":"INTERVENTIONAL","sponsor":"Sara Elva Espinosa Padilla, MD","enrollment":6,"countries":["Mexico"],"start_date":"2024-07-11","completion_date":"2026-08-20","last_update_date":"2026-09-15","source_url":"https://clinicaltrials.gov/study/NCT07820579","editorial_summary":"A registered PHASE2 study indexed because it matched monitored longevity research terms. Registry status: Completed. Registration does not establish safety or effectiveness.","first_seen_at":"2026-09-15T12:17:11.225469+00:00","last_seen_at":"2026-09-25T06:05:19.382+00:00","metadata":{"sex":"ALL","acronym":null,"age_range":"16 Years","comparator":null,"organization":"National Institute of Pediatrics, Mexico","interventions":["Rapamune (sirolimus/rapamycin)"],"registry_source":"ClinicalTrials.gov","outcome_measures":["Adverse effects and laboratory safety after treatment — From enrollment to the end of treatment at 6 months","Neurological function assessed by the Scale for the Assessment and Rating of Ataxia — From enrollment to the end of tre…","Immunological and laboratory evaluation — From enrollment to the end of treatment at 6 months","Percentage of conjunctival vascular area — From enrollment to the end of treatment at 6 months","Image Analysis — From enrollment to the end of treatment at 6 months","Cognitive performance assessed by the Cerebellar Cognitive Affective Syndrome Scale — From enrollment to the end of tre…","Executive functioning assessed by the Behavior Rating Inventory of Executive Function, Second Edition — From enrollment…","Emotional and behavioral functioning assessed by the Child Behavior Checklist — From enrollment to the end of treatment…"],"design_description":"NA · SINGLE GROUP · TREATMENT · NONE","source_has_results":false},"controlled_terms":["Ataxia-Telangiectasia (A-T)","Ataxia-Telangiectasia","Rapamune (sirolimus/rapamycin)"],"relevance_confidence":100,"source_quality_score":100,"freshness_score":100,"publication_state":"published","match_explanation":"Title contains controlled term: sirolimus.","quality_checked_at":"2026-09-25T06:05:19.924533+00:00","duplicate_cluster_key":"id:nct07820579","duplicate_of_id":null,"evidence_snapshot":{"status":"structured","version":1,"duration":"2024-07-11 to 2026-08-20","comparator":null,"confidence":"structured-source","population":"ALL · 16 Years","provenance":{"duration":"start_date and completion_date","comparator":"registry arm fields","population":"registry eligibility fields","intervention":"registry intervention fields","participants":"enrollment","study_design":"study_type and registry design fields","evidence_stage":"phases","reported_outcome":"not available","outcomes_measured":"registry outcome-measure fields"},"generated_at":"2026-09-25T06:05:19.382Z","intervention":["Rapamune (sirolimus/rapamycin)"],"participants":6,"study_design":"NA · SINGLE GROUP · TREATMENT · NONE","subject_scope":"Human clinical study registration","evidence_stage":"PHASE2","safety_context":"Eligibility, adverse-event details, and clinical decisions must be checked in the official registry and with qualified clinicians.","source_support":"Structured registry protocol metadata; no finding-level conclusion is generated.","main_limitation":"This is a study registration. No reusable structured result is available here, so it cannot show whether the intervention worked or was safe.","reported_outcome":null,"outcomes_measured":["Adverse effects and laboratory safety after treatment — From enrollment to the end of treatment at 6 months","Neurological function assessed by the Scale for the Assessment and Rating of Ataxia — From enrollment to the end of tre…","Immunological and laboratory evaluation — From enrollment to the end of treatment at 6 months","Percentage of conjunctival vascular area — From enrollment to the end of treatment at 6 months","Image Analysis — From enrollment to the end of treatment at 6 months","Cognitive performance assessed by the Cerebellar Cognitive Affective Syndrome Scale — From enrollment to the end of tre…","Executive functioning assessed by the Behavior Rating Inventory of Executive Function, Second Edition — From enrollment…","Emotional and behavioral functioning assessed by the Child Behavior Checklist — From enrollment to the end of treatment…"],"regulatory_context":"Trial registration is not regulatory approval and does not establish that an intervention is available."},"clinical_trial_topics":[{"topic_slug":"rapamycin","is_published":true,"match_reasons":["Title contains controlled term: sirolimus.","Abstract contains controlled term: sirolimus.","Source terminology contains: rapamycin, sirolimus.","Abstract supplies longevity context: aging.","Study type is explicitly identified as INTERVENTIONAL."],"matched_fields":["title","abstract","controlled terminology","abstract context","study type"],"relevance_score":100,"intelligence_topics":{"name":"Rapamycin","slug":"rapamycin"}}],"content_sources":{"name":"ClinicalTrials.gov","homepage_url":"https://clinicaltrials.gov/"}},"canonical_url":"https://www.immortal.life/trials/3145","automation_disclosure":"Generated automatically from cited source metadata. No scientist, clinician, researcher, editor, or human reviewer evaluates this publication before release."}