[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07125066":3,"trial-entities:NCT07125066":84,"trial-summary:NCT07125066":88},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":22,"primary_purpose":23,"phases":24,"enrollment_info":26,"interventions":29,"primary_outcomes":38,"secondary_outcomes":42,"sex":51,"minimum_age":52,"maximum_age":52,"healthy_volunteers":15,"eligibility_criteria":53,"std_ages":62,"locations":66,"central_contacts":76,"overall_officials":77,"references":82,"see_also_links":83},"NCT07125066","STUDY25060137","An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)","An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2-hydroxyglutaric Aciduria (C-2HGA)","ENROLLING_BY_INVITATION","2027-12","2026-06","2026-06-08","2025-07-30","Jerry Vockley, MD, PhD","OTHER",false,"This is an individual patient research protocol to treat a patient diagnosed with Combined D,L-2-hydroxyglutaric aciduria (C-2HGA) with ACER-001.","Patient will continue to be seen during routine outpatient clinic visits every 6 months. At those visits the following will be collected: routine metabolic laboratory tests (including a complete metabolic profile, complete blood count, blood phenylacetylglutamine level, and urine organic acids), physical examinations, and an update of clinical history, especially regarding seizures and respiratory status.\n\nOnce consent is signed, the patient will continue to take ACER-001 as prescribed. The ACER-001 will be provided to the patient at no cost.",[19],"Combined D,L-2-hydroxyglutaric Aciduria",[21],"Combined D,L-2-hydroxyglutaric aciduria","INTERVENTIONAL","TREATMENT",[25],"PHASE1",{"count":27,"type":28},1,"ESTIMATED",[30],{"type":31,"name":32,"description":33,"armGroupLabels":34,"otherNames":36},"DRUG","Sodium phenylbutyrate","open-label design with doses of sodium phenylbutyrate",[35],"sodium phenylbutyrate",[37],"Olpruva",[39],{"measure":40,"timeFrame":41},"Number of treatment related adverse events","2 years",[43,45,47,49],{"measure":44,"timeFrame":41},"Change in the amount of urine D,L 2-Hydroxyglutaric acid in urine",{"measure":46,"timeFrame":41},"Change in phenylacetylglutamine in urine",{"measure":48,"timeFrame":41},"Change in the number of seizures reported by parents",{"measure":50,"timeFrame":41},"Change in ventilator support","MALE",null,{"inclusion":54,"exclusion":58,"raw_text":61},[55,56,57],"Single patient with Combined D,L-2-hydroxyglutaric aciduria","Parental permission must be given at the start of the protocol.","Parents willing to adhere to the protocol",[59,60],"Refusal or withdrawal of consent.","Drug Intolerance.","Inclusion Criteria:\n\n* Single patient with Combined D,L-2-hydroxyglutaric aciduria\n* Parental permission must be given at the start of the protocol.\n* Parents willing to adhere to the protocol\n\nExclusion Criteria:\n\n* Refusal or withdrawal of consent.\n* Drug Intolerance.",[63,64,65],"CHILD","ADULT","OLDER_ADULT",[67],{"facility":68,"city":69,"state":70,"zip":71,"country":72,"geoPoint":73},"Children's Hospital of Pittsburgh of UPMC","Pittsburgh","Pennsylvania","15224","United States",{"lat":74,"lon":75},40.44062,-79.99589,[],[78],{"name":79,"affiliation":80,"role":81},"Gerard Vockley, MD, PhD","UPMC Children's Hospital of Pittsburgh","PRINCIPAL_INVESTIGATOR",[],[],{"nct_id":4,"conditions":85,"biomarkers":87},[86],"Combined D-2-hydroxyglutaric aciduria and L-2-hydroxyglutaric aciduria",[],{"nct_id":4,"found":15,"summary":52,"prompt_version":52}]