[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"UCB BIOSCIENCES, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":62},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,44],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100631650","phase-3-a-phase-3-study-of-fenfluramine-hydrochloride-in-rett-syndrome-100631650",false,"NCT07503444","A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome","A Phase 3 Randomized, Double-Blind, Placebo Controlled, Parallel Group, Multicenter Study With Open-Label Extension to Evaluate the Efficacy And Safety of Fenfluramine Hydrochloride in Study Participants With Rett Syndrome","Inclusion Criteria:\n\n* Participant has typical or classic Rett Syndrome (RTT) according to the RettSearch Consortium 2010 revised criteria\n* Participant has a documented disease-causing mutation in the methyl-CpG-binding protein 2 (MECP2) gene\n* Participant meets criteria for postregression for at least 6 months prior to Screening, defined as:\n* No loss or degradation of ambulation (including gait, coordination, or independence of walking\u002Fstanding);\n* No loss or degradation of hand function; no loss or degradation of speech (including babbling, words, or previously developed communicative vocalizations);\n* No loss or degradation of nonverbal communicative or social skills (including eye gaze, using body to indicate communicative intent, or social attentiveness)\n* Participant has an Rett Syndrome Clinical Severity Scale (RTT-CSS) rating of 10 to 36 (inclusive)\n* Participant has a Clinical Global Impression-Severity (CGIS) score of ≥4\n* Participant has a legal representative capable of providing signed informed consent on behalf of the participant as described in the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.\n* Participant is aged 5 to 35 years of age (inclusive) at the time of first administration of investigational intervention.\n* Male or female.\n* Participant has a consistent caregiver who is ≥18 years of age at the Screening Visit. The caregiver needs to be able to complete the caregiver assessments defined for the entire study. Every attempt should be made to have the same evaluator complete the assessments for the duration of the study.\n\nExclusion Criteria:\n\n* Participant has a history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.\n* Participant has clinically significant abnormality in vital signs according to the Investigator\n* Participant has an exclusionary cardiovascular or cardiopulmonary abnormality based on echocardiogram (ECHO), electrocardiogram (ECG), or physical examination, and is not approved for entry by the central cardiac reader. Exclusionary abnormalities include, but are not limited to:\n\n  1. Greater than trace aortic valve regurgitation.\n  2. Greater than mild mitral valve regurgitation.\n  3. Possible signs of pulmonary arterial hypertension (PAH) with abnormal pulmonary artery systolic pressure (PASP) or PASP ≥35 mmHg.\n  4. Evidence of left ventricular dysfunction (systolic or diastolic).\n  5. Clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, or patent ductus arteriosus with reversal of shunt (right to left shunt). Note: Patent foramen ovale without a reversal of shunt or a bicuspid aortic valve is not considered exclusionary\n* Participant has a clinically significant medical condition, including chronic obstructive pulmonary disease, interstitial lung disease, portal hypertension, or need for invasive mechanical ventilation (eg, via tracheostomy), or has had clinically relevant symptoms or a clinically significant illness currently or in the 4 weeks prior to the Screening Visit that would negatively impact study participation, collection of study data, or pose a risk to the participant\n* Participant is taking \\>4 concomitant antiseizure medications (ASMs). Rescue medications are not included in the count","ALL","5 Years","35 Years",{"count":20,"type":21},200,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).",[27],"Rett Syndrome",[29,30],"Fenfluramine HCl","RTT","NOT_YET_RECRUITING","2026-06-25",{"date":34,"type":35},"2026-06-26","ACTUAL",{"date":37,"type":21},"2026-06-30",{"date":39,"type":21},"2030-11-15",{"name":41,"class":42},"UCB BIOSCIENCES, Inc.","INDUSTRY",9,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":50,"phases":4,"briefSummary":51,"conditions":52,"keywords":54,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":61,"locationsCount":4},"100561463","doxecitine-and-doxribtimine-expanded-access-100561463","NCT06590493","Doxecitine and Doxribtimine-Expanded Access","Inclusion Criteria:\n\nTK0113:\n\n* Pediatric and adult patients with a diagnosis of TK2d based on confirmed reportable variant(s) in the TK2 gene in countries where UCB has an affiliate\u002Flocal safety officer Signs and symptoms compatible with TK2d disease\n* Risk of major disability or death resulting from TK2d\n* The patient must be willing to receive treatment with doxecitine and doxribtimine via this program, which includes signing an authorization form for sharing genetic test results, medical data and other related information with UCB, its third-party agents and health authorities\n* The patient\u002Flegal guardian or representative has given informed consent (and age-appropriate assent) to treatment prior to administering doxecitine and doxribtimine in a manner consistent with all national requirements. This also includes consent for the transmission of a copy of the anonymized data, such as serious adverse event (SAE) and pregnancy reports (in compliance with local regulatory authority requirements) to UCB third-party agents where allowable by local regulations and to the country regulatory authority as required.\n* The patient does not qualify for or is unable to participate in a UCB sponsored ongoing clinical trial evaluating doxecitine and doxribtimine.\n\nTK0115:\n\n* Diagnosis of TK2d based on confirmed reportable variant(s) in the TK2 gene\n* Age of TK2d symptom onset ≤ 12 years\n* The patient or care provider must be willing to receive treatment with doxecitine and doxribtimine via this program\n* Patient has consented to the contraception, pregnancy, and lactation requirements where relevant\n* The patient does not qualify for or is unable to participate in a UCB-sponsored clinical trial evaluating doxecitine and doxribtimine for the treatment of patients with TK2d\n\nExclusion Criteria:\n\nTK0113:\n\n* Confirmed diagnosis of other genetic or polygenic disease likely to confound clinical presentation of TK2 deficiency\n* Patient has a hypersensitivity to any of the excipients in doxecitine and doxribtimine\n* Inability to tolerate oral or gastric tube administration of doxecitine and doxribtimine\n* History of liver disease, or liver function test results (alanine aminotransferase\\[ALT\\], aspartate transaminase \\[AST\\], or total bilirubin) ≥3× upper limit of normal at Screening without prior Sponsor approval\n* Patients with elevated transaminases (ALT, AST \\> 3xupper limit of normal) without increase in bilirubin should be further evaluated to exclude other causes of liver injury and may be enrolled with UCB approval or may be rescreened\n* Renal insufficiency requiring dialysis\n* Discontinuation of prior nucleos(t)ide treatment for TK2d because of adverse event(s)\n* Any other concurrent inborn errors of metabolism\n* Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in lactic acidosis\n* Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the patient or compromise his\u002Fher ability to comply with, or adversely impact, protocol requirements\n* Concurrent participation in another interventional trial or named patient program where investigational drug is received\n* Any other reason that UCB may determine that the patient is unsuitable for named patient \u002F compassionate use of doxecitine and doxribtimine\n\nTK0115:\n\n* History of liver disease or liver function test results (alanine aminotransferase \\[ALT\\], aspartate transaminase \\[AST\\], or total bilirubin) ≥ 3 × upper limit of normal (ULN) at Baseline or bilirubin \\> 1.5 × ULN. Isolated bilirubin \\> 1.5 × ULN is acceptable if bilirubin is fractionated and direct bilirubin \\\u003C 35%\n* Patients with elevated transaminases (ALT and\u002For AST \\> 3 × ULN) without increase in bilirubin should be further evaluated to exclude other causes of liver injury and may be enroled with UCB approval\n* Patient has a medical, mental, or any other extenuating condition or circumstance that may, in the opinion of the treating physician, pose an undue safety risk to the patient or compromise his\u002Fher ability to comply with, or adversely impact, protocol requirements\n* Renal insufficiency requiring dialysis\n* Discontinuation of prior nucleoside treatment for TK2d due to adverse event(s) (AEs)\n* Concurrent participation in another interventional trial or named patient program where investigational drug is received\n* Known hypersensitivity to doxecitine and doxribtimine or any excipients of the drug substance (silicon dioxide and magnesium stearate)\n* Inability to tolerate oral or enteral feeding tube administration of doxecitine and doxribtimine\n* Female patients will not be eligible to participate if they are pregnant, plan to become pregnant during the course of the program, or are breastfeeding","EXPANDED_ACCESS","This Expanded Access Record for doxecitine and doxribtimine includes the following managed access programs and status:\n\n* TK0113: Available\n* TK0115: No longer Available",[53],"Thymine Kinase 2 Deficiency",[53,55,56],"TK2","TK2d","AVAILABLE","2026-06-03",{"date":60,"type":35},"2026-06-05",{"name":41,"class":42},""]