{"id":"39d2e153-3400-5041-9868-09922063ed73","stable_key":"f992b796-377d-53bf-a39b-7e5d41dbe194:b5-clin-fort-adl-null","predicate":"does-not-significantly-improve","statement":"FORT found no significant difference in 24-week PKAN-ADL change between fosmetpantotenate and placebo: adjusted difference −0.09 points, 95% CI −1.69 to 1.51, P=0.9115.","claim_class":"observational","status":"source_derived_draft","evidence_grade":"ungraded","direction":"neutral","is_public":true,"mechanism_event_id":"e79b1dcf-7352-515d-9ae4-fc4e52d0ce89","mechanism_event_label":"The prodrug did not improve the trial’s primary daily-function outcome.","subject":{"id":"231b8e3d-46a2-5938-96da-07a0ca09e1d3","slug":"fosmetpantotenate","display_name":"Fosmetpantotenate","entity_type_key":"small_molecule"},"object":{"id":"b54c61fa-516f-523f-ba4c-46aeb79ad036","slug":"pkan-activities-of-daily-living-score","display_name":"PKAN activities-of-daily-living score","entity_type_key":"cellular_process"},"evidence_count":1,"mechanism_event":{"id":"e79b1dcf-7352-515d-9ae4-fc4e52d0ce89","stable_key":"f992b796-377d-53bf-a39b-7e5d41dbe194:b5-clin-fort-adl-null-event","event_type":"observed_intervention","label":"The prodrug did not improve the trial’s primary daily-function outcome.","description":"FORT found no significant difference in 24-week PKAN-ADL change between fosmetpantotenate and placebo: adjusted difference −0.09 points, 95% CI −1.69 to 1.51, P=0.9115.","status":"provisional","compartment":null,"participants":[{"entity":{"id":"496571a5-06f5-5aa5-b95b-aae8b6556c7f","slug":"pank2","display_name":"Human pantothenate kinase 2 / PANK2","entity_type_key":"protein"},"role":"genetically impaired pathway machinery","stoichiometry":null,"state_label":"","sequence_order":0,"notes":""},{"entity":{"id":"231b8e3d-46a2-5938-96da-07a0ca09e1d3","slug":"fosmetpantotenate","display_name":"Fosmetpantotenate","entity_type_key":"small_molecule"},"role":"subject","stoichiometry":null,"state_label":"","sequence_order":1,"notes":""},{"entity":{"id":"b54c61fa-516f-523f-ba4c-46aeb79ad036","slug":"pkan-activities-of-daily-living-score","display_name":"PKAN activities-of-daily-living score","entity_type_key":"cellular_process"},"role":"target","stoichiometry":null,"state_label":"","sequence_order":2,"notes":""}]},"contexts":[{"dimension":"availability_state","value_text":"machinery_impairment","comparator":null,"unit":null,"notes":"Imported condition classification; unverified.","entity":null},{"dimension":"cross_nutrient","value_text":"false","comparator":null,"unit":null,"notes":"","entity":null},{"dimension":"evidence_location","value_text":"Full primary BioC Methods, Results and Discussion.","comparator":null,"unit":null,"notes":"","entity":null},{"dimension":"experimental_model","value_text":"FORT randomized double-blind multicenter placebo-controlled trial; 84 patients aged 6–65 with pathogenic PANK2 variants","comparator":null,"unit":null,"notes":"","entity":null},{"dimension":"exposure","value_text":"24 weeks; fosmetpantotenate 41 versus placebo 43. 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Sponsor-funded trial; no general safety or dietary-efficacy conclusion.","comparator":null,"unit":null,"notes":"","entity":null},{"dimension":"nutrient_topic","value_text":"Pantothenic acid (vitamin B5) research collection; topical membership is not evidence of a direct dietary effect.","comparator":null,"unit":null,"notes":"","entity":{"slug":"pantothenate","display_name":"Pantothenate (vitamin B5)","entity_type_key":"small_molecule"}},{"dimension":"organism","value_text":"Homo sapiens","comparator":null,"unit":null,"notes":"","entity":null},{"dimension":"plain_language","value_text":"The prodrug did not improve the trial’s primary daily-function outcome.","comparator":null,"unit":null,"notes":"","entity":null},{"dimension":"primary_references","value_text":"[b5-clin-fort2021] Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration. (2021). https://pubmed.ncbi.nlm.nih.gov/33200489/ DOI: 10.1002/mds.28392","comparator":null,"unit":null,"notes":"","entity":null},{"dimension":"tissue_or_cell_type","value_text":"Patient/surrogate functional rating and clinician-rated motor performance","comparator":null,"unit":null,"notes":"","entity":null},{"dimension":"trigger_kind","value_text":"machinery_impairment","comparator":null,"unit":null,"notes":"Imported condition classification; unverified.","entity":null}],"evidence":[{"id":"fc878b8e-1981-5c4d-a96c-bf82bab115e1","evidence_kind":"source_excerpt","locator":"Lines 1439-1451","start_line":1439,"end_line":1451,"excerpt":"### b5-clin-fort-adl-null\nFORT found no significant difference in 24-week PKAN-ADL change between fosmetpantotenate and placebo: adjusted difference −0.09 points, 95% CI −1.69 to 1.51, P=0.9115.\nCondition category: machinery_impairment\nnutrient_topic: Pantothenic acid (vitamin B5) research collection; topical membership is not evidence of a direct dietary effect.\nplain_language: The prodrug did not improve the trial’s primary daily-function outcome.\norganism: Homo sapiens\ntissue_or_cell_type: Patient/surrogate functional rating and clinician-rated motor performance\nexperimental_model: FORT randomized double-blind multicenter placebo-controlled trial; 84 patients aged 6–65 with pathogenic PANK2 variants\nlimitations: Genetic disease, not nutritional B5 deficiency. No measure of brain target engagement was available, so a negative functional endpoint does not locate the failed biochemical step. Sponsor-funded trial; no general safety or dietary-efficacy conclusion.\nexposure: 24 weeks; fosmetpantotenate 41 versus placebo 43. Adults and children at least 40 kg: 300 mg three times/day; children 20–<40 kg: 150 mg three times/day; children <20 kg: 75 mg three times/day.\ncross_nutrient: false\nevidence_location: Full primary BioC Methods, Results and Discussion.\n[b5-clin-fort2021] Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration. (2021). https://pubmed.ncbi.nlm.nih.gov/33200489/ DOI: 10.1002/mds.28392","model_system":"FORT randomized double-blind multicenter placebo-controlled trial; 84 patients aged 6–65 with pathogenic PANK2 variants","directness":"author_interpretation","verification_status":"source_derived_draft","notes":"Exact curation-document quotation, not publisher quotation. Study references: [b5-clin-fort2021] Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration. (2021). https://pubmed.ncbi.nlm.nih.gov/33200489/ DOI: 10.1002/mds.28392","relationship":"supports","weight":1.0,"link_notes":"","source":{"id":"0716b500-dd0b-5425-b9d0-d88bc9c09e86","stable_key":"import-f992b796-377d-53bf-a39b-7e5d41dbe194","title":"Pantothenic acid (vitamin B5): coenzyme A, deficiency and nutrient interactions (2026-09-17)","document_type":"imported_text","citation_label":"AI-assisted literature curation; primary study URLs and scope retained in the document and extraction. Not publisher full text.","file_path":"","sha256":"b5a49f5a2373ca6064c9a4e014e052dad2f6f199a82bb09b26b82b831c36110b","revision_id":"78f3e793-f084-5ee2-8703-b661c4b650bf","review_status":"unverified_draft","notes":""}}],"relations":[],"conflicts":[],"corrections":[],"research":null}