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This study examines the drug TPN-101, compared with a placebo, in patients with ALS (amyotrophic lateral sclerosis) and/or FTD (frontotemporal dementia) who have a repeat expansion in the C9orf72 gene. Patients with a genetically confirmed C9orf72 hexanucleotide repeat expansion who meet defined diagnostic criteria could take part. It was carried out as a Phase 2 study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Documentation of a clinical genetic test demonstrating a hexanucleotide repeat expansion (HRE) in the C9orf72 gene * Has a reliable caregiver/informant to accompany the patient to all study visits For patients with ALS (with or without FTD): * Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria * Onset of weakness within 3 years prior to Screening * Slow vital capacity (SVC) ≥ 60% of predicted normal adjusted for sex, age, and height (from the sitting position) * Able to perform reproducible pulmonary function tests. * ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 and score of 3 or 4 on Item #3 (swallowing) at Screening For patients with FTD: * A gradual, progressive decline in behavior, language, or motor function consistent with mild cognitive impairment, mild behavioral impairment, mild cognitive/behavioral impairment, behavioral variant FTD, primary progressive aphasia, or amnestic syndrome * CDR Dementia Staging Instrument plus National Alzheimer's Coordinating Center Behavior and Language Domains (CDR plus NACC FTLD) global score of 0.5-2.0 at Screening Exclusion Criteria: * Presence of other significant neurological or psychiatric disorders * History of clinically significant brain abnormality * Clinically significant medical illness * Tracheostomy or diaphragmatic pacing * Autoimmune disease requiring treatment or management (quiescent rheumatoid arthritis, psoriasis, or controlled Type 1 diabetes are acceptable) * History of human immunodeficiency virus (HIV) or hepatitis B infection, or any active infection during Screening, unless the patient will have been symptom-free for at least 30 days prior to randomization