Informations générales (source: ClinicalTrials.gov)
A Phase 2a Study of TPN-101 in Patients With Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated With Hexanucleotide Repeat Expansion in the C9orf72 Gene (C9ORF72 ALS/FTD)
Interventional
Phase 2
Transposon Therapeutics, Inc. (Voir sur ClinicalTrials)
octobre 2021
septembre 2023
08 mars 2025
This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients
with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated
with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).
Etablissements
| Les établissements d'Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| AP-HP - Hôpital La Pitié-Salpêtrière | Contact (sur clinicalTrials) | ||||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| CHU Dupuytren, Limoges - 87042 - Limoges - France | Contact (sur clinicalTrials) | ||||
| CHU Lille - CMRR Hôpital Roger Salengro - 59037 - Lille - France | Contact (sur clinicalTrials) | ||||
Critères
Tous
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
- 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
- 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