A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD
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|ClinicalTrials.gov Identifier: NCT04993755|
Recruitment Status : Recruiting
First Posted : August 6, 2021
Last Update Posted : December 9, 2022
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis Frontotemporal Dementia||Drug: TPN-101, 400 mg/day Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||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)|
|Actual Study Start Date :||October 1, 2021|
|Estimated Primary Completion Date :||September 1, 2023|
|Estimated Study Completion Date :||September 1, 2023|
|Experimental: TPN-101, 400 mg/day||
Drug: TPN-101, 400 mg/day
400 mg/day of study investigational drug TPN-101 once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).
|Placebo Comparator: Placebo||
Placebo once daily for 24 weeks (double-blind treatment) followed by 400 mg/day TPN-101 for 24 weeks (open-label treatment).
- Assess the safety and tolerability of TPN-101 in patients with C9ORF72 amyotrophic lateral sclerosis (ALS)/frontotemporal dementia (FTD) [ Time Frame: 48 weeks ]Incidence and severity of spontaneously reported treatment-emergent adverse events (TEAEs) associated with TPN-101 v. placebo administered for up to 48 weeks in patients with C9ORF72 ALS/FTD
- Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF) [ Time Frame: 48 weeks ]
- Assess the pharmacodynamic effect of TPN-101 on neurodegeneration as measured by changes in the levels of CSF and blood neurofilament light (NfL) [ Time Frame: 48 weeks ]
- Assess the clinical effect of TPN-101 as measured by changes in score on the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: 48 weeks ]The ALSFRS-R measures speech, salivation, swallowing, handwriting, cutting food and handling utensils (with or without gastrostomy), dressing and hygiene, turning in bed and adjusting bed clothes, walking, climbing, stairs, and breathing. Scores range from 0 to 40, with higher scores indicating that more function is retained.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04993755
|Contact: Jay Sotoemail@example.com|