Study Purpose:
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).Study Status:
Recruiting
Disease:
Amyotrophic Lateral Sclerosis , Frontotemporal Dementia
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
TPN-101, 400 mg/day, Placebo
Placebo:
Yes
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
N/A
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
Coordinating Center Contact Information
Jay Soto / email hidden; JavaScript is required / 310-261-5312
United States
Full Study Summary:
Study Sponsor:
Transposon Therapeutics, Inc.
Estimated Enrollment:
40
Estimated Study Start Date:
10 / 01 / 2021
Estimated Study Completion Date:
09 / 01 / 2023
Posting Last Modified Date:
11 / 08 / 2022
Date Study Added to neals.org:
08 / 06 / 2021
Minimum Age:
18 Years
Maximum Age:
N/A
Can participants use Riluzole?
Yes
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
University of California San Diego | Recruiting
Dominic Ferrey, MD
La Jolla, California
92037
United States
University of California Irvine - ALS & Neuromuscular Center | Recruiting
Namita A Goyal, MD
Orange, California
92868
United States
University of California at San Francisco/UCSF | Recruiting
Adam Boxer, MD, PhD
San Francisco, California
55455
United States
John Hopkins University | Recruiting
Chiadi Onyike, M.B.B.S., MD
Baltimore, Maryland
21287
United States
Johns Hopkins Outpatient Center | Recruiting
Jeffrey D Rothstein, MD, PhD
Baltimore, Maryland
21287
United States
Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic | Recruiting
Doreen Ho, MD
Boston, Massachusetts
02114
United States
Mayo Family Clinic Northwest | Recruiting
Bradley Boeve, MD
Rochester, Minnesota
55905
United States
Hospital for Special Surgery | Recruiting
Dale J Lange, MD
New York, New York
10021
United States
Columbia University Medical Center - The Neurological Institute of New York | Recruiting
Lawrence Honig, MD, PhD
New York, New York
10032
United States
The University of North Carolina at Chapel Hill, Department of Neurology | Recruiting
Andrea Bozoki, MD
Chapel Hill, North Carolina
27599
United States
VIB-KU Leuven Center for Brain & Disease Research | Recruiting
Philip Van Damme, MD, PhD
Leuven, Flemish Brabankt
3000
Belgium
CHU Lille - CMRR Hôpital Roger Salengro | Recruiting
Florence Pasquier, MD, PhD
Lille 59037
France
CHU Dupuytren, Limoges | Recruiting
Philippe Couratier, MD, PhD
Limoges 87042
France
Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux | Recruiting
Richard Levy, MD, PhD
Paris 75013
France
Universitaetsklinikum Ulm - Klinik fuer Neurologie | Recruiting
Albert C Ludolph, MD
Ulm, Baden-Wuerttemberg
89081
Germany
Complejo Hospitalario Universitario de Santiago (CHUS) | Recruiting
Julio Pardo Fernandez, MD, PhD
Santiago de Compostela, A Coruña
15706ES
Spain
Hospital de la Santa Creu i Sant Pau | Recruiting
Alberto Lleo Bisa, MD, PhD
Barcelona 08025
Spain
Hospital Universitario Vall d'Hebron | Recruiting
Raul Juntas Morales, MD
Barcelona 08035ES
Spain
Hospital Universitari I Politècnic La Fe | Recruiting
Miquel Baquero Toledo, MD
Valencia 46026
Spain