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)

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).

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT04993755

Neals Affiliated?

Yes

Coordinating Center Contact Information


Jay Soto / .(JavaScript must be enabled to view this email address) / 310-261-5312
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 2-arm study with a long-term, open-label treatment phase in patients with C9ORF72 ALS and/or FTD. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open-label Treatment Period, and a Follow-up Visit 4 weeks post-treatment.

Study Sponsor:

Transposon Therapeutics, Inc.

Participant Duration:

Estimated Enrollment:

40

Estimated Study Start Date:

10/01/2021

Estimated Study Completion Date:

09/01/2023

Posting Last Modified Date:

04/22/2022

Date Study Added to alsconsortium.org:

11/03/2021
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:

    Have documentation of a clinical genetic test demonstrating the presence of a confirmed repeat expansion in the C9orf72 gene from a CLIA certified laboratory
    Score ≥ 18 on the Mini-Mental State Exam (MMSE) at Screening
    Have a reliable caregiver 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)
    • ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 at Screening

    For patients with FTD:

    • A gradual, progressive decline in behavior, language, or motor function consistent with C9ORF72 hexanucleotide expansion-related syndrome such as behavioral variant FTD, primary progressive vaphasia, 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 significant brain abnormality, including, but not limited to, prior hemorrhage or infarct, cerebral contusion, encephalomalacia, aneurysm, vascular malformation, subdural hematoma, hydrocephalus, space-occupying lesion (e.g., abscess or brain tumor such as meningioma); symptoms or signs of elevated intracranial pressure, e.g., symptoms or history of head injury or abnormal funduscopic exam. If there is history or evidence on neurologic exam suggesting possible subdural hematoma (SDH), patients should be fully evaluated, including magnetic resonance imaging (MRI) if indicated, to exclude significant, new SDH

  • Site Contact Information

    University of California San Diego
    La Jolla, California 92037
    United States

    University of California Irvine
    Orange, California 92868
    United States

    University of California at San Francisco
    San Francisco, California 55455
    United States

    Johns Hopkins
    Baltimore, Maryland 21287
    United States

    Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

    Mayo Clinic
    Rochester, Minnesota 55905
    United States

    Columbia University Medical Center
    New York, New York 10032
    United States

    Hospital for Special Surgery
    New York, New York 10021
    United States

    The University of North Carolina at Chapel Hill
    Chapel Hill, North Carolina 27599
    United States

    VIB-KU Leuven Center for Brain & Disease Research
    Leuven,
    Belgium

    CHU Lille - CMRR Hôpital Roger Salengro
    Lille,
    France

    CHU Dupuytren, Limoges
    Limoges,
    France

    Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux
    Paris,
    France

    Universitaetsklinikum Ulm
    Ulm,
    Germany

    Universitair Medisch Centrum (UMC) Utrecht
    Utrecht,
    Netherlands

    Hospital Universitario Vall d'Hebron
    Barcelona,
    Spain

    Hospital de la Santa Creu i Sant Pau
    Barcelona,
    Spain

    Complejo Hospitalario Universitario de Santiago (CHUS)
    Santiago de Compostela,
    Spain

    Hospital Universitari I Politècnic La Fe
    Valencia,
    Spain