RECRUITING

A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)

Talk to us

Conditions

Frontotemporal DementiaFTDFTD-GRNDementia, FrontotemporalGene TherapyAAVIntrathalamicIntraparenchymalProgranulinBehavioral Variant FTDPrimary Progressive AphasiaPGRNGranulinDementiaDementia Gene TherapyAAV9

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). FTD-GRN is an early-onset form of dementia, a progressive brain disorder that affects behavior, language and movement. These symptoms result from below normal levels of a protein called progranulin (PGRN) in the brain, which leads to the death of nerve cells (neurons), affecting the brain's ability to function. The main questions that the study aims to answer are: 1. Is a one-time treatment with AVB-101 safe for patients with FTD-GRN? 2. Does a one-time treatment with AVB-101 restore PGRN levels to at least normal levels? 3. Could AVB-101 work as a treatment to slow down or stop progression of FTD-GRN? In this study there is no placebo (a dummy pill or treatment used for comparison purposes), so all participants will receive a one-time treatment of AVB-101 delivered directly to the brain, with follow-up assessments for 5 years.

Official Title

A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

Quick Facts

Study Start:2023-08-30
Study Completion:2030-10-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06064890

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:30 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male or female, 30 to 75 years of age
  2. * Carriers of a pathogenic GRN mutation
  3. * FTD as evidenced by CDR + NACC FTLD global score of 0.5, 1.0, or 2.0
  4. * Presence of 1 or more of the criteria for diagnosis of possible bvFTD or PPA
  5. * A protocol defined minimum thalamic volume on each side on Screening MRI
  6. * Able and willing to comply with all procedures and the study visit schedule
  7. * Able and willing to give written informed consent prior to study participation, and agree to designate a legal representative to act on their wishes to continue participation should they lose capacity to consent at some point during the study
  8. * An identified, informed study partner who is able and willing to support the participant in the study and to provide assessments of the participant during the study
  1. * Severe dementia, defined as CDR + NACC FTLD global score of 3.0, or other symptoms that preclude the ability to comply with study procedures and/or pose unacceptable safety risk to the subject
  2. * Any concurrent disease that may cause cognitive impairment unrelated to mutations in the GRN gene, such as other causes of dementia, neurosyphilis, hydrocephalus, stroke, small vessel ischemic disease, uncontrolled hypothyroidism, or vitamin B12 deficiency
  3. * Clinically significant abnormality on MRI at Screening considered to be a contraindication to Intrathalamic infusion
  4. * Surgically significant pattern of brain atrophy on MRI at Screening that interferes with planned neurosurgical trajectory
  5. * Previous treatment with any gene or cell therapy
  6. * Previous treatment with any investigational medicinal product (IMP) within 60 days or 5 half-lives (whichever is longer) prior to study drug treatment
  7. * Concomitant disease, any clinically significant laboratory abnormality, or treatment which, in the opinion of the Investigator, may pose an unacceptable safety risk to the participant or interfere with study conduct or the participant's ability to comply with study procedures including neurosurgical administration under anesthesia

Contacts and Locations

Study Contact

AviadoBio Clinical Trials
CONTACT
+44 203-089-7917
clinicaltrials@aviadobio.com

Study Locations (Sites)

The Ohio State University (OSU) Wexner Medical Center
Columbus, Ohio, 43210
United States

Collaborators and Investigators

Sponsor: AviadoBio Ltd

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-08-30
Study Completion Date2030-10-31

Study Record Updates

Study Start Date2023-08-30
Study Completion Date2030-10-31

Terms related to this study

Keywords Provided by Researchers

  • Gene Therapy
  • AAV
  • Intrathalamic
  • Intraparenchymal
  • Progranulin
  • Behavioral Variant FTD
  • Primary Progressive Aphasia
  • PGRN
  • Granulin
  • Dementia
  • Dementia Gene Therapy
  • AAV9

Additional Relevant MeSH Terms

  • Frontotemporal Dementia
  • FTD
  • FTD-GRN
  • Dementia, Frontotemporal