RECRUITING

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

Conditions

Huntington Disease Huntington's Disease SPK-10001 Adult-onset HD HD Rare Disease Chorea Movement Disorder Spark Therapeutics Cognition Gene Therapy AAV mHTT

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 main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.

Official Title

A Phase 1/2, Randomized, Sequential, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of a One-Time, Bilateral, Intraparenchymal Infusion of SPK-10001 Into the Caudate and Putamen in Participants With Huntington's Disease

Quick Facts

Study Start:2025-02-21

Study Completion:2035-01-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06826612

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:25 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Have confirmed huntingtin (HTT) cytosine-adenine-guanine (CAG) repeat length ≥40 on genetic testing and confirmation diagnostic test by the central laboratory (CL) at screening. * Have striatal atrophy demonstrated by caudate/intracranial volume less than the age-adjusted cutoff values associated with HDISS Stage 1. * Have UHDRS Total Motor Score (TMS) equal to or greater than the age-adjusted cutoff value associated with HDISS Stage 2. * Have UHDRS Total Functional Capacity (TFC) greater than or equal to 11. * Use of cholinesterase inhibitors, memantine, amantadine, or riluzole must have been at stable dosing for at least 12 weeks before screening and baseline and anticipated to remain stable during the first 12 months after SPK-10001 administration. * Antidepressant or benzodiazepine use must have been at stable dosing for at least 12 weeks before screening and baseline and anticipated to remain stable during the first 12 months after SPK-10001 administration. * Antipsychotics for motor symptoms or mood stabilization (i.e., irritability or aggressive behavior) and/or tetrabenazine, valbenazine, or deutetrabenazine must have been at a stable dose for at least 12 weeks before screening and baseline and are anticipated to remain stable during the first 12 months after SPK-10001 administration.	* A safe trajectory is not able to be identified for targeting placement of the cannula into the caudate or putamen on both sides of the brain due to extent of atrophy or other anatomical features. * Have received an antisense oligonucleotide therapy during the past year. * History of deep brain stimulation. * History of or intention to undergo gene therapy, cell transplantation, or brain surgery during the course of the study. * Have participated in an investigational drug study with a systemic administration within 6 weeks or 5 half-lives of screening, whichever is longer.

Inclusion Criteria

Exclusion Criteria

* Have confirmed huntingtin (HTT) cytosine-adenine-guanine (CAG) repeat length ≥40 on genetic testing and confirmation diagnostic test by the central laboratory (CL) at screening.
* Have striatal atrophy demonstrated by caudate/intracranial volume less than the age-adjusted cutoff values associated with HDISS Stage 1.
* Have UHDRS Total Motor Score (TMS) equal to or greater than the age-adjusted cutoff value associated with HDISS Stage 2.
* Have UHDRS Total Functional Capacity (TFC) greater than or equal to 11.
* Use of cholinesterase inhibitors, memantine, amantadine, or riluzole must have been at stable dosing for at least 12 weeks before screening and baseline and anticipated to remain stable during the first 12 months after SPK-10001 administration.
* Antidepressant or benzodiazepine use must have been at stable dosing for at least 12 weeks before screening and baseline and anticipated to remain stable during the first 12 months after SPK-10001 administration.
* Antipsychotics for motor symptoms or mood stabilization (i.e., irritability or aggressive behavior) and/or tetrabenazine, valbenazine, or deutetrabenazine must have been at a stable dose for at least 12 weeks before screening and baseline and are anticipated to remain stable during the first 12 months after SPK-10001 administration.

* A safe trajectory is not able to be identified for targeting placement of the cannula into the caudate or putamen on both sides of the brain due to extent of atrophy or other anatomical features.
* Have received an antisense oligonucleotide therapy during the past year.
* History of deep brain stimulation.
* History of or intention to undergo gene therapy, cell transplantation, or brain surgery during the course of the study.
* Have participated in an investigational drug study with a systemic administration within 6 weeks or 5 half-lives of screening, whichever is longer.

Contacts and Locations

Study Contact

Huntington Study Group (HSG)

CONTACT

800-487-7671

info@hsglimited.org

Clinical Trial Director

CONTACT

Spark Therapeutics, Inc.

Study Locations (Sites)

University of Cincinnati/Cincinnati Children's Hospital

Cincinnati, Ohio, 45221

United States

Collaborators and Investigators

Sponsor: Spark Therapeutics, Inc.

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 Date2025-02-21

Study Completion Date2035-01-12

Study Record Updates

Study Start Date2025-02-21

Study Completion Date2035-01-12

Terms related to this study

Keywords Provided by Researchers

Huntington's Disease
SPK-10001
Adult-onset HD
HD
Rare Disease
Chorea
Movement Disorder
Spark Therapeutics
Cognition
Gene Therapy
AAV
mHTT

Additional Relevant MeSH Terms

Huntington Disease