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

Launched by SPARK THERAPEUTICS, INC. · Feb 10, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called SPK-10001 to see if it is safe and effective for people with Huntington's Disease. Huntington's Disease is a genetic condition that affects movement, thinking, and behavior. The researchers want to find out if this treatment can help manage some of the symptoms of the disease. The trial is currently looking for participants aged between 18 and 75 who have specific genetic markers and certain levels of motor and functional abilities related to Huntington's Disease.

To join the study, participants must have a confirmed genetic test showing they have Huntington's Disease, along with signs of brain changes related to the condition. They also need to be on stable doses of certain medications for at least 12 weeks before joining. Throughout the study, participants will receive the gene therapy and will be monitored closely for any side effects and changes in their symptoms. This trial is important because it could lead to new treatment options for people living with Huntington's Disease.

Gender

ALL

Eligibility criteria

• Key Inclusion 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.
• Key Exclusion Criteria:
• • 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.
• • Additional protocol-defined inclusion/exclusion criteria apply.