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New treatment option for Huntington Disease
Official title A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
ClinicalTrials.gov ID: NCT06826612
What this study is testing
What is SPK-10001?
SPK-10001 is an investigational medicine, being studied as a potential treatment for huntington disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 25 to 65
You may be able to join if
- Have confirmed huntingtin (HTT) cytosine-adenine-guanine (CAG) repeat length ≥40 on genetic testing and confirmation diagnostic test by the central...
- 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...
You likely can't join if
- 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...
- 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...
See the full eligibility 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...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
- Cincinnati, Ohio, United States
- Columbus, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Pittsburgh, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 25 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States; Cincinnati, Ohio, United States; Columbus, Ohio, United States; Philadelphia, Pennsylvania, United States; Pittsburgh, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.