Clinical Trials in the UK / NCT07378644
Recruiting
Phase 2/3
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
NCT07378644 · tracked via the Priya Life Science UK tracker
Condition(s) studied
Huntington Disease
Investigational drug(s) / intervention(s)
SKY-0515 →SKY-0515 Placebo
SKY-0515: Type: Small molecule Dosage Level(s): Low, mid, high Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 18 months Use: Experimental
SKY-0515 Placebo: Route of Administration: Oral Dosage Frequency: Once daily Blinded Treatment Duration: 18 months Use: Experimental
Study summary
The goal of this clinical trial is to test if the drug SKY-0515, an oral medication, can lower harmful proteins linked to Huntington's Disease (HD) and improve the symptoms of participants with HD. This study includes men and women aged 25 and older who have HD confirmed by genetic testing and meet certain requirements for physical ability and independence.
Eligibility
Inclusion Criteria:
* 25 years or older.
* Huntington's Disease confirmed through genetic testing, with a specific change in exon 1 of the HTT gene (CAG repeat of 40 or more).
* Total Functional Capacity (TFC) score of 10 or more).
* Total Motor Score (TMS) of 6 or more).
* Independence Score (IS) of 70 or more).
* Women who can have children must have a negative pregnancy test before starting and use two types of birth control during the study and for 30 days after the last dose of the study drug.
* Men must agree to use birth control during the study and for 90 days after the last dose.
* Agree to sign a consent form and follow the study's rules and schedule.
Exclusion Criteria:
* Other Serious health problems or brain/spinal issues that could interfere with the study or make procedures unsafe.
* Conditions that interfere with protocol-specified assessments, like an implanted medical device or difficulty getting an MRI.
* Cancer, except for some types of skin cancer, or a history of cancer in the last five years.
* Severe allergies or have reacted badly to similar drugs in the past.
* Taking medications or treatments that might interfere with the study.
* Participated in another study or taken experimental drugs in the last two months (or longer for some drugs).
* Any kind of gene therapy.
* History of suicidal thoughts, severe depression, or have attempted suicide in the past year.
* Liver function tests show significant abnormalities.
* Positive for hepatitis B, hepatitis C, or HIV.
* Pregnancy, breastfeeding, or planning to become pregnant during the study.
Primary outcome measure(s)
- Change From Baseline in the Z-score from the Composite Unified Huntington's Disease Rating Scale (cUHDRS) — 72 weeks
cUHDRS includes the Total Functional Capacity (range, 0-13; higher score means better functioning), Total Motor Score (range, 0-124; higher score means worse motor severity), Symbol Digit Modality Test (range, 0-110, correctly paired numbers-symbols in 90 seconds; higher score means better cognitive performance), and Stroop Color and Word Test (range, 0-no max value, correctly read color words in 45 seconds; higher score means better cognitive performance) scores. A z-score for each test is calculated, which alone can be used to describe relationship between an individual's test score and the mean score of a target population. A z-score of 0 is the mean, and ±1 is 1 standard deviation from the mean. For cUHDRS, z-scores of each test are summed, whereby a higher cUHDRS score is better (score of -3.06-no max value) and a change of ≥1.2 is a meaningful worsening, shown to track functional decline.
Trial sites (24)
| Facility | City | Region | Status |
|---|---|---|---|
| CenExel Denver CO | Englewood | Colorado | Not Yet Recruiting |
| Hospital El Cruce | Buenos Aires | Buenos Aires | Recruiting |
| Buenos Aires British Hospital | CABA | Buenos Aires | Recruiting |
| IADIN (Instituto Argentino de Investigación Neurológica) | CABA | Buenos Aires | Recruiting |
| CINME Centro de Investigaciones Metabólicas | CABA | Buenos Aires | Recruiting |
| Sanatorio Mater Dei | CABA | Buenos Aires | Recruiting |
| Fundación Scherbovsky | Mendoza | Mendoza Province | Recruiting |
| Centro de Pesquisas Clínicas do Hospital das Clínicas da Universidade Federal de Minas Gerais - Filial EBSERH | Belo Horizonte | Minas Gerais | Enrolling By Invitation |
| Hospital Angelina Caron | Campina Grande do Sul | Paraná | Not Yet Recruiting |
| PUCPR - Centro de Pesquisa da Pontifícia Universidade Católica do Paraná | Curitiba | Paraná | Recruiting |
| Instituto de Neurologia de Curitiba | Curitiba | Paraná | Recruiting |
| Hospital Universitario Pedro Ernesto (HUPE) | Rio de Janeiro | Rio de Janiero | Recruiting |
| Hospital Universitário Onofre Lopes (HUOL) | Natal | Rio Grande do Norte | Not Yet Recruiting |
| Faculdade de Medicina de Ribeirão Preto - FMRP | Ribeirão Preto | São Paulo | Not Yet Recruiting |
| Hospital da Universidade Federal de São Paulo - UNIFESP | São Paulo | São Paulo | Not Yet Recruiting |
| PSEG Centro de Pesquisa Clinica | São Paulo | São Paulo | Active Not Recruiting |
| North York General Hospital | Toronto | Ontario | Recruiting |
| Pontificia Universidad Católica de Chile | Santiago | Santiago Metropolitan | Recruiting |
| CETRAM (Centro de Trastornos del Movimiento) | Santiago | Santiago Metropolitan | Recruiting |
| Clínica de la Costa S.A.S | Barranquilla | Atlántico | Recruiting |
| Pineo Medical Ecosystem | Tbilisi | Georgia | Recruiting |
| Simon Khechinashvili University Hospital | Tbilisi | Georgia | Recruiting |
| Isar-Amper-Klinikum, Zentrum für Neuro- und Gerontopsychiatrie (ZNGP) | Taufkirchen | Germany | Not Yet Recruiting |
| Hospital Universitario Ramon y Cajal | Madrid | Madrid | Not Yet Recruiting |
Other trials for the same condition
Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease
Active, not recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07378644 on ClinicalTrials.gov ↗ ← All trials in the UK