SKY-0515 for adults with Huntington's disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a randomized, double-blind, placebo-controlled, multi-site trial testing an oral small molecule, SKY-0515, across three dose levels versus placebo. SKY-0515 is designed to reduce both mutant and wild-type huntingtin (HTT) protein levels and to lower PMS1, a protein linked to somatic CAG repeat expansion that drives disease progression. Eligible participants are adults (25+) with genetically confirmed HD (CAG ≥ 40) who meet specified motor and functional thresholds and can undergo MRI and required safety monitoring. Safety, pharmacodynamic markers in blood, MRI measures, and clinical outcomes including the UHDRS will be used to compare the treatment arms to placebo.

Why it matters

Who can join: the full rules

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.

Where it's running

• CenExel Denver CO
  Englewood, Colorado, United States Recruiting
• Hospital El Cruce
  Buenos Aires, Buenos Aires, Argentina Recruiting
• Buenos Aires British Hospital
  Caba, Buenos Aires, Argentina Recruiting
• IADIN (Instituto Argentino de Investigación Neurológica)
  Caba, Buenos Aires, Argentina Recruiting
• CINME Centro de Investigaciones Metabólicas
  Caba, Buenos Aires, Argentina Recruiting
• Sanatorio Mater Dei
  Caba, Buenos Aires, Argentina Recruiting

Study contacts

• Study coordinator: Study Director, MD
• Email: sky0515trials@skyhawktx.com
• Phone: 6178580041

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Pridopidine for Huntington's Disease: a 1-year, randomized, placebo-controlled study

  This trial tests whether pridopidine can slow the progression of Huntington's disease in adults compared with placebo over one year.

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• Low-intensity focused ultrasound targeting the globus pallidus to improve movement in Huntington's disease

  This trial tests whether different pulse frequencies of low-intensity focused ultrasound (LIFU) aimed at a deep brain target can safely reduce chorea and other motor symptoms in adults with Huntington's disease.

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• Safety and tolerability of human neural stem cell implants for Huntington's disease

  This research will test whether an implant of hNSC-01 human neural stem cells is safe and tolerable for adults with Huntington's disease.

  Recruiting Phase 1/2 University of California, Irvine · Irvine, California · NCT07451613

• Votoplam for adults with early symptomatic Huntington's disease

  This trial will test whether votoplam (HTT227) is safe and can slow disease progression in adults with early symptomatic Huntington's disease.

  Recruiting at 117 sites Phase 3 Novartis · Birmingham, Alabama and 116 other locations · NCT07326709

• Comparing three social cognition tests in adults with early Huntington disease

  This project will test whether the DMASC-MC and two established social cognition tests can detect social thinking problems in adults with early Huntington disease.

  Recruiting Region Skane · Lund · NCT07253038

• Wearable monitoring of walking and arm movement in adults with neuromuscular or metabolic movement disorders

  This project uses a wrist/ankle wearable to continuously monitor walking and upper-limb movement in ambulant adults with neuromuscular diseases or obesity to see if digital measures like stride speed track real-life motor function.

  Recruiting Centre Hospitalier Universitaire de Liege · Liège · NCT07136844

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.