Not yet recruitingRare disease

Phase 2/3 Randomized Study of SKY-0515 for Safety and Efficacy in Adults with Huntington’s Disease

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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study examines Huntington’s Disease, a rare inherited brain disorder that causes movement problems, thinking difficulties, and changes in behavior. The investigational drug being tested is SKY-0515, an oral tablet given in different strengths. Some participants will receive the active drug while others will receive a matching placebo. The purpose of the study is to see if SKY-0515 can improve the signs and symptoms of Huntington’s Disease.

Participants will take the assigned tablets each day for about a year and will attend regular visits where doctors will check safety, record any side effects, and measure changes in disease features. The main way the study looks at benefit is the change from the start to week 72 in the cUHDRS, a score that combines movement, thinking, and daily function into one number. Additional information will be gathered from separate scores such as the Total motor score (which rates movement problems), the Total functional capacity (which looks at ability to do everyday tasks), the Independence scale (which measures how much help a person needs), the Stroop Color and Word test (which checks attention and mental flexibility), and the Symbol digit modalities test (which evaluates processing speed).

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Randomization and assignment
After you join the study you will be randomly placed into one of two groups. one group receives sky-0515 tablets, the other group receives a placebo tablet that contains no active medicine.
Step 2
Receiving study medication
You will be given a supply of tablets together with written instructions. the sky-0515 tablets are available in three strengths: 4 mg, 6 mg, or 9 mg. the placebo tablets look the same but contain only an inert substance called micronized methylcellulose.
Step 3
Starting medication
You will take one tablet by mouth each day as directed in the instructions. the tablet you take will contain the dose assigned to you (either 4 mg, 6 mg, or 9 mg of sky-0515 or the placebo). you will continue this daily dosing for the entire study period, which lasts up to 72 weeks.
Step 4
Clinic visits and assessments
You will attend scheduled clinic visits during the study. at the first visit (baseline) and again at week 72 you will undergo a series of evaluations to measure the effect of the medication on your condition.
The evaluations include the composite unified huntington's disease rating scale (cuhdrs), the total motor score (tms), the total functional capacity (tfc), the independence scale (is), the stroop color and word test (scwt), and the symbol digit modalities test (sdmt). each test is explained by study staff in simple language.
Step 5
Ongoing monitoring
Throughout the 72‑week period you may be asked to report any side effects or health changes. study staff will review this information at each clinic visit to ensure safety.
Step 6
Study completion
At the end of week 72 the study medication will be stopped and final assessments will be performed. the results of these assessments will be used to determine how well sky-0515 works compared with the placebo.

Who can join the trial?

9 criteria

Age 25 years or older and able to understand and sign a written consent form, meaning you can make an informed decision about joining the study.
Genetically confirmed Huntington’s Disease: you must have a DNA test that shows more than 40 repeats of a specific gene segment (CAG repeat) in the HTT gene, which confirms the diagnosis.
Mild stage of disease (HD‑ISS Stage 2 or 3) as measured by a special rating scale:
- Functional ability score (TFC) of 10 or higher – meaning you can still perform many daily activities.
- Motor score (TMS) of 6 or lower – indicating relatively mild movement problems.
- Independence score (IS) of 70 or higher – showing a good level of independence.
- Pregnancy testing for women who could become pregnant: you must have a negative pregnancy test, be medically sterile, or be postmenopausal (no periods for 12 months, or 6 months with hormone levels indicating menopause, or after surgical removal of ovaries).
- Contraception for women who could become pregnant: if you are sexually active with a male partner, you must use two forms of birth control (one barrier method like a condom and another highly effective method) from the start of the study until at least 30 days after the last dose, and you must not donate eggs during that time.
- Contraception for men with partners who could become pregnant: you must use a male condom and ensure your partner uses a highly effective birth‑control method for the study period and for at least 90 days after the last dose, and you must not donate sperm during that time.
- Informed consent and study compliance: you must be able to sign the consent form and agree to follow all study visits, procedures, and rules.

Who cannot join the trial?

13 criteria

Having another serious neurological or medical problem (not related to Huntington’s disease) that started within the last three months and could affect your ability to join the study or the study results.
Currently taking medicines that are known to change the levels of the huntingtin protein (HTT) or its messenger RNA (mRNA – the genetic instructions that tell cells how to make a protein).
Having received any other experimental drug (not SKY‑0515) within the past 60 days or until that drug has cleared from your body (usually five half‑lives – the time it takes for half of a drug to leave the body).
Participating in another interventional study or program (such as special exercise, cognitive therapy, brain stimulation, etc.) within 90 days before screening or at any time during this study.
Having any history of gene therapy (treatment that changes your DNA).
Being at risk for suicide, which includes any suicide attempt in the past year, uncontrolled major depression, or any other serious suicide risk judged by the doctor.
Having an active infection with hepatitis B, hepatitis C, or HIV, shown by positive blood tests (for example, hepatitis B surface antigen, detectable viral RNA for hepatitis C or HIV).
Being pregnant, planning to become pregnant during the study, or currently breastfeeding.
Having known substance abuse or medical, psychological, or social problems that the doctor thinks could interfere with your participation or the study results.
Having a medical history of brain or spinal disease that would make a lumbar puncture (a needle test of spinal fluid) unsafe.
Having any condition that would prevent you from completing required tests, such as an implanted shunt or anything that makes MRI scans impossible.
Having an active cancer or a history of cancer in the past five years, except for certain skin cancers that were surgically removed or low‑grade cervical changes.
Having a known allergy or severe reaction (hypersensitivity or anaphylaxis) to the study drug or any of its ingredients.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Charite Universitaetsmedizin Berlin KöR
Verified
Berlin, Germany
Hospital Clinic De Barcelona
Verified
Barcelona, Spain
Hospital De La Santa Creu I Sant Pau
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Barcelona, Spain

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Investigated drugs

SKY-0515 is an experimental oral tablet being studied as a possible treatment for Huntington’s disease. In this trial, participants take the tablet to see how it affects the symptoms and progression of the disease. The study looks at how well the drug works, its safety, and how the body responds to different amounts of the medication. By comparing the results with those from participants who receive a placebo, researchers can determine whether SKY-0515 could become a new therapy for people with Huntington’s disease.

What is already known about the treatment

SKY-0515 4 mg tablet
This oral tablet is taken by mouth and contains 4 mg of the experimental drug SKY‑0515. It is currently being studied in a Phase 2/3 clinical trial and has not yet received regulatory approval. The tablet is tested for Huntington’s disease and works by lowering the amount of the abnormal huntingtin protein that harms brain cells. It is classified as an investigational disease‑modifying small‑molecule therapy.
SKY-0515 6 mg tablet
This oral tablet is taken by mouth and provides 6 mg of the investigational compound SKY‑0515. It remains in clinical development and is not approved for routine medical use. The medication is being evaluated for Huntington’s disease and acts by reducing the production of the toxic huntingtin protein that causes neuronal damage. It belongs to the class of experimental disease‑modifying oral agents.
SKY-0515 9 mg tablet
This oral tablet is taken by mouth and delivers 9 mg of the new drug SKY‑0515. It is still in the Phase 2/3 research stage and has no market authorization. The tablet is aimed at treating Huntington’s disease by decreasing levels of the harmful huntingtin protein that leads to brain cell loss. It is considered an investigational small‑molecule disease‑modifying therapy.

Investigated diseases

Huntington’s Disease - It is a genetic disorder that slowly damages brain cells, leading to problems with movement, thinking, and mood. Symptoms typically begin in adulthood and become more noticeable over time. Coordination and speech get increasingly difficult as the disease advances. Memory, planning, and other thinking skills gradually decline. Mood swings and behavioral changes may also appear as the condition progresses.