FALCON-HD
Recruiting
Official title: A Phase 2/3 Randomized, Double Blind, Placebo-Controlled, Dose Ranging Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington’s Disease
Randomized, double-blind, placebo-controlled study. Looking at the safety, pharmacodynamics, and efficacy of SKY-0515, a novel, orally(by mouth) given small molecule.
The goal of this clinical trial is to test if the drug SKY-0515 can lower harmful proteins linked to Huntington’s Disease (HD) and improve the symptoms of participants with HD.
It will aim to lower both the normal(HTT) and mutant(mHTT) allele.
Phase: 2 and 3
Subtype: Interventional Therapeutic
Primary Purpose: Treatment
Intervention Type: small molecule
Primary Intervention: SKY-0515
Mode of Action: HTT Lowering
Duration: 18 months
Ages Eligible
for Study:
25 Years and older
(Adult, Older Adult )
Sexes Eligible
for Study:
All
Accepts Healthy Volunteers:
No
- 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.
- 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.
LOCATIONS
COUNTRIES
Georgia
TRIAL SITE: Simon Khechinashvili University Hospital
33 Chavchavadze Ave, Tbilisi, Georgia
Email: sky0515trials@skyhawktx.com
TRIAL SITE: Pineo Medical Ecosystem
93 Gorgasali St, Tbilisi, Georgia
Email: sky0515trials@skyhawktx.com
