A Phase Ia, Multicenter, Double-Blind, Placebo-controlled Study to Evaluate the Safety of CYT-108 for the Therapy of Mild to Moderate Primary Osteoarthritis of the Knee
The goal of this Phase 1 study is to investigate the safety of CYT-108, our experimental recombinant protease inhibitor drug candidate for osteoarthritis, in a population of patients suffering from osteoarthritis of the knee. The main questions this study aims to answer are: 1. are two doses of CYT-108, delivered 12 weeks apart, safe when injected directly into the joint? 2. does administration of CYT-108 result in a reduction in pain, stiffness, and improvement in daily physical function? Participants will either receive a placebo (Phosphate Buffer Saline, PBS) or CYT-108, and will be asked to report their pain/stiffness at weeks 1, 4, 8, 12, 16, and 26 (in a physician's office) after the initial injection, using a questionnaire to be provided by the physician. In addition to reporting the magnitude of pain, participants will also be asked about the onset of pain reduction. Researchers will compare the pain/stiffness scores between the CYT-108-treated to placebo-treated groups along the 26 week duration of the study. Participants will also receive blood draws along the course of the study, and researchers will analyze this blood for the presence of cartilage degradation product in attempt to identify disease modification (i.e., a reduction in cartilage degradation) in response to CYT-108 treatment.
• Participant with mild to moderate primary knee OA.
• Provides signed written informed consent before any study procedure is performed.
• Is willing and able to complete effectiveness and safety questionnaires and can read and understand study instructions.
• Adult aged 18 years or older at the time of informed consent.
• Participants of childbearing potential must have a negative serum pregnancy test at screening and negative urine pregnancy test pre-dose on Day 1, and must agree to remain sexually abstinent, or use medically effective contraception (refer to Appendix 14.2) or have a partner who is sterile or same sex, from Screening until at least 3 months after the last treatment. Participants must not be planning to father children or donate sperm from Screening until at least 3 months after the last treatment.
• Male participants must agree to use a double method of contraception to prevent partner's pregnancy during and 3 months after the last treatment. Male participant should not donate sperm during the same period (for more detail see appendix 14.2).
• Is ambulatory (assistive devices or knee braces are allowed if used during the study).
• Has symptomatic OA in the target knee (one knee) either medial or lateral.
‣ X-ray confirmation of OA at the target knee prior to screening with a grade 2 or 3 score on the K-L grading scale (Kellgren, 1957) using X-ray performed within 6 months of Screening.
⁃ Knee pain as demonstrated by an average WOMAC pain scale score of 1.5 - 3 at Screening and pre-dose Day 1.
• Remains symptomatic despite having received standard of- care therapy, such as daily doses of NSAID or any pain medication leading to the study screening.
⁃ Has not received corticosteroid injection within 6 weeks prior to screening.
⁃ Has not received hyaluronic acid treatment, Platelet rich plasma (PRP) or any other protein based or stem cells treatment, or any investigational drug within 12 weeks prior to screening.