About the Study
Osteoarthritis (OA) is one of the most common diseases in this country. It occurs when the protective cartilage that cushions the ends of the bones wears down over time. Main symptoms of OA are pain, swelling and stiffness of the joint, and reduced ability to perform daily activities. There is currently no cure and the drugs that are used to help people with OA are not as good as we would like them to be – they do nothing to stop disease progression. The reason we are doing this research is to find out if the gene therapy being studied, GNSC-001 is safe and to see which of two (2) dose levels performs better in these terms. We will also monitor your pain level and your ability to perform daily activities. This information will help inform further studies with GNSC-001, which may show whether GNSC-001 can slow down or stop disease progression.
The DONATELLO Study is investigating a single injection of study drug or placebo into the knee.
The study drug (or investigational drug) is one that is being tested in clinical research studies and is not approved for use by the general public. Placebo is a drug that looks like a study drug but has no active ingredients in it.
This research will involve a single injection into your knee using a syringe and needle (intra-articular injection). Depending on your group assignment you may be given a separate medication to take by mouth at the beginning of the research that may change how GNSC-001 works. Because we are looking at changes in your knee OA, it is important to maintain good bone health. The study doctor may discuss supplementation with Calcium and Vitamin D3 to ensure optimal bone health is maintained. After the knee injection is given on Day 1, there will be five (5) follow up visits to the clinic over a 2-year period. Following that, there will be three (3) additional telephone calls with the clinic, once per year over the following 3 years.
There will be 5 treatment groups in this study: a high dose of GNSC-001, a low dose of GNSC-001, and a placebo group (placebo means a sterile solution with no gene therapy). In this informed consent document, we will refer to the GNSC-001 (high or low dose) and Placebo as “study drug” from here on. The group to which you are assigned will determine whether the study doctor will also prescribe a short course (43 days) of prednisone (a commercially available oral steroid) to be taken by mouth to see whether this type of drug can have an effect on GNSC-001.
What is a Clinical Study?
Clinical studies show us what is effective in medicine and health care. In a clinical study, volunteers help researchers look at new ways to prevent, detect, or treat conditions. They take place in doctors’ offices, medical centers, community hospitals and clinics, and veterans’ hospitals around the world.
Before a treatment can be approved for everyday use and made accessible to most patients, the U.S. FDA requires that a potential therapy’s safety and efficacy be evaluated in clinical studies. Clinical studies do not just study new drugs — they also help improve treatments already given to patients.
How Are the Study Drugs Being Tested?
In this study, we are seeking 50 people that meet eligibility requirements during screening who will be randomly allocated to 1 of 5 treatment arms (single dose). The study will consist of a Screening period, Baseline/Treatment Day, Primary Follow-up period (12 months), and a Long-term Follow up period (48 months).
During the study, participants will visit the study site regularly for several types of tests and assessments. There are a total of 7 study visits and 4 telephone visits planned over the 266-week period. These may include:
- Vital signs measurements (body temperature, breathing rate, blood pressure, and heart rate)
- Physical examinations
- Electrocardiograms (to measure the electrical activity of the heart)
- Blood and urine tests
- Questionnaires
Not all these activities will occur on every visit.
If you qualify and choose to participate, you may have access to a new investigational drug that may help improve your symptoms.
Who Can Participate?
You may be eligible to participate in the Knee Osteoarthritis research study if you:
- Are between 40 to 75 years old.
- Have osteoarthritis in at least one knee diagnosed by a physician.
- Moderate to severe symptoms in the knee despite having previous therapy of at least 2 three-month therapies such as activity modification, weight loss, physical therapy, opioids, anti-inflammatory medications, injection of hyaluronic acid, or steroids.
- Are not pregnant or breast feeding and must comply with birth control requirements.
- Do not have inflammatory arthritis (such as gout or rheumatoid arthritis) or connective tissue or other immunological diseases.
- Do not have any form of joint degeneration as part of another syndrome (e.g., Ehler’s Danlos, Stickler syndrome, etc.).
- Do not have a history of cancer or have received treatment for cancer within the last 5 years, except for skin cancer.
- Do not have acute or chronic medical conditions (e.g., cardiovascular disease, bleeding disorder)
- Have not had surgery in the knee within the 6 months.
- Do not have a history of human immunodeficiency virus (HIV), hepatitis B or C, or tuberculosis infection, or active hepatitis A.
- Do not have septic arthritis in the knee.
- Do not have an infectious disease affecting the knee or overlying skin.
- Have not had an infection requiring systemic treatment within last 4 weeks
- Do not have diabetes or pre-diabetes.
- Do not have osteoporosis.
- Do not have glaucoma.
As a study participant, all study-related care will be provided to you at no cost, including the study drug.
Participants will be paid up to $775 for study participation.
What Should I Expect?
If you are interested in learning more about the DONATELLO study and if it may be a good fit for you, please submit the short questionnaire. The answers you submit will be used for study purposes only.
Once you submit the form, a member from our team will reach out to you and connect you with a member of the research team if it seems you may qualify for this study.
Please note that answering these questions does not obligate you to participate in the study. If you have any questions, simply email
[email protected]
or call (415) 326-8831.
How is Knee Osteoarthritis Diagnosed?
A healthcare provider will be able to diagnose knee osteoarthritis with a combination of medical history, physical examination, imaging studies, and sometimes laboratory tests.
Who Does Knee Osteoarthritis Affect?
Osteoarthritis affects over 32.5 million Americans and is the leading cause of disability among the elderly. Knee Osteoarthritis accounts for more than 80% of the disease’s total burden and affects at least 19% of American adults aged 45 years and older. Substantial evidence indicates that knee osteoarthritis is proximately caused by the breakdown of joint tissues from mechanical loading and inflammation.
