Myositis may knock me down, but I will keep getting back up.
A clinical research study evaluating a drug that may help slow the progression and relieve symptoms of dermatomyositis and anti-synthetase syndrome is now enrolling.
About the HZNP-DAX-205 DM/ASIM Study
The HZNP-DAX-205 DM/ASIM Study is evaluating a study drug to see if it may safely and efficaciously reduce the symptoms of two types of rare myositis: dermatomyositis (DM) and anti-synthetase inflammatory myositis (ASIM). These disorders cause chronic inflammation of the muscles that can lead to permanent muscle damage. If you still have symptoms even with available medications, or you cannot tolerate the side effects of those medications, this research study may be an option for you.
The research study will last up to 60 weeks and will include up to 16 visits to the study site. During the first part of the Study Treatment Period, you will have a 1 in 2 (50%) chance of receiving the study drug or a placebo. (A placebo looks like the study drug but contains no active drug.) During the second part of the Study Treatment Period, all participants will be eligible to receive the active study drug.
The study drug will be given by subcutaneous (under the skin) injection once every 4 weeks at the study site starting at Visit 2. You may also be able to continue taking your current DM or ASIM medications for your symptoms during the research study.
You may be eligible to participate in this research study if you:*
- Are aged 18 to 75 years old
- Have been diagnosed with DM or ASIM
- Currently experience symptoms of DM or ASIM
*The research study team can discuss any other additional criteria with you.
Why Participate?
If you qualify and decide to take part, you could receive:
- All study-related care and the study drug at no charge
- Regular monitoring of your DM or ASIM and overall health by physicians who specialize in these conditions
- Reimbursement for transportation costs to attend study visits, as needed
- The opportunity to help advance scientific knowledge of DM or ASIM
Please know that your safety while participating in the HZNP-DAX-205 DM/ASIM Study is our highest priority. Research team members are available to discuss any questions or concerns you may have at any point throughout the research study. They can also inform you about COVID-19 safety protocols.
Your participation in any research study is voluntary, and you are always free to withdraw at any time, for any reason. Your privacy will be maintained throughout the research study.
DM and ASIM Overview
DM and ASIM are part of a group of rare conditions known as myositis that affect the muscles and, in some cases, other parts of the body such as the skin, lungs, or heart. Myositis is caused by a failure of the body’s natural defense system (immune system) to protect muscles and instead attacks and damages them. This causes people with myositis to feel muscle weakness, swelling, and pain. They may also experience general tiredness, a sense of unwellness, weight loss, and low-grade fevers.1,2
The most noticeable symptoms of DM occur on the skin, including3,4:
- A distinctive reddish-purple (heliotrope) rash on the upper eyelids
- Scaling and redness on the knuckles, elbows, or knees
- A rash across the neck, shoulders, and back (shawl sign)
Other DM symptoms include3,4:
- Difficulty with movement such as raising arms or climbing stairs
- Trouble speaking or swallowing
People with ASIM are more likely to have lung complications, which can cause shortness of breath and dry cough. Other symptoms of ASIM include5,6:
- Thickening and cracking of skin on the hands (mechanic’s hands)
- Joint pain, stiffness, and swelling (polyarthritis)
While there are medications that treat the symptoms of these conditions, they may not control those symptoms well enough, and they may have significant side effects. This shows a need for different treatment options.
References
- Inflammatory Myopathies Fact Sheet. National Institute of Neurological Disorders and Stroke. Accessed November 2, 2022. https://www.ninds.nih.gov/inflammatory-myopathies-fact-sheet
- About Myositis. The Myositis Association. Accessed November 25, 2022. https://www.myositis.org/about-myositis/
- Dermatomyositis. National Organization for Rare Disorders. Accessed November 2, 2022. https://rarediseases.org/rare-diseases/dermatomyositis/
- Dermatomyositis. The Myositis Association. Accessed November 2, 2022. https://www.myositis.org/about-myositis/types-of-myositis/dermatomyositis
- Antisynthetase Syndrome. National Organization for Rare Disorders. Accessed November 2, 2022. https://rarediseases.org/rare-diseases/antisynthetase-syndrome/
- Antisynthetase Syndrome. The Myositis Association. Accessed November 2, 2022. https://www.myositis.org/about-myositis/complications/antisynthetase-syndrome/
Frequently Asked Questions
This section will help answer some important questions you may have.
About the HZNP-DAX-205 DM/ASIM Study
The research study is evaluating a study drug to see if it may safely and efficaciously reduce the symptoms of two rare muscle disorders: DM and ASIM.
A placebo looks like the study drug but contains no active drug. Researchers use a placebo to see if the study drug works better or is safer than taking nothing.
During the frst part of the Study Treatment Period in this study, you will have a 1 in 2 (50%) chance of receiving the study drug or a placebo. During the second part of the Study Treatment Period in this study, all participants will be eligible to receive the active study drug. You may also be able to continue taking your current DM or ASIM medications for your symptoms during the research study.
The research study will last up to 60 weeks.
Deciding to participate in this, or any research study, is a personal decision. Before making your decision, the study team will review all aspects of the research study with you. If you decide to participate, you will be given a document called an Informed Consent Form that provides, in writing, information on the research study’s purpose, possible benefits and risks, assessments, procedures, and more. No study-related procedures can be done until you sign the Informed Consent Form. You will also have the opportunity to ask the study team questions so you can decide if participating is right for you.
Privacy, Stopping Participation, Costs, and Permission
The information you provide will not be shared with anyone who is not directly associated with the HZNP-DAX-205 DM/ASIM Study without your permission, except as required by law or as set forth in the Informed Consent Form, which is the document you would sign before participating in the research study.
Yes, participation in research studies is always voluntary. If you decide to participate, you are free to withdraw at any time, for any reason, without any penalty or effect on your future medical care. If you do decide to leave the research study early, you should talk to your study doctor about leaving so that they can schedule a final evaluation.
No. However, all study-related care and the study drug will be provided at no cost. You may be reimbursed (paid back) for some of your transportation costs related to the research study. The study team can provide more information about this.
No, your doctor does not have to give you permission to participate. However, you are welcome to talk to your doctor about participating in the HZNP-DAX-205 DM/ASIM Study. With your permission, the study doctor can keep your regular doctor updated about your condition during the research study.
About Research Studies
A research study, also known as a clinical trial, is a scientific study that evaluates the safety and efficacy of a study drug as well as how your body processes that drug. Qualified doctors, nurses, and other medical professionals are responsible for conducting the research study.
It is only through the completion of research studies that study drugs can be evaluated and, if proven safe and efficacious, approved for a particular use. Different regulatory bodies in different countries (such as the US Food and Drug Administration [FDA]) will provide this approval after a thorough review. In order to ensure that a research study is ethical, and that participants’ rights are protected, Institutional Review Boards (IRBs), Ethics Committees (ECs), or the European Union (EU) review the detailed plans and goals for the study.
A study drug is a medication that has not been approved for prescription use by regulatory or health authorities, such as the US Food and Drug Administration (FDA) or European Medicines Agency (EMA).
Only participants who meet all the required eligibility criteria for the research study may take part. The study team will review your medical history and current medical status against the eligibility criteria to determine if you are eligible to take part. You may also be asked to provide information from your medical records to help the study team determine whether or not you may be eligible.
Horizon Therapeutics plc is conducting this research study. Horizon is a biotechnology company headquartered in Dublin, Ireland. Its US headquarters is in Deerfield, Illinois.