Stuttering, formally known as childhood onset fluency disorder or COFD, can affect every aspect of a person’s life. The frustration of knowing exactly what you want to say but being physically unable to say it can lead to feelings of stress, anxiety, depression, shame, and embarrassment. You may blame yourself and become isolated, distancing yourself from friends and family.
If your treating physician has signposted you to this webpage, they believe you are eligible to take part in a clinical trial about stuttering, called the ORPHEUS study. This website has been designed to help you understand more about the study.
If you landed on this page from a web search, the ORPHEUS Study is a clinical trial looking at whether an investigational medicine, or ‘active medicine’, can improve speech fluency in men aged 18-55 whose stuttering started as a child.
If you have any questions, you will find the contact details of the study team on this website.
What is stuttering?
Stuttering, more formally known as childhood onset fluency disorder or COFD, is a speech problem which affects a person’s ‘speech fluency’. This means that people who stutter often have significant interruptions in the flow of their words when they are talking.
Most people can have interruptions in their speech or struggle to get their words out from time to time. For people who stutter, this difficulty with speech fluency doesn’t go away and is a lifelong condition. Stuttering may cause feelings of stress, anxiety, depression, shame, and embarrassment.
Stuttering can affect people differently: it commonly starts as difficulty beginning a word, but it can also start as repeating a sound or a word, lengthening sounds in a word, pausing between words, or pausing in the middle of a word. The condition is often accompanied with physical tension, such as tightening your jaw, feeling short of breath, clenching your fists.
Although there are currently no approved medicines to treat stuttering, many people undergo speech therapy to help improve their symptoms.
What is a clinical trial?
Clinical trials help the medical community discover possible ways to help treat a range of conditions, diseases, and disorders.
They are designed to help answer important questions about investigational medicines or ‘active medicines’, such as:
- How does this medicine work?
- How will it affect certain conditions, diseases, or disorders?
- Is this medicine safe and well-tolerated at various doses?
To find the answers to questions like these, we ask for the help of people like you to take part in clinical trials.
Why join a clinical trial?
Thousands of volunteers all around the world take part in clinical trials every year, and they decide to do it for many different reasons. For every medicine on a pharmacy shelf, volunteers like you have taken part in clinical trials and the data collected has helped make that medicine available for people to take.
Taking part in a clinical trial like ORPHEUS may help to improve medical knowledge about stuttering and has the potential to improve the quality of life for millions of people who stutter worldwide.
About the ORPHEUS study
Scientists believe that stuttering may be caused by having too much of a chemical, called dopamine, in the brain. So, it is thought that medicines which lower the amount of dopamine in the brain can help reduce stuttering. The ORPHEUS study is trying to find out if an investigational medicine, or ‘active medicine’, may help to stabilize the levels of dopamine in your brain and can help to improve speech fluency in men aged 18-55 whose stuttering started as a child.
The ORPHEUS study has been designed to understand more about how safe and tolerable the active medicine is. It has previously been tested in clinical trials for other medical conditions.
- The active medicine will be compared to a placebo. A placebo looks like the active medicine but does not contain any active ingredients. The active medicine and placebo are referred to collectively as the ‘study medicine’. The study medicines are taken as capsules, once a day by mouth.
- There is a 50/50 chance of receiving either the active medicine or the placebo, and neither you nor the study team will know which study medicine you take during the study.
What does taking part involve?
The ORPHEUS study will take around 18 weeks for you to complete. During this time, you will need to visit the study site about 12 times. There will be 2 additional visits carried out remotely.
We will ask you to take the study medicine capsules once a day, in the morning, by mouth with food. You will receive a supply of the capsules to take at home between study visits.
You will be seen by a study doctor with experience in managing stuttering in adults, and your health will be monitored closely. During most study visits, you will be asked to answer a number of questionnaires about your stuttering, as well as about your physical and mental wellbeing. Some study visits will also involve physical and neurological examinations. A physical examination ensures you are in good health by monitoring vitals like your weight and blood pressure, while a neurological examination checks how your nervous system is functioning.
What medicine will be tested?
The ‘active medicine’ is what’s known as an investigational medicine. Investigational medicines are designed to help treat a condition, but they have not yet been through all the necessary stages of medicines development.
Investigational medicines cannot be approved for use until clinical trials, like the ORPHEUS study, have collected enough data to show that it is safe and effective.
The active medicine in the ORPHEUS study has not been approved to treat people who stutter.
Frequently Asked Questions
Why do we need clinical trials?
Clinical trials are important to understand more about whether a potential treatment may be better than current treatments. Clinical trials also help us learn more about the side effects of a potential treatment. Thousands of people all around the world take part in clinical trials every year. The medicines we buy at the pharmacy or at the doctors have all been tested in clinical trials before.
Is taking part in a clinical trial voluntary?
Yes, taking part in any clinical trial is completely voluntary. If you decide to stop taking part at any time, it will not affect your future medical care in any way.
What is informed consent?
Before you can join any clinical trial, you need to agree to join by giving your consent. You must understand everything about the study before you give your consent. This is called being ‘informed’. You should not feel any pressure to give your consent. Feel free to discuss the study with family and friends before you make your decision.
What is the active medicine in the ORPHEUS study?
In this study we are comparing an active medicine with a placebo. The active medicine is an investigational medicine. The placebo looks identical to the active medicine but does not contain the active ingredient.
How does the active medicine work?
Scientists believe that stuttering may be caused by having too much of a chemical, called dopamine, in the brain. So, it is thought that medicines which lower the amount of dopamine in the brain can help reduce stuttering. The ingredient in the active medicine may help to stabilize the levels of dopamine in your brain and may help you to control your stutter.
How will I receive the study medicine?
You will receive the study medicine from the study team. The study medicine will be in capsule form. You will need to take two capsules once per day, in the morning, with food.
How many visits would I have to make to the study site?
You would need to make approximately 12 visits to your chosen study site, and 2 remote visits, over a period of around 18 weeks.
Are there any risks involved in joining the study?
There are some risks linked to taking any medicine, and the investigational medicine on the ORPHEUS study is no different. You may experience side effects, or find that your symptoms do not improve or get worse. It is important that you tell your study doctor if you are worried about anything or if anything about your health changes.
What happens after the study ends?
You will no longer receive the study medicine after your part in the study has ended, even if you found it helpful. For any medical care you can contact your usual treating physician, as you did before the study.
All of the data collected from each volunteer is anonymised and then analyzed by a team of scientists, who then produce a report with the findings for the professional bodies that oversee the development of investigational medicines. You cannot be identified in this report and your name will not appear in any results published.
What happens if I change my mind about taking part?
You are free to leave the study at any time without giving a reason. It will not affect your regular medical care.
What happens to my personal information?
Information about your personal health will be kept private. If you decide to contact us, your details will be transferred to the study team at the site you’ve chosen. Someone from the study team will contact you to better understand if the study is right for you. Your personal information will not be revealed to anyone outside of this study team without your consent unless required by law or regulations of the local regulatory agency in your country.