The purpose of this pilot study is to investigate the safety and feasibility of using daratumumab subcutaneously (under the skin) in patients with moderate to severe ME / CFS. We also want to investigate how daratumumab affects the course of the disease and the symptom picture in ME / CFS.
About the study
pilot This is a study - a small, preparatory study that can be used as a basis for planning larger studies later. Therefore, we will only include up to six participants in the study.
The main purpose is to investigate whether it is safe and feasible to treat patients with moderate to severe ME / CFS with the drug daratumumab. We also want to investigate whether daratumumab affects the course of the disease and the symptom picture in ME / CFS.
Information about participation
The study is open for inclusion from 01.06.2022 until 01.08.2022
Who can participate?
We recruit participants for the study from a database of people who have expressed interest in participating in clinical studies at ME / CFS in Helse Bergen. Participants in previous studies may be considered.
What does the study involve
- Participants must live within affordable travel distance of Haukeland University Hospital.
- To participate in the study, you must be diagnosed with ME / CFS according to the Canada criteria from 2003.
- You must be between 18 and 65 years old, with a duration of illness of at least two years.
- The degree of illness must be from moderate (substantially housebound) to severe (substantially bedridden). In addition, the ME / CFS disease must have started at a defined time, typically after an infection.
- Pregnancy or breast-feeding precludes participation, and effective contraception should be used for 24 weeks after the last injection.
Everyone who is assessed for the study must undergo a medical examination with a clinical examination, interview and blood tests to rule out another disease.
Throughout the study, we regularly ask you to complete a set of questionnaires and to use a Fitbit watch to record your activity level (number of steps per day and resting heart rate). To map your "normal" symptom and activity pattern, we will start with a period of at least 12 weeks without any kind of treatment, but only registration of symptoms and activity.
Treatment is given at the cancer ward's outpatient clinic. We give daratumumab as an injection into the stomach four times at two week intervals. We take blood samples before each treatment, and a doctor or nurse will register if you have experienced side effects since the last time. This is followed by a period of follow-up (nine months from the first treatment) where you will still have to fill in a questionnaire regularly, register activity with a Fitbit watch and attend regular doctor's appointments with blood tests.
Because this study has an additional focus on safety and side effects, patients start treatment in two stages. An independent safety committee conducts an evaluation after the first two patients have completed 12 weeks of registration and four daratumumab treatments. If the treatment of the first two patients does not have unexpected and serious side effects, the last four patients will be able to start treatment.
The total duration of the study from inclusion to the last visit will be between one and a half years.
This study is funded by funds raised by patients and patient associations for research on the treatment of ME / CFS. We do not provide compensation to participants in the study. No deductible shall be paid for visits or treatments at the hospital. Parking is free, and reimbursement can be sought from Patient Travel for travel expenses at regular rates.
Advantages and disadvantages
The advantage of participating in the study is that you can access a treatment that can be shown to improve the symptoms of ME / CFS disease. However, if you get a beneficial effect on the disease picture after daratumumab injections, we do not know how long a response will be. Daratumumab has not previously been tried as a treatment for patients with ME / CFS. We can therefore not know that the drug will have a beneficial effect on patients. There may also be side effects that have not been observed with the treatment of other diseases with daratumumab.
As a participant in the study, you will contribute to increased knowledge about the disease and will thus be able to help other patients in the future. If during this pilot study you should experience a significant improvement in your ME / CFS disease, but later relapse, there is no possibility of new treatment with the antibody daratumumab. If the treatment principle should prove to lead to beneficial effects on the disease picture, we want to apply for funding to be able to carry out a larger study.
A possible side effect is allergic or other reaction after daratumumab injection. Such a reaction rarely occurs after injections under the skin and is usually mild, but a severe reaction can occur with symptoms such as fever, chills, rash and drop in blood pressure.
Therefore, the patients in the study should be pretreated with medication, and closely observed after injection. When daratumumab is injected under the skin of the abdomen, a thin needle is used and daratumumab is inserted within 3-5 minutes. In order for daratumumab to be absorbed by the body, it is combined with another substance called hyaluronidase. Hyaluronidase is an enzyme (egg white substance) that regulates how quickly daratumumab is absorbed by the body. The most common side effects of hyaluronidase are local, usually mild and transient. These may be redness, pain, itching, burning, numbness or rash at the injection site.
In case you need a blood transfusion, tests should always be performed to ensure that the blood you receive is right for you. Daratumumab treatment may affect one of these tests called indirect antiglobulin testing. Therefore, some extra samples will be taken from you before the first treatment with daratumumab. These tests will make it easy to find the right blood for you if you need a blood transfusion despite treatment with daratumumab, for example if you are involved in an accident. You will be provided with a card with information that you have received daratumumab and that this affects blood testing. You should always have this card with you during the period you are being treated with daratumumab and for at least 6 months after stopping treatment.
Because daratumumab affects plasma cells and reduces antibody production, there may be an increased risk of infections. The risk of this is relatively low after only four injections, and blood samples with examination of antibody level are checked in the study. You will also be given medicine to prevent the flare-up of viral infusions.
Study nurse Kari Sørland
Phone: 55 97 04 39
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