Taking part in a clinical trial
Before a new medication can be approved for public use, researchers must learn more about it through clinical research studies, also referred to as clinical trials. Clinical trials gather valuable information that could lead to better treatment and, with the right breakthrough, better outcomes.
- The goal of this process is to learn whether the study drug works and is safe for people
- Clinical trials are important to the future of medicine
Why participate?
Clinical trials offer opportunities for study participants to partner with researchers to help in the development of new potential treatments for diseases.
The possible benefits from taking part in the HARBOR study include
- Contributing to research that may help others in the future who have indolent systemic mastocytosis or smoldering systemic mastocytosis
- Patient and caregiver may be compensated for time and travel
- Patients will receive study-related care and evaluations with close monitoring by a healthcare professional
Every clinical trial has risks. If you are considering participating in the study, the study staff will discuss specific risks with you before you decide whether to participate.
Before you decide to join a clinical trial, you may want to discuss the study information with your primary healthcare provider, other healthcare professionals, and your family members and friends.
Informed consent
Informed consent is a process designed to help you fully understand what it means to take part in a clinical trial, so you can make an informed decision about whether to take part.
- You will receive an informed consent form, which describes the study in detail, including the purpose of the study, procedures, its possible benefits, risks, and more
- Your study doctor or care team will review the informed consent form with you and be available to answer any questions you may have
- If you decide that you are interested in joining the study after reviewing this information, the study team will ask you to sign the informed consent form
- You can decide to stop participating in the study at any time, for any reason
Frequently asked questions
The purpose of this study is to see if the study drug, called elenestinib can help people who have indolent systemic mastocytosis (ISM). This study will also examine the safety of the study drug and how people feel while taking it.
The study will test the study drug against a placebo. A placebo looks like the study drug, but it has no active ingredients. In addition to the study drug or placebo, participants will continue to receive medication (that they were taking before starting the study) for symptom management.
In this study, researchers want to learn
- whether the study drug is more effective than placebo (along with medication[s] for symptom management) in alleviating symptoms in participants who have ISM
- whether the study drug can reduce how often anaphylactic reactions occur
- whether the study drug can improve bone health
- whether the study drug can improve quality of life
- more about the safety and potential side effects of the study drug
The HARBOR study will also include two exploratory groups that will enable the researchers to learn more about the effects of the study drug in participants who have
- smoldering systemic mastocytosis
- ISM that has been previously treated with a selective KIT inhibitor
Study participants will be randomly assigned (like a coin flip) to receive either the study drug or placebo. Neither the participants nor the study doctor will know to which group the participants have been assigned (this is called a double-blinded study).
During the 48-week double-blind period, participants will have a
- 67% (2 in 3) chance of receiving the study drug and a
- 33% (1 in 3) chance of receiving placebo (a placebo looks like the study drug, but it has no active ingredient)
In addition to the study drug or placebo, participants will continue to receive medication (that they were taking before starting the study) for symptom management.
There may be risks associated with your participation in this study. These risks may be related to the study drug or to some of the procedures or tests performed in this study.
The study drug has been studied in healthy volunteers and people who have systemic mastocytosis and was found to be well tolerated. However, the safety information for this study drug is considered limited, so it may cause side effects that are not known at this time.
As with any investigational treatment, there is a risk that unexpected adverse effects may occur. Almost all medications, both old and new, can cause reactions. Please ask your study doctor or other study staff members for more information regarding the possible side effects.
After the screening visit(s), eligible participants will be enrolled in the double-blind period of the study. During the double-blind period, neither the participants nor study doctor will know to which group the participants have been assigned. The double-blind portion of the study lasts 48 weeks.
Participants who complete the double-blind period will have the option to receive the study drug during an open-label extension period (when all participants receive the study drug, regardless of whether they were initially assigned study drug or placebo). The open-label extension period will continue for approximately 4 years. The total participation time for each person in the HARBOR study is approximately 5 years.
Study participants are required to attend study visits or appointments. The length of time between visits varies throughout the study and may be as often as once a week to once every 12 weeks. As a participant, it is very important for you to attend all study visits and follow the instructions from the study team.
During study visits, participants will undergo certain tests and assessments both before and after starting study drug. Some assessments will be performed to determine whether participants are eligible to enroll in the study; others will be performed to collect data throughout the study. These data are crucial for the study sponsor to evaluate the safety and efficacy of the study drug. Tests and procedures will include physical exams; blood tests; electrocardiograms, or ECGs; urine tests; magnetic resonance imaging, or MRI, scans; questionnaires; and other assessments.
Taking part in this study is voluntary. You can choose to leave the study at any time and for any reason. Choosing to leave the study will not impact your current or future medical care.
There are no costs to participate in the HARBOR study. Participants will not have to pay for the study medicine, study visits, or tests or procedures that are needed as part of this study.
Travel and other study-related expenses, such as meals, parking, and hotels, will generally be reimbursed by the study sponsor. Financial assistance and reimbursement procedures will vary by study site and country. Please speak with a member of your study team for more information.
Helpful links
Where can I learn more about taking part in a clinical trial?
- ClinicalTrials.gov
www.clinicaltrials.gov - National Institutes of Health
www.nih.gov/health/clinicaltrials
Where can I learn more about indolent systemic mastocytosis?
- National Institutes of Health
https://rarediseases.info.nih.gov/diseases/8616/systemic-mastocytosis - National Organization for Rare Disorders
https://rarediseases.org/rare-diseases/mastocytosis/
Thank you for your interest in the HARBOR study!