For your patients who are living with indolent systemic mastocytosis
The HARBOR Study
The HARBOR study is researching whether the investigational drug elenestinib is safe and effective in treating indolent systemic mastocytosis (ISM) in adults.
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 anaphylaxis occurs
- 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
Please consider encouraging your eligible patients to participate in the phase 2/3 HARBOR study of elenestinib.
Please visit harborclinicaltrial.com to refer your eligible patients. You can learn more about the HARBOR study at clinicaltrials.gov by searching for NCT04910685. For more information, please email us at medinfo@blueprintmedicines.com or call us at 1-888-BLUPRNT.
Thank you for your interest in the HARBOR study.
We appreciate your support in helping us learn how we can improve health outcomes in patients who are living with ISM or smoldering systemic mastocytosis.
The HARBOR study will be conducted at study sites around the world, including North America, Europe, and Australia. The study is expected to enroll approximately 350 participants who have indolent systemic mastocytosis (ISM) for a 48-week placebo-controlled treatment period, followed by an open-label extension period in which all participants will receive elenestinib. The open-label extension period will continue for at least 4 years. The total time for the HARBOR study is approximately 5 years.
- 75 sites around the world
- Approximately 350 participants
- 48-week treatment period
- Open-label extension
- Total study time is approximately 5 years
- Additional exploratory cohorts (open-label, for up to 5 years)
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- Approximately 20 participants who have smoldering systemic mastocytosis
- Approximately 40 participants who have ISM and have been previously treated with a selective KIT inhibitor
Study objectives
Primary objective and endpoint
The HARBOR study will determine whether elenestinib and symptom-directed therapy (SDT) are more efficacious than placebo and SDT in reducing the total symptom score on the Indolent Systemic Mastocytosis-Symptom Assessment Form, based on the mean change from baseline to 48 weeks.
Key secondary objectives
To determine whether elenestinib and SDT are more efficacious than placebo and SDT in
- reducing tryptase levels
- reducing KIT D816V variant allele frequency
- achieving symptom control, as evidenced by mild or absent symptoms of ISM, in participants who previously had moderate-to-severe symptoms
- improving bone mineral density
- decreasing the frequency of anaphylactic events
- improving quality of life
Safety objective
To evaluate the safety and tolerability of elenestinib as determined by adverse events, serious adverse events, and changes in safety laboratory parameters, vital signs, and electrocardiogram evaluations.
To be eligible for the study, patients must
- be at least 18 years of age at the time of signing the informed consent form
- be able to give written informed consent
- have an Eastern Cooperative Oncology Group Performance Status of 0 to 2
- have a centrally confirmed diagnosis of indolent systemic mastocytosis (ISM)
- have inadequate ISM symptom control on at least 2 symptom-directed therapies
- have moderate-to-severe symptoms based on the Indolent Systemic Mastocytosis-Symptom Assessment Form total symptom score
* Additional inclusion criteria apply.
For more information, contact Blueprint Medicines.
Patients who meet any of the following criteria will be ineligible to participate in the study:
- A diagnosis of other systemic mastocytosis (SM) subclassifications, including cutaneous mastocytosis only, SM with an associated hematologic neoplasm of non–mast cell (MC) lineage, aggressive SM, MC leukemia, MC sarcoma
- A diagnosis of another myeloproliferative disorder
- Organ damage attributable to SM
- Clinically significant, uncontrolled, cardiovascular disease
- Previous treatment with a targeted KIT inhibitor (except for of one of the exploratory cohorts)
- Treatment (before the first dose of study drug) with radiotherapy or psoralen and ultraviolet A therapy or any hematopoietic growth factor less than 14 days before screening
* Additional exclusion criteria apply.
For more information, contact Blueprint Medicines.
Find a study site
Thank you for your interest in the HARBOR study!