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HARBOR: Study of BLU-263 for Indolent Systemic Mastocytosis
EU CTIS ID: 2024-516728-32-00
What this study is testing
(Part 1) To determine the recommended dose(s) of elenestinib (Part 2) To assess if treatment with elenestinib + SDT reduces total symptom score (TSS) compared to placebo + SDT, as assessed using the ISM-SAF (Part 3) - To assess the safety and tolerability of treatment with elenestinib. - To assess the efficacy of treatment with elenestinib.
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- All patients. 1. Patient must be ≥ 18 years of age at the time of signing the informed consent/assent.
- Patients in Part K Previously Treated With Approved Selective KIT Inhibitors: 12. Patient has a centrally confirmed diagnosis of ISM. In consultation with the Sponsor, archival biopsy may be used if completed within the past 12 months and there is no evidence of progressive disease.
- 13. Patients with 14-day average TSS obtained no earlier than 15 days after the discontinuation of the prior approved selective KIT inhibitor may be enrolled.
- Patients with ISM in Part 1, 2 and PK groups. 4. Patient has confirmed diagnosis of ISM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria. Archival biopsy may be used if completed within the past 12 months.
- 2. Patient must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2. With ISM in Part 1 and Part 2
- Patients with ISM in Part 1. 3. Patient must have moderate-to-severe symptoms based on minimum mean total symptom score (TSS) of the ISM Symptom Assessment Form (ISM-SAF) over the 14-day eligibility screening period. With ISM in Part 1, Part 2 and PK groups
You likely can't join if
- Patient has been diagnosed with any of the following WHO SM sub-classifications: cutaneous mastocytosis only, SM-AHN, ASM, MCL, Mast cell sarcoma.
- Patient has been diagnosed with another myeloproliferative disorder.
- Patient has organ damage attributable to SM.
- Patient has a QT interval corrected using Fridericia's formula (QTcF) > 470 msec (for females) or > 450 msec (for males).
- Patient has clinically significant, uncontrolled, cardiovascular disease.
- Patient has a history of a primary malignancy that has been diagnosed or required therapy within 3 years. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.
See the full eligibility criteria
Who can join
- All patients. 1. Patient must be ≥ 18 years of age at the time of signing the informed consent/assent.
- Patients in Part K Previously Treated With Approved Selective KIT Inhibitors: 12. Patient has a centrally confirmed diagnosis of ISM. In consultation with the Sponsor, archival biopsy may be used if completed within the past 12 months and there is no evidence of progressive disease.
- 13. Patients with 14-day average TSS obtained no earlier than 15 days after the discontinuation of the prior approved selective KIT inhibitor may be enrolled.
- Patients with ISM in Part 1, 2 and PK groups. 4. Patient has confirmed diagnosis of ISM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria. Archival biopsy may be used if completed within the past 12 months.
- 2. Patient must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2. With ISM in Part 1 and Part 2
- Patients with ISM in Part 1. 3. Patient must have moderate-to-severe symptoms based on minimum mean total symptom score (TSS) of the ISM Symptom Assessment Form (ISM-SAF) over the 14-day eligibility screening period. With ISM in Part 1, Part 2 and PK groups
- 5. Patient must have failed to achieve adequate symptom control for 1 or more Baseline symptoms, as determined by the Investigators best clinical judgment, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab.
- 6. Patients must have SDT for ISM symptom management stabilized without any new or worsening of symptoms and/or AEs for at least 14 days prior to starting 14-day ISM-SAF TSS eligibility period (Part 1 only).
- Patients in Part 2: 17. Patient has a centrally confirmed diagnosis of ISM. Archival biopsy may be used if completed within the past 12 months.
- 7. For patients receiving corticosteroids, the dose must be ≤ 20 mg/d prednisone or equivalent, and the dose must be stable for ≥ 14 days before beginning the 14-day eligibility Screening Period for assessment of ISM-SAF for Part 1 or PK.
- 8. The patient’s ISM symptom-directed therapies (eg, H1 and H2 blockers) must be stable (same dose, no new medications ≥ 14 days before beginning the 14-day eligibility Screening Period for the assessment of ISM-SAF).
- 15. Treatment with a prior approved selective KIT inhibitor for ISM.
- 14. Patients must have a washout period of their prior approved selective KIT inhibitor for ISM of at least 28 days prior to the first elenestinib dose.
- 19. Patient must have failed to achieve adequate symptom control for 1 or more Baseline symptoms as determined by the Investigator’s best clinical judgment, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab.
- 18. Patient must have moderate to severe symptoms during the eligibility period.
- 21. If the patient is receiving corticosteroids, the dose must be ≤ 20 mg/day prednisone or equivalent, and the dose must be stable for ≥ 14 days before beginning the 14-day eligibility Screening Period for assessment of ISM-SAF.
- 22. If on a bone-targeted therapy other than calcium and vitamin D, including hormone replacement therapy, patients must be on a stable dose for least 24 weeks prior to first treatment day unless the administration frequency of the drug is less frequent than once every 24 weeks for which at least 2 consecutive doses must have been administrated to the patient following the schedule.
- 10. Accrual may be limited to patients who have specific disease manifestations (ie, GI involvement) to better explore the impact of these features on PK.
- 20. The patient’s ISM symptom-directed therapies (e.g., H1 and H2 blockers) must be stable (same dose, no new medications ≥ 14 days before beginning the 14-day eligibility Screening Period for the assessment of ISM-SAF).
- Patients with SSM in Part S: 11.Patient has confirmed diagnosis of SSM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria . No archival BM biopsies will be accepted without approval from the Sponsor.
What rules you out
- Patient has been diagnosed with any of the following WHO SM sub-classifications: cutaneous mastocytosis only, SM-AHN, ASM, MCL, Mast cell sarcoma.
- Patient has been diagnosed with another myeloproliferative disorder.
- Patient has organ damage attributable to SM.
- Patient has a QT interval corrected using Fridericia's formula (QTcF) > 470 msec (for females) or > 450 msec (for males).
- Patient has clinically significant, uncontrolled, cardiovascular disease.
- Patient has a history of a primary malignancy that has been diagnosed or required therapy within 3 years. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.
- Time since any cytoreductive therapy including mastinib and midostaurin should be at least 5 half-lives or 14 days (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy < 28 days or 5 half-lives of the drug (whichever is longer), before beginning the screening assessments. For omalizumab, washout is not necessary, and patients can continue on omalizumab therapy.
- Patient has received radiotherapy or PUVA therapy < 14 days before beginning the screening assessments.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Switzerland
- Australia
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Switzerland; Australia; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.