EUCTR2020-005173-28-PT进行中（未招募）1 期
A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis - HARBOR
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 403
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •All Patients
- •-1. Patient must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2.
- •Part 1 only
- •-2. 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.
- •Part 1 and Part 2
- •3. 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.
- •4. Patient must have failed to achieve adequate symptom control for 1 or more Baseline symptoms, as determined by the Investigator, with at least
- •2 of the following symptomatic therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium,
- •corticosteroids, or omalizumab.
- •5. Patients must have BSC for ISM symptom management stabilized for at least 14 days prior to starting screening procedures.
- •6. For patients receiving corticosteroids, the dose must be = 20 mg/d prednisone or equivalent, and the dose must be stable for = 14 days.
- •7. Patients must have mMCAS, confirmed by Central Pathology Review of BM biopsy. An archival biopsy may be used if completed within the past 12
- •8. Patients must have tryptase < 20 ng/mL.
- •9. Patients must have KIT D816V in peripheral blood (PB) or BM and/or CD25+ Mast cells in BM.
- •10. Patients must have symptoms consistent with mast cell activation (despite BSC) in at least two organ systems characterized by cutaneous
- •flushing, tachycardia, syncope, hypotension, diarrhea, nausea, vomiting and gastro-intestinal cramping) and serum blood tryptase (sBT) levels
- •above 8 ng/mL OR Severe (Ring and Messmer grading = II, recurrent anaphylaxis, including but not limited to hymenoptera venom, drug or food,
- •regardless of sBT levels.
- •Optional PK Group
- •11. See inclusion criteria for All patients and Part 1/Part 2
- •12. Accrual may be limited to patients who have specific disease manifestations (ie, GI involvement) or are taking acid-reducing agents to better explore the impact of these features on PK.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 3
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 383
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 17
排除标准
- •Key Exclusion Criteria:
- •1. Patient has been diagnosed with any of the following WHO systemic mastocytosis (SM) sub-classifications: cutaneous mastocytosis only,
- •smoldering SM, SM with associated hematologic neoplasm, aggressive SM, mast cell leukemia, or mast cell sarcoma.
- •2. Patient has been diagnosed with another myeloproliferative disorder.
- •3. Patient has organ damage C-findings attributable to SM.
- •4. Patient has clinically significant, uncontrolled, cardiovascular disease
- •5. Patient has a QT interval corrected using Fridericia's formula (QTcF) > 480 msec.
- •6. Patient has previously received treatment with any targeted KIT inhibitors.
- •7. 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.
- •8. 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 period.
- •9.Patient has received radiotherapy or psoralen and ultraviolet A (PUVA) therapy < 14 days before beginning the screening period.
研究者
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