PACTR202607461619486进行中（未招募）1 期
First-in-Human (FIH) Phase 1 Clinical Trial of the 3'UTRMYCN M1-14 for MYCN Driven Pediatric and Adult Cancers
UTR Therapeutics Inc0 个研究点目标入组 56 人开始时间: 2027年1月20日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 56
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 Day(s) 至 1 Month(s)（—）
- 性别
- All
入选标准
- •For Childhood and Adolescent Cancers
- •1 6months-12 years, male and female., 13-18yrs male & female.
- •2 Diagnosis of Rhabdomyosarcoma, Neuroblastoma, Retinoblastoma, Wilms tumor, Medulloblastoma, and Acute lymphoblastic leukaemia
- •3 Fresh Tumor tissues or slides or blocks must be available for retrospective analysis.
- •4 Tumor biomarkers:
- •MYCN+ tumor confirmed by IHC and or qPCR
- •MYOD1+/- tumor confirmed by IHC and or qPCR
- •TP53 +/- tumor confirmed by IHC and or qPCR
- •8 Measurable disease by RECIST version 1.1
- •9 Must have received previous treatment with the following medications: Olaparib, actinomycin D, cisplatin, cyclophosphamide, bevacizumab, epirubicin and paclitaxel.
- •10 Adequate bone marrow function:
- •HB (g/dl) HCT (%) MCV (fl) RDW (%) PLT (10^3ml) WBC (10^3/ml)
- •6 months-2 years 11-13.5 31-42 73-85 12.3-15.6 150-350 6-17
- •2-6 years 11-13.7 34-44 75-86 12-14.6 150-350 5-15.5
- •6-12 years 11.2-14.5 35-44 78-90 11.9-13.8 150-350 4.5-13.5
- •12- <18 years female 11.4-14.7 36-46 80-96 11.9-14.6 150-350 4.5-13.5
- •12- <18 years male 12.4-16.4 40-51 80-96 11.9-13.7 150-350 4.5-13.5
- •> 18 years female 12-14 36-41 80-90 150-350 4.5-11
- •> 18 years male 13.5-15.5 41-47 80-90 150-350 4.5-11
- •11 Adequate cardiac function: left ventricular ejection fraction (LVEF) ? 55%, fractional shortening fraction (FS) ? 28%
- •12 Adequate neurologic function: In patients ? 16 years, ECOG/WHO performance status < 2 or Karnofsky performance ?
- •< 16 years, Lansky performance score > 50%.
- •13 No history of autoimmune disorder.
- •14 No history of coagulation or platelet disorders.
- •15 Able to receive medication intravenously.
- •16 Not pregnant or trying to conceive.
- •17 Not breastfeeding.
- •Ability to give to informed consent or assent to consent.
- •Rhabdomyosarcoma:
- •Histological diagnosis of rhabdomyosarcoma (embryonal or alveolar)
- •Past history or not of Surgery
- •Past history or not of radiation therapy
- •Past history of chemotherapy: Vincristine, Actinomycin D, Cyclophosphamide treatment
- •Inclusion Criteria for Adults:
- •Adult patients (>18 years)
- •Biopsy-confirmed diagnosis of , neuroendocrine prostate cancer, Small Cell Lung Cancer, Glioblastoma multiforme, Spinal Ependymoma, T-cell acute lymphoblastic leukaemia, high grade serous ovarian cancer, basal cell carcinoma, Sarcomas, triple negative breast cancer (TNBC).
- •MYCN positivity (+, ++, +++) tested by histology at trial enrolment, or history of prior histologic diagnosis.
- •treatment for their cancer type with incurable advanced disease.
- •Performance status of 0 or 1 on Eastern Cooperative Oncology Group (ECOG)
- •Able to provide written informed consent at the time of study enrolment.
- •Participants who are physically able to become pregnant must use an effective form of birth control from 14 days prior to enrollment through 6 months following the last dose of 3’UTRMYCN M1-
- •Participants who can father a child must use an effective form of birth control from Day 0 through 3 months following the last dose of 3’UTRMYCN M1-
- •Laboratory values (Hematology): Absolute neutrophil count = 1,000 cells/mm3; Platelet count = 75,000 cells/mm3; Hemoglobin = 8.0 g/dL.
- •Laboratory values (Renal): Serum creatinine < 1.5 × upper limit of normal (ULN) or creatinine clearance
- •= 40 mL/min based on the Cockcroft-Gault glomerular filtration rate estimation
- •Laboratory values (Coagulation): Prothrombin/International Normalized Ratio (PT/INR) or prothrombin ti
排除标准
- •1 Abnormal cardiac function: left ventricular ejection fraction (LVEF) < 55%, fractional shortening fraction (FS) < 28%.
- •2 Uncontrolled cardiac arrhythmia.
- •3 QTc prolongation on ECG.
- •4 Abnormal neurologic function: In patients ? 16 years, ECOG/WHO performance status > 2 or Karnofsky performance <
- •In patients < 16 years, Lansky performance score < 50%.
- •5 History of cerebrovascular accident.
- •6 Evidence of brain metastasis.
- •7 History of coagulopathy or platelet disorders.
- •7 History of autoimmune disorder.
- •9 Pregnant or breastfeeding.
- •10 Trying to conceive/refusing contraceptive use during the study period (in sexually active patients).
- •Exclusion Criteria for Adults:
- •Previous mRNA drug treatment or currently being treated with other investigational agents.
- •Liver dysfunction or failure, renal failure, or severe cardio-respiratory disease.
- •Baseline reticulocytosis or monocytosis (>2xULN).
- •Significant medical illnesses that in the investigator’s opinion cannot be adequately controlled or would compromise the patient’s ability to tolerate therapy (e.g., history of allergic reactions attributed to compounds of similar chemical or biologic composition to 3’UTRMYCN M1-14).
- •Any history of other malignancies, unless in complete remission and off all therapy for that disease for a minimum of 3 years.
- •History of thromboembolic disorder or deep vein thrombosis, unless resolved and off therapeutic anticoagulation.
- •Presence of abnormal hematological or biochemical parameters as defined in this protocol (e.g., anemia, thrombocytopenia).
- •Active infection currently being treated with systemic antibiotics.
- •Pregnancy or lactation.
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