A phase 1/2 study of SLN124 in patients with Polycythemia Vera

You likely can't join if

• Drug intolerance: a. History of intolerance to oligonucleotides, or GalNAc, or any component of SLN124. b. History of intolerance to s.c. injections
• Use of any investigational drug less than 6 weeks prior to the first dose of trial drug or not recovered from effects of any prior investigational agent (excludes patients with PV who have previously participated and completed Phase 1 of this protocol).
• Use of any investigational or marketed GalNAc targeting product less than 48 weeks prior to the first dose of trial drug or not recovered from effects of prior administration of any investigational or marketed GalNAc targeting product taken more than 48 weeks before the first dose of the trial drug (excludes patients with PV who have previously participated and completed Phase 1 of this protocol).
• For Phase 2, biochemical and hematological parameters: a. Biochemical evidence of significant liver disease during screening, defined as ALT and/or AST ≥ 3 × ULN; or total bilirubin ≥ 2 × ULN. b. Hematological parameters at screening as follows: platelets > 1,000,000/L; or WBC count > 30,000/L; or peripheral blasts > 1%.
• Co-morbidity: a. Any serious medical or surgical condition, laboratory abnormality, or active psychiatric disorder that, in the opinion of the Investigator, places the patient at significant or unacceptable risk if he/she were to participate in the trial. b. Any known underlying disease that, in the opinion of the Investigator, might confound the ability to interpret data from the trial. c. Infection requiring hospitalization or intravenous antimicrobial therapy, or opportunistic infection within 12 weeks prior to the start of dosing. d. Any infection requiring systemic antimicrobial therapy within 4 weeks prior to first dose. Prophylactic antibiotics are allowed. e. For Phase 1, history of malignancies. Exceptions are resolved local basal cell carcinoma [BCC] and non-melanoma skin tumors that have been resolved no less than 6 months from screening, carcinoma in situ of the cervix, carcinoma in situ of the breast, incidental histological finding of prostate cancer [T1a or T1b using the Tumor, Node, Metastasis staging classification system]. f. For Phase 2, history of malignancies in the last 3 years including those that are currently undergoing active investigations for suspected or recurrence of malignancies before screening. Exceptions are (i) local BCC and non-melanoma skin tumors that have been resolved no less than 6 months from screening, (ii) treated carcinoma in situ of the cervix, (iii) treated carcinoma in situ of the breast, (iv) incidental histological finding of localized prostate cancer [T1a or T1b using the Tumor, Node, Metastasis staging classification system] requiring active surveillance only. g. For Phase 1, significant renal impairment, defined as estimated glomerular filtration rate (using the Chronic Kidney Disease Epidemiology Collaboration equation) < 45 mL/min/1.73m2 at screening. h. h. For Phase 2, significant renal impairment, defined as estimated glomerular filtration rate <30mL/min/1.73m2 at screening (using the Chronic Kidney Disease Epidemiology Collaboration equation) < 30 mL/min/1.73m2 at screening. i. History and/or clinical evidence of cirrhosis. j. Active infectious hepatitis A, B, or C virus. k. Known history or clinical evidence of alcohol and/or drug misuse within 2 years prior to screening. l. Clinically significant or symptomatic cardiac disease including cardiac arrhythmias. m. Clinically significant pulmonary disease.
• For Phase 1, Biochemical and hematological parameters: a. Biochemical evidence of significant liver disease during screening, defined as alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 1.5 × upper limit of normal (ULN); total bilirubin to ≥ ULN; prothrombin time or International Normalized Ratio (INR) ≥ ULN (Isolated INR ≤ 1.3 is acceptable in the absence of other changes in liver function). b. Hematological parameters at screening as follows: platelets > 1,000,000/L; or white blood cell (WBC) count >25,000/L; or peripheral blasts > 1%.