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NADPH Oxidase Correction in mRNA-transfected Granulocyte-enriched Cells in Chronic Granulomatous Disease (CGD)
Bu çalışma, kronik granülomatöz hastalık (chronic granulomatous disease, CGD) olan hastalarda, bağışıklık hücrelerine mRNA aktarılarak eksik olan NADPH oksidaz işlevinin düzeltildiği gp91 Grans adlı deneysel hücre tedavisini incelemektedir. Çalışmaya, 18-75 yaş arasında, CGD tanısı doğrulanmış erkek hastalar alınmaktadır. Çalışma Faz 1 aşamasında olup halen katılımcı almaktadır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
* INCLUSION CRITERIA: Individuals must meet all of the following criteria to be eligible for study participation: * Males aged 18 to 75 years * CGD confirmed by DHR and deficiency subtype confirmed by protein analysis and/or genetic sequencing * Has a physician at home for follow-up care * Able to provide informed consent * For men who engage in activities that can result in pregnancy, agree to use contraception when engaging in sexual activities that can result in pregnancy. Contraception must be used from screening through 3 months after the CGD-Grans infusion. Acceptable methods of contraception include the following: * Hormonal contraception * Male or female condom EXCLUSION CRITERIA: Individuals meeting any of the following criteria will be excluded from study participation: * Clinically unstable due to moderate to severe acute systemic infections as defined by persistent resting tachypnea, tachycardia, or hypoxia of \>20% from baseline and hypotension. * Current or history of stage 4 chronic kidney disease or estimated glomerular filtration rate \[eGFR\] \<30 mL/min/1.73 m\^2 within 90 days of baseline. * Unstable diabetes mellitus with hemoglobin A1c \>7.0% and fasting serum glucose \>200 mg/dL at screening. * Current or history of heart failure stage D as defined by the American College of Cardiology Foundation/American Heart Association guidelines. * History of arrhythmias that are symptomatic and deemed clinically unsafe for participation by NIH CC Cardiology consultation. * Current or history of invasive cancers that require chemotherapy within 5 years of screening. * Active hepatitis B, C, or HIV infections at screening. * Unstable hypertension requiring addition of new anti-hypertensives within 2 weeks of screening. * Impaired renal function that is unstable, with serum creatinine \>3.0 mg/dL and rising. * Serum transaminases and bilirubin that are \>3 x the upper limit of normal. NOTE: For prospective subjects who, per PI assessment at screening, have abnormal liver function tests, and/or a significant history of liver disease, and/or liver-related complications of CGD, and who otherwise meet eligibility criteria \[i.e. those who do NOT meet any of the exclusion set forth herein\], a hepatology consult will be required at screening, and participation must be approved in writing by hepatology to the PI. * Electrocardiogram abnormalities indicative of acute myocardial injury, or arrhythmias that presents anesthetic risks, at screening. * Anemia with hemoglobin \<8 g/dL (transfusions to correct anemia permitted). * Thrombocytopenia (platelets \<50 x10\^9 cells/L) (platelet transfusions to correct thrombocytopenia permitted). * Profound thrombocytopenia (platelet counts \<10,000/microliter) that is not reversible with platelet transfusions. * Abnormal prothrombin time/partial thromboplastin time (PT/PTT) values outside the ranges accepted at the NIH CC that are not corrected or that cannot be attributed to presence of Lupus anticoagulant (commonly found in CGD patients). * Inherited bleeding disorder that precludes line placement. * Severe oxygen-dependent pulmonary disease that increases risks of procedures that may require sedation. * History of or current evidence of alcohol or illicit drug abuse or dependence. * Participation in a clinical protocol that includes an intervention that, in the opinion of the investigator, may affect the results of the current study. Subjects will be selected in an equitable manner from the available pool of potentially eligible individuals, without regard to factors such as gender, race, ethnicity, socioeconomic status, etc, except for age and sex.