A Phase 1/2 Multiple Ascending-Dose Study in Subjects With Homocystinuria Due to Cystathionine ß-Synthase (CBS) Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of ACN00177 - SA notification 100D New site and PI

Aeglea Biotherapeutics, Inc.0 个研究点目标入组 25 人开始时间: 2020年1月14日最近更新: 2020年9月28日

适应症

试验速览

暂无简介。

•1. Diagnosis of homocystinuria due to CBS deficiency
•2. Capable of providing signed informed consent/assent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol
•3. Is =12 years of age at the time of signing the informed consent/assent
•4. Plasma tHcy >80 µM
•5. Female subjects of child-bearing potential must have a negative serum pregnancy test during the screening period and a negative urine pregnancy test prior to dosing on the first day of treatment
•6. If the subject (male or female) is engaging in sexual activity, he/she must be unable to become pregnant/cause pregnancy or must agree to use highly effective contraception as specified in the full protocol
•7. Subjects receiving pyridoxine and/or betaine must be on a stable dose of the medication(s) for at least 6 weeks prior to the first administration of study drug and be willing and able to remain on a stable dose for the duration of the study
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 13
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 13
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•1. Other medical conditions or co-morbidity(ies) that, in the opinion of the investigator, would interfere with study compliance or data interpretation (eg, severe intellectual disability that precludes completion of the required study assessments)
•2.Currently participating in another therapeutic clinical study or has received any investigational agent within 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug in this study
•Surgery requiring general anesthesia within the 8 weeks prior to first dose of study drug
•4. Active infection requiring anti-infective therapy <2 weeks prior to the first dose of study drug in this study; anti-infective therapy that completes =2 weeks prior to first dose of study drug is acceptable
•5. Pregnant or nursing
•6. Females of child-bearing potential who are using or plan to use estrogen-containing contraception during the study
•7.History of hypersensitivity to polyethylene glycol (PEG) that, in the judgment of the investigator, puts the subject at unacceptable risk for adverse events (AEs)

发起方