A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects witha Urea Cycle Disorder with Inadequate Control on Standard of Care - Kaleido K020-218

Kaleido Biosciences0 个研究点目标入组 29 人开始时间: 2020年10月22日最近更新: 2020年11月3日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方
入组人数: 29

研究概览入排标准研究者记录与标识符相似试验

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

•1. Is able and willing, or has a legally authorized representative (LAR) who is able and willing, to give written informed consent. When the LAR provides informed consent, the subject must also provide written assent if able.
•2. Is willing to comply with specified procedures as stated in the protocol
•3. Has any confirmed UCD other than NAGS deficiency, with the diagnosis confirmed through genetic testing, enzymatic analysis, or analysis of ammonia and amino acid concentrations, as appropriate for the UCD subtype; documentation may be from the historical record
•4. Is male or female, 12 to 70 years of age (inclusive)
•5. If = 18 years old, has a body mass index (BMI) = 20.0 and < 40.0
•kg/m2. If < 18 years old, has a BMI between 5th percentile and 95th
•percentile and weight greater than 5th percentile according to age, sex,
•and regionally appropriate growth chart
•6. Has evidence of poorly controlled disease on the current SOC, as
•evidenced by: 1 plasma ammonia or glutamine concentration > ULN
•within 12 months before Day -1 and at least 1 plasma ammonia
•concentration > 100 µg/dL (71.39 µmol/L) during the Screening Period
•7. If NBT is part of SOC, is on a stable dose and regimen of NBT for at least 4 weeks before Screening and the dose is expected to remain stable during the study
•8. Is willing to maintain a stable diet throughout the course of the study, and is willing to continue usual exercise routine
•9. Is willing to continue taking any current supplements and vitamins (with the exception of prebiotic or probiotic supplements) that the subject is currently taking, for the duration of the study
•10. If taking probiotics or prebiotics, is on a stable dose and regimen for at least 4 weeks before Screening and the dose and regimen are expected to remain stable during the study
•11. Has negative urine screen for drugs of abuse at Screening
•12. If subject is female: subject is surgically sterile or post-menopausal (12 months with no menses without an alternate medical cause); or if subject is a female of childbearing potential (FCBP), must have a negative urine pregnancy test at Screening, must not be lactating, and must agree to abstain from sexual activity or agree to use a highly effective method of contraception for the duration of the study and for 30 days after the last dose of KB195
•13. If subject is male and has a female partner of childbearing potential, must agree to abstain from sexual activity or agree to use a highly effective contraceptive method for the duration of the study and for 90 days after the last dose of KB195
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 4
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 24
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

排除标准

•1. Is at high risk for metabolic decompensation, as indicated by:
•- More than 4 hospitalizations within 1 year before the Screening Visit for management of acute metabolic decompensation due to UCD, OR
•- An ammonia concentration of = 100 µg/dL (71.39 µmol/L) with clinical signs or symptoms of hyperammonemia during Screening (if a subject meets all other study requirements, at the PI’s discretion the subject may be re-screened 1 time after their clinical symptoms have resolved), OR
•- Other evidence of metabolic instability as judged by the PI.
•2. Has had a substantive change (as judged by the PI) in diet or any other aspect of UCD management within 4 weeks before the Screening Visit
•3. Has used a systemic anti-infective (for example, an antibiotic, antifungal, or antiviral product) within 4 weeks before the Screening Visit, or use is anticipated during the study (if a subject fails this criterion and meets all other study requirements, at the PI’s discretion and after consultation with the Sponsor, the subject may be re-screened 1 time)
•4. Has been diagnosed with Citrullinemia Type II
•5. Is receiving any systemically administered immunosuppressant medication on a chronic basis at a dose > 10 mg/day of prednisone or equivalent
•6. Has changed the use or dose of any drug or other compound to modulate GI motility (including stool softeners, laxatives, bismuth, or fiber supplements), within 4 weeks before the Screening Visit, or the use or dose is expected change during the course of the study. Note that proton pump inhibitors and antacids are permitted, provided they are taken at baseline and continued throughout the study. (If a subject fails this criterion and meets all other study requirements, at the PI’s discretion the subject may be re-screened 1 time)
•7. Has a history of or active GI or liver disease that may affect assessment of the efficacy, safety, or tolerability of KB195, including but not limited to the following: chronic diarrhea, malabsorption syndrome, inflammatory bowel disease, irritable bowel syndrome, liver cirrhosis or failure, autoimmune disease, or GI malignancy
•8. Has a clinically significant or uncontrolled concomitant illness that would put the subject at risk or jeopardize the objectives of the study
•9. Has a prior solid organ transplantation including liver transplantation, or is anticipated to receive a liver transplant during study participation
•10. Has used an investigational drug, product, or device within 30 days before the Screening Visit; or is enrolled in another investigational drug, product, or device study within 30 days before the Screening Visit
•11. Has a contraindication, sensitivity, or known allergy to any ingredient of the study drug
•12. Is considered, in the opinion of the PI, to likely be a poor attendee or unlikely for any reason to be able to comply with the study procedures (for example, planned procedures or traveling that would occur during the duration of the study)

研究者

发起方

Kaleido Biosciences

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