2023-504135-40-00招募中3 期
A Phase 3, Parallel-group Treatment, Blinded, Randomized, Placebo-controlled Study to Assess the Efficacy and Safety of Pegtibatinase Administered Subcutaneously in Addition to Standard of Care in Participants with Classical Homocystinuria due to Cystathionine Beta Synthase Deficiency (HARMONY)
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 22
- 试验地点
- 31
研究概览
简要总结
Efficacy: To determine the effect of pegtibatinase administered subcutaneously on plasma tHcy and methionine (Met) as compared with placebo in participants with HCU receiving standard of care Safety: To determine the safety and tolerability of pegtibatinase administered subcutaneously in participants with HCU
入排标准
- 年龄范围
- 0 years 至 65+ years（18-64 Years, 65+ Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Individuals eligible to be enrolled into this study must have a diagnosis of HCU based on clinical, biochemical, and/or molecular genetic testing. Approximately 25% of participants may be enrolled with a Screening plasma tHcy ≥50 to <80 µM, and the remaining majority (approximately 75%) of participants will be enrolled with a Screening plasma tHcy ≥80 µM.
- •Individuals must also be ≥12 to ≤65 years of age at Screening, and be willing to maintain a stable diet with consistent levels of DIPI, and HCU-related therapies such as betaine, pyridoxine, and medical food (eg, metabolic formula), if applicable, as part of pre-study treatment
排除标准
- •Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or disorders of cobalamin metabolism; body weight ≥160 kg (measured from screening through the PND)
- •Any prior exposure to pegtibatinase or pegtarviliase
- •Use or planned use of any injectable drug containing PEG (other than pegtibatinase or PEG containing vaccines)
- •History of a severe immune reaction to a PEG containing product; organ transplant; or intensive chronic immunosuppressive treatment within the 6 months prior to screening
研究组 & 干预措施
Pegtibatinase
Test
干预措施: Pegtibatinase (Drug)
0.9 % sodium chloride saline solution (NaCL)
Placebo
干预措施: 0.9 % sodium chloride saline solution (NaCL) (Drug)
研究者
Travere Call Center
Scientific
Travere Therapeutics Inc.
研究点 (31)
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