NCT06079281进行中（未招募）3 期
A Phase 3, Randomized, Double-blinded, Placebo-controlled, Multicenter Study to Evaluate Efficacy and Safety of ALXN1850 (Recombinant Alkaline Phosphatase) Administered Subcutaneously in Adolescent (12 to < 18 Years of Age) and Adult Participants With Hypophosphatasia Who Have Not Previously Been Treated With Asfotase Alfa
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 124
- 试验地点
- 66
- 主要终点
- Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169)
研究概览
简要总结
The primary objective of this study is to assess the efficacy of ALXN1850 versus placebo on functional outcomes in adolescent and adult participants with HPP who have not previously been treated with asfotase alfa.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 130 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of HPP documented in the medical records
- •Must meet 1 of the following criteria:
- •Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified laboratory (Section 8.7 )
- •Plasma PLP above the upper limit of normal (ULN) during the Screening Period (central or local laboratory results allowed per local regulations)
- •Must meet 1 of the following criteria without a probably cause other than HPP:
- •Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory
- •Two documented serum ALP activity results, at least 15 days apart, below the age- and sex-adjusted local laboratory normal range during the 24 months before the Day 1 Visit. Note: Local laboratories need to be CLIA or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval.
- •Two separate 6MWTs at below 85% of the predicted distance (for age, sex, weight, and height) during the Screening Period without a probable cause other than HPP
排除标准
- •History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, neurological disorders, or any other disorders that are capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study intervention; or interfering with the interpretation of data as determined by the Investigator
- •Diagnosis of primary or secondary hyperparathyroidism
- •Hypoparathyroidism, unless secondary to HPP
- •Any new fracture within 12 weeks before Day 1 (excluding pseudofractures)
- •Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period
- •History of allergy or hypersensitivity to any ingredient contained in ALXN1850 or the placebo comparator (Table 9)
研究组 & 干预措施
Placebo Group
Placebo Comparator
During the Randomized Evaluation Period, the placebo group will receive placebo on Day 1, followed by once every 2 weeks (q2w) via SC injection for 24 weeks. Participants will enter the OLE Period and receive bodyweight dependent doses of either 20mg, 35mg, or 50mg of ALXN1850 and continue q2w dosing with ALXN1850 for up to 132 weeks.
干预措施: Placebo (Drug)
ALXN1850 Group
Experimental
Starting at Day 1 of the Randomized Evaluation Period, the ALXN1850 group will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once q2w via SC injection, for 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.
干预措施: Placebo (Drug)
ALXN1850 Group
Experimental
Starting at Day 1 of the Randomized Evaluation Period, the ALXN1850 group will receive bodyweight dependent doses of either 20mg, 35mg or 50mg of ALXN1850 once q2w via SC injection, for 24 weeks. Participants will enter the OLE Period and continue q2w dosing with ALXN1850 for up to 132 weeks.
干预措施: ALXN1850 (Drug)
结局指标
主要结局
Change from Baseline in 6-Minute Walk Test (6MWT) at the end of the Randomized Evaluation Period (Day 169)
时间窗: Baseline, Day 169
次要结局
- Change from Baseline in 30-second Sit to Stand (STS) Test Score at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Change from Baseline in Lower Extremity Functional Scale (LEFS) Score at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Change from Baseline in BPI-SF pain severity score at the end of the Randomized Evaluation Period (Day 169) (adult cohort only)(Baseline, Day 169)
- Change from Baseline in FACIT-Fatigue score at the end of the Randomized Evaluation Period (Day 169) (adult cohort only)(Baseline, Day 169)
- Change from Baseline in Timed Up-and-Go (TUG) at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Change from Baseline in Percent Predicted 6MWT at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
- Radiographic Global Impression of Change (RGI-C) Score at the end of the Randomized Evaluation Period (Day 169)(Day 169)
- RGI-C Responder at the end of the Randomized Evaluation Period (Day 169)(Day 169)
- Change from Baseline in Rickets Severity Score (RSS) at the end of the Randomized Evaluation Period (Day 169)(Baseline, Day 169)
研究者
研究点 (66)
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相关资讯
FDA Grants Priority Review to Alexion's Efzimfotase Alfa BLA for Hypophosphatasia- The FDA accepted and granted Priority Review to Alexion's Biologics License Application for efzimfotase alfa in hypophosphatasia patients aged two years and older.
- The PDUFA action date is anticipated in the first half of 2027, with regulatory submissions also under review in Japan and other markets.
- The filing rests on the three-trial Phase III programme HICKORY, MULBERRY and CHESTNUT, which enrolled 196 patients across 22 countries.
- MULBERRY met its primary radiographic endpoint with a median RGI-C difference of 1.67 versus placebo, while HICKORY missed statistical significance on the six-minute walk test.11 hours agoAstraZeneca's Efzimfotase Alfa Shows Promise in Phase III Hypophosphatasia Trials with Mixed Results- Efzimfotase alfa met its primary endpoint in pediatric patients with hypophosphatasia, demonstrating statistically significant bone health improvements in the MULBERRY trial.
- The investigational therapy failed to achieve statistical significance in its primary endpoint for adolescents and adults in the HICKORY trial, though it showed benefits in specific subgroups.
- The drug offers potential advantages over current treatment with less frequent dosing every two weeks and maintained efficacy when patients switched from existing therapy.5 months ago