EUCTR2015-003131-35-DE进行中（未招募）1 期
A Phase 2a Randomized, Multi-center, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients with Pediatric-Onset Hypophosphatasia
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 36
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
- •Patients must meet all of the following criteria for enrollment in this study:
- •1. Patients or their legal representative(s) must provide written informed consent prior to undergoing any study-related procedures
- •2. Patient has pediatric-onset HPP, defined as onset of first sign(s)/symptom(s) of HPP prior to 18 years of age
- •3. Documented diagnosis of HPP as indicated by a documented history of HPP-related skeletal abnormalities and 1 or more of the following:
- •- Documented TNSALP gene mutation(s) from a certified laboratory
- •- Serum ALP level below the age-adjusted normal range AND plasma PLP above the upper limit of normal at Screening.
- •4. Patients must have a plasma PPi level of =3.9 µM at Screening
- •5. Patients must be =18 years of age at Screening
- •6. Sexually active male and female patients of childbearing potential must agree to use a highly effective method of birth control during the study and for 3 months following the last dose of study drug. Male patients must also not donate sperm during the Screening and treatment periods and for at least 3 months after the last dose of asfotase alfa. Highly effective contraceptive methods are as follows:
- •a. Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation- Oral
- •b. Progestogen-only hormonal contraception associated with inhibition of ovulation- Oral
- •c. Intrauterine device
- •d. Intrauterine hormone-releasing system
- •e. Bilateral tubal occlusion
- •f. Vasectomy with documented medical assessment of surgical success
- •g. Condom and spermicide
- •7. Female patients of childbearing potential must have a negative pregnancy test at the time of enrollment
- •8. Female patients not of child-bearing potential due to surgical sterilization (at least 6 weeks after surgical bilateral oophorectomy with or without hysterectomy or at least 6 weeks after tubal ligation) confirmed by medical history, or menopause
- •9. Patients must be willing to comply with study procedures and the visit schedule
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 36
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Patients will be excluded from participation in this study if they meet any of the following exclusion criteria:
- •1. Investigational site personnel directly affiliated with this study and/or their immediate families. Immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
- •2. Employees of Alexion Pharmaceuticals
- •3. Currently enrolled in a clinical study involving another study drug or nonapproved use of a drug or device, or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study (excluding the HPP registry, in which concurrent enrollment is allowed).
- •4. Participated, within the last 30 days, in a clinical study involving a study drug (other than the study drug used in this study). If the previous study drug has a long half-life, 3 months or 5 half-lives (whichever is longer) should have passed.
- •5. Have completed or withdrawn from this study or any other study investigating asfotase alfa in the previous 3 years. This exclusion criterion does not apply to patients who are re-screened prior to randomization or patients enrolled in the HPP registry.
- •6. Women who are pregnant, planning to become pregnant, or breastfeeding
- •7. Serum 25-OH Vitamin D below 20 ng/mL at Screening
- •8. Screening serum creatinine or parathyroid hormone (PTH) levels 1.5 times the upper limit of normal
- •9. Medical condition, serious concurrent illness and/or injury, recent orthopedic surgery, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance or study endpoints, including all prescribed evaluations and follow-up activities
- •10. Prior treatment with bisphosphonates within 2 years of study entry for any length of time or for more than 2 consecutive years at any prior timepoint
- •11. Treatment with PTH, strontium, or sclerostin inhibitors within 6 months prior to the first dose of study drug
- •12. Unwilling or unable to comply with the use of a data collection device on which study patients will directly record data
研究者
相似试验
进行中（未招募）
1 期
Study to investigate the function of three medicinal products (monoclonal antibodies) on a population of patients with colorectal cancer that has spread from its site of origin to another part of the body.Metastatic Colorectal CarcinomaEUCTR2018-000618-39-ESSymphogen A/S54
进行中（未招募）
1 期
Study to investigate the function of three medicinal products (monoclonal antibodies) on a population of patients with colorectal cancer that has spread from its site of origin to another part of the body.EUCTR2018-000618-39-ITSYMPHOGEN A/S54
进行中（未招募）
1 期
A trial to learn how well REGN9933 works for preventing blood clots after knee replacement surgery in adult participantsCTIS2022-501470-18-00Regeneron Pharmaceuticals Inc.432
进行中（未招募）
不适用
Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase DeficiencyPatients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)MedDRA version: 17.1Level: LLTClassification code 10041515Term: Sphingomyelin lipidosisSystem Organ Class: 100000004850EUCTR2010-023953-12-GBGenzyme Corporation15
进行中（未招募）
不适用
Safety, Efficacy, and Pharmacokinetics Study of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase DeficiencyPatients with acid sphingomyelinase deficiency (Niemann-Pick Type B disease)MedDRA version: 16.0Level: LLTClassification code 10041515Term: Sphingomyelin lipidosisSystem Organ Class: 100000004850EUCTR2010-023953-12-ITGenzyme Corporation15