A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of Navepegritide Administered Once Weekly for 52 Weeks in Adolescents (12-18 Years of Age) With Achondroplasia.

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.

开始日期2024-12-10

申办/合作机构

Ascendis Pharma A/S

CTIS2023-508341-40-00

/ Not yet recruiting临床2期

COACH: A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children with Achondroplasia - ASND0042

开始日期2024-07-26

申办/合作机构

Ascendis Pharma Growth Disorders A/S

NCT06433557

/ Active, not recruiting临床2期

A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia

This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.

开始日期2024-07-26

申办/合作机构

Ascendis Pharma Growth Disorders A/S

100 项与 Navepegritide 相关的临床结果

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100 项与 Navepegritide 相关的转化医学

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100 项与 Navepegritide 相关的专利（医药）

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项与 Navepegritide 相关的文献（医药）

2024-11-01·Expert Review of Endocrinology & Metabolism

Progress in managing children with achondroplasia

Review

作者: Savarirayan, Ravi ; Taylor-Miller, Tashunka

INTRODUCTION:

Achondroplasia is a heritable disorder of the skeleton that affects approximately 300,000 individuals worldwide. Until recently, treatment for this condition has been purely symptomatic. Efficacious treatment options for children are now approved or are in clinical trials.

AREAS COVERED:

This review discusses key advances in the therapeutic management of children with achondroplasia, including vosoritide, the first approved drug, and other emerging precision therapies. These include navepegritide, a long-acting form of C-type natriuretic peptide, and infigratinib, a tyrosine kinase receptor inhibitor, summarizing trial outcomes to date.

EXPERT OPINION:

The advent of the first approved precision therapy for achondroplasia in vosoritide has been a paradigm shifting advance for children affected by this condition. In addition to changing their natural growth history, it is hoped that it will decrease their medical complications and enhance functionality. These new treatment options highlight the importance of prompt prenatal identification and subsequent testing of a suspected fetus with achondroplasia and counseling of families. It is hoped that, in the near future, families will have the option to consider a range of effective targeted therapies that best suit their child with achondroplasia, starting from birth should they choose.

2023-11-01·EClinicalMedicine

Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial

Article

作者: Ma, Nina S ; Zarate, Yuri A ; Savarirayan, Ravi ; Winding, Bent ; Mao, Meng ; Sousa, Sérgio B ; McDonnell, Ciara ; Bober, Michael B ; Hoernschemeyer, Daniel G ; Bacino, Carlos A ; Smith, Alden ; Legare, Janet M ; Volck, Birgitte ; Schnabel, Dirk ; Chakraborty, Mukta ; Hofman, Paul L ; Ljungberg, Merete ; Giwa, Adebola ; White, Klane K ; Shu, Aimee D ; Högler, Wolfgang ; Quattrin, Teresa ; Abuzzahab, M Jennifer

Background:

TransCon CNP (navepegritide) is an investigational prodrug of C-type natriuretic peptide (CNP) designed to allow for continuous CNP exposure with once-weekly dosing. This 52-week phase 2 (ACcomplisH) trial assessed the safety and efficacy of TransCon CNP in children with achondroplasia.

Methods:

ACcomplisH is a global, randomised, double-blind, placebo-controlled, dose-escalation trial. Study participants were recruited between June 10, 2020, and September 24, 2021. Eligible participants were prepubertal, aged 2-10 years, with genetically confirmed achondroplasia, and randomised 3:1 to once-weekly subcutaneous injections of TransCon CNP (6, 20, 50, or 100 μg CNP/kg/week) or placebo for 52 weeks. Primary objectives were safety and annualised growth velocity (AGV). ACcomplisH is registered with ClinicalTrials.gov (NCT04085523) and Eudra (CT 2019-002754-22).

Findings:

Forty-two participants received TransCon CNP at doses of 6 μg (n = 10; 7 female), 20 μg (n = 11; 3 female), 50 μg (n = 10; 3 female), or 100 μg (n = 11; 6 female) CNP/kg/week, with 15 receiving placebo (5 female). Treatment-emergent adverse events (TEAEs) were mild or moderate with no grade 3/4 events reported. There were 2 serious TEAEs that were assessed as not related to TransCon CNP. Eleven injection site reactions occurred in 8 participants receiving TransCon CNP and no symptomatic hypotension occurred. TransCon CNP demonstrated a dose-dependent improvement in AGV. At 52 weeks, TransCon CNP 100 μg CNP/kg/week significantly improved AGV vs placebo (least squares mean [95% CI] 5.42 [4.74-6.11] vs 4.35 [3.75-4.94] cm/year; p = 0.0218), and improved achondroplasia-specific height SDS from baseline (least squares mean [95% CI] 0.22 [0.02-0·41] vs -0·08 [-0.25 to 0.10]; p = 0.0283). All participants completed the randomised period and continued in the ongoing open-label extension period receiving TransCon CNP 100 μg CNP/kg/week.

Interpretation:

This phase 2 trial suggests that TransCon CNP is effective, safe, with low injection site reaction frequency, and may provide a novel, once-weekly treatment option for children with achondroplasia. These results support TransCon CNP at 100 μg CNP/kg/week in the ongoing pivotal trial.

Funding:

Ascendis Pharma, A/S.

2022-11-01·BRITISH JOURNAL OF CLINICAL PHARMACOLOGY2区 · 医学

Phase 1 safety, tolerability, pharmacokinetics and pharmacodynamics results of a long‐acting C‐type natriuretic peptide prodrug, TransCon CNP

2区 · 医学

Article

作者: Christoffersen, Eva Dam ; Breinholt, Vibeke Miller ; Mygind, Per Holse ; Ota, Sho ; Will Charlton, R. ; Zhang, Ying ; Viuff, Dorthe

Aim:

TransCon CNP is a novel prodrug designed to provide sustained release of C‐type natriuretic peptide (CNP) for once‐weekly therapy, addressing the pathology leading to aberrant skeletal development in achondroplasia. This phase 1 trial was initiated to assess the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of TransCon CNP.

Methods:

This randomized, placebo‐controlled, single‐ascending dose phase 1 trial was performed at two sites in Australia and enrolled 45 healthy adult males. Subjects received placebo or TransCon CNP (single‐ascending dose cohorts [3, 10, 25, 75 or 150 μg CNP/kg]). The primary endpoint was frequency of adverse events and other safety outcomes. Other endpoints included PK and PD measured by cyclic guanosine‐monophosphate (cGMP) and amino‐terminal propeptide of CNP (NTproCNP).

Results:

TransCon CNP provided continuous systemic exposure to CNP over at least 7 days post‐dose. Plasma and urine levels of cGMP were significantly increased in subjects administered TransCon CNP at 75‐150 μg CNP/kg, indicating target engagement of active CNP at the natriuretic peptide receptor‐B (NPR‐B) for at least 1 week post‐dose. TransCon CNP was well‐tolerated, with no serious treatment‐emergent adverse events or discontinuations. Extensive cardiac safety assessments did not reveal any clinically relevant effects on electrocardiogram parameters, including heart rate, PR, QRS and QTcF intervals.

Conclusions:

Safety and PD data from this phase 1 trial support that TransCon CNP is well tolerated, with a PK profile compatible with a once‐weekly dosing regimen. Further studies are ongoing to evaluate the potential of TransCon CNP to positively impact abnormal endochondral ossification in children with achondroplasia.

项与 Navepegritide 相关的新闻（医药）

2025-06-03

·PipelineReview

FDA Accepts TransCon® CNP NDA for Priority Review

– Once-weekly TransCon CNP demonstrated significantly higher annualized growth velocity, the primary endpoint, compared to placebo – Multiple benefits beyond linear growth were observed compared to placebo, with a safety and tolerability profile similar to placebo – The FDA grants Priority Review designation for the evaluation of medicines that, if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition 1 COPENHAGEN, Denmark I June 02, 2025 I Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for TransCon CNP (navepegritide) for the treatment of children with achondroplasia and has set a Prescription Drug User Fee Act (PDUFA) goal date of November 30, 2025 to complete its review. The FDA also informed Ascendis that they are not currently planning to hold an advisory committee meeting to discuss this application. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to treat people living with achondroplasia by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle. “Too many profound medical needs endure for people living with achondroplasia,” said Chandler Crews, Founder of The Chandler Project. “Therapies that could address some of the underlying, serious complications of achondroplasia offer welcome potential to improve health outcomes beyond what currently approved therapies and interventions offer.” “TransCon CNP is designed to provide sustained exposure to CNP, resulting in continuous inhibition of the fibroblast growth factor receptor 3 (FGFR3) pathway that is overactive in achondroplasia. In clinical trials, these pharmacological effects have been associated with improvements in lower limb alignment, spinal canal dimensions, muscle strength, and growth compared to placebo,” said Janet Legare, M.D., Professor of Pediatrics at the University of Wisconsin School of Medicine and Public Health. “As a practicing physician, I am encouraged to see the FDA designating priority review for TransCon CNP as a potential new treatment option for children with achondroplasia.” “Our clinical trials of TransCon CNP are the first ever to demonstrate improvements beyond linear growth at 52 weeks compared to placebo,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. “People living with achondroplasia and their physicians have expressed an urgent need for a meaningful treatment option to address the complications of achondroplasia. We look forward to working with the FDA during its review to make TransCon CNP available as quickly as possible.” About Achondroplasia Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal deformities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition’s many complications. About Ascendis Pharma A/S Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. SOURCE: Ascendis Pharma

优先审批申请上市

2025-05-13

·药时空

疗效持续4年！维昇药业特立帕肽前药甲状腺功能减退2期临床长期随访结果

5月12日（当地时间），Ascendis Pharma（中国子公司：维昇药业）宣布Yorvipath（Palopegteriparatide，中文名：帕罗培特立帕肽，研发名称：TransCon PTH）成人甲状腺功能减退2期临床研究（PaTH Forward）214周最新研究结果证实其长期持久的疗效。PaTH Forward研究（NCT04009291)为在4周随机、双盲、安慰剂对照研究后进入为期266周的开放标签扩展研究。214周时，59例患者中的56例（95%)仍在接受治疗。214周研究结果如下：98%患者白蛋白校正血清钙水平继续维持正常范围内,93%患者摆脱现有疗法（定义：钙＜600mg/天&无需Vd）；骨转换标志物CTx（胶原C端肽）和P1NP（总I型胶原氨基端延长肽）从基线正常低端增加，26周到达峰值后下降并在214周维持稳定在基线水平之上；骨骼动力学改善：BMD维持在年龄和性别匹配正常范围内；67.8%患者eGFR（肾小球滤过率）具有临床意义增加（≥5ml/min/1.73m^2),eGFR从第4周明显变化。安全性方面，耐受性良好，无新的安全性信号，TEAE多为轻或中度，无重度或药物相关停药。Yorvipath（Palopegteriparatide，中文名：帕罗培特立帕肽，研发名称：TransCon PTH）为特立帕肽（PTH 1-34）前药，每天一次即可在24h内持续释放PTH。2018年，Ascendis授予Visen（维昇药业）大中华区开发和商业化TransCon hGH、TransCon PTH和TransCon CNP的独家权利。Yorvipath在2023年6月获得欧洲药品管理局批准上市，作为成人慢性甲状腺功能减退患者的替代性疗法。商品名与FDA批准的相同，规格有三种：168μg/0.56ml、294μg/0.98ml、420μg/1.4ml，剂型均为预充笔注射液。FDA的审批过程颇为曲折，2022年8月ascendis Pharma递交NDA申请获FDA受理并获得优先审评，2023年5月FDA下发完整回复函（CRL）提出药械组合的剂量传输控制策略问题，同年12月ascendis Pharma重新递交NDA申请并获FDA受理，而在2024年5月FDA又因审查过程中重大变更导致审评时限延长3月至2024年8月14日。2024年8月，FDA最终批准上市，用于甲状腺功能减退患者的替代性疗法。维昇药业2024年8月公布帕罗培特立帕肽用于治疗成人慢性甲状旁腺功能减退症的中国3期临床试验（PaTHway China），26周随机、双盲、安慰剂对照部分的数据：与安慰剂组相比，帕罗培特立帕肽组达到了主要复合终点的患者比例显著更高。识别微信二维码，可添加药时空小编请注明：姓名+研究方向！

优先审批上市批准临床2期突破性疗法临床3期

2025-05-05

·GlobeNewswire-Health Care

Ascendis to Share Its Latest Endocrinology Rare Disease Data at ESPE & ESE 2025

COPENHAGEN, Denmark, May 05, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it will share the latest data from its hypoparathyroidism, achondroplasia, and growth hormone deficiency (GHD) programs during ESPE & ESE 2025, the joint congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) being held May 10-13, 2025, in Copenhagen. Four oral presentations will feature Ascendis clinical trial results, including 4-year efficacy and safety data from the Phase 2 PaTH Forward Trial of TransCon PTH (palopegteriparatide) in adults with chronic hypoparathyroidism; Week 52 growth and bone morphometry data from the pivotal ApproaCH Trial of TransCon CNP (navepegritide) in children with achondroplasia; and efficacy and safety data from the Phase 3 foresiGHt Trial of TransCon hGH (lonapegsomatropin) in adults with growth hormone deficiency. “As new treatments and clinical practices transform the outlook for rare endocrine diseases, we are pleased to partner with trial investigators and leading experts to share new data highlighting clinical benefits of our novel therapies with attendees of ESPE & ESE 2025,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. The full range of Ascendis Pharma events and updates at ESPE & ESE 2025 include the following: Hypoparathyroidism Sunday, May 1109:55 - 10:25 CEST Congress TheaterFoyer F5Product TheaterRestoring Physiological Levels of PTH in Chronic HypoparathyroidismClick here to see the full session outline.Sunday, May 1116:55-17-55 CEST& Monday, May 1217:15-17:45 CESTPoster HallPoster P238Estimating the Risk of Chronic Kidney Disease Progression in Chronic Hypoparathyroidism: A Restrospective Matched Cohort Study, Using Real-World Data from England Monday, May 1213:00 - 14:30 CESTRoom D5SymposiumBeyond Conventional Care: Redefining Treatment Success in Chronic HypoparathyroidismClick here to see the full session outline.Monday, May 1215:55-16:05 CESTRoom C2Oral PresentationLong-Term Efficacy & Safety of Palopegteriparatide Treatment in Adults with Chronic Hypoparathyroidism: 4-Year Results from the Phase 2 PaTH Forward TrialPresented by Dr. Andrea PalermoAchondroplasiaSunday, May 1115:55-17:25 CESTRoom C2SymposiumAssessing HRQoL in Achondroplasia Across the Life CourseClick here to see the full session outline.Monday, May 1215:15-15:25 CESTRoom C2Oral PresentationEffects of Navepegritide on Bone Morphometry in Children with Achondroplasia: 52-Week Results from the ApproaCH Clinical TrialPresented by Dr. Leanne WardTuesday, May 1315:05-15:15 CESTRoom D2Oral PresentationEffects of Navepegritide on Growth in Children with Achondroplasia: 52-Week Results from the ApproaCH Clinical TrialPresented by Dr. Hanne HoveGrowth Hormone DeficiencyTuesday, May 1314:45-14:55 CESTRoom D2Oral PresentationResults of the foresiGH Trial Support the Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults with Growth Hormone Deficiency (GHD)Presented by Dr. Aleksandra Gilis-Januszewska More information about the events and presentations listed here is available to registered attendees of the Joint ESPE & ESE 2025 congress. About HypoparathyroidismHypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune and idiopathic causes. About AchondroplasiaAchondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal deformities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition’s many complications. About Adult Growth Hormone DeficiencyGrowth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels. Adult GHD is a condition in which an individual’s body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression. About Ascendis Pharma A/SAscendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) the clinical benefits of Ascendis’ novel therapies, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iii) Ascendis’ application of its TransCon technologies to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including tariffs and trade policies. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, and TransCon are trademarks owned by the Ascendis Pharma group. © May 2025 Ascendis Pharma A/S. Investor Contacts: Scott Smith Ascendis Pharma ir@ascendispharma.com Media Contact:Melinda BakerAscendis Pharmamedia@ascendispharma.com Patti BankICR Healthcare+1 (415) 513-1284patti.bank@icrhealthcare.com

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100 项与 Navepegritide 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
软骨发育不全	申请上市	美国	Ascendis Pharma A/S	2025-03-31

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05598320 (ApproaCH, PipelineReview) 人工标引	临床2/3期	软骨发育不全	84	TransCon CNP (ages 2-11 years)	積窪網網製膚遞構廠繭(繭糧遞衊積築積衊鏇窪) = 艱襯膚鏇廠餘淵構蓋鑰鹽構觸選襯夢醖鹹獵膚 (觸構製糧窪鹽繭襯鏇製 ) 更多	积极	2024-09-16
				Placebo (ages 2-11 years)	積窪網網製膚遞構廠繭(繭糧遞衊積築積衊鏇窪) = 廠糧夢鹹簾積鑰壓糧遞鹽構觸選襯夢醖鹹獵膚 (觸構製糧窪鹽繭襯鏇製 ) 更多
NCT04085523 (ACcomplisH, Company_Website) 人工标引	临床2期	软骨发育不全	57	TransCon CNP	齋糧廠廠製築餘淵鹽壓(鹹艱鏇襯構構遞繭製築) = patients treated for one year at the pivotal 100μg/kg/week dose demonstrated significant improvements in well-being and physical functioning compared to placebo. 糧膚構餘鬱遞醖鑰構糧 (範壓製網構艱鹹蓋糧鬱 ) 更多	积极	2024-06-24
				Placebo
NCT04085523 (ACcomplisH, CTgov)	临床2期	软骨发育不全	57	TransCon CNP (TransCon CNP 6 mcg)	醖願顧範襯鏇繭鏇積壓(糧觸網觸選齋淵淵艱觸) = 襯衊觸願構膚蓋膚製鹽獵蓋醖廠鹹餘廠廠顧鏇 (觸積選壓獵鹽鹽蓋鹽範, 蓋簾顧醖鑰遞鑰憲選憲 ~ 鬱齋衊積夢積廠願網選) 更多	-	2024-03-07
				TransCon CNP (TransCon CNP 20 mcg)	醖願顧範襯鏇繭鏇積壓(糧觸網觸選齋淵淵艱觸) = 繭廠鏇積夢簾範鬱夢鹽獵蓋醖廠鹹餘廠廠顧鏇 (觸積選壓獵鹽鹽蓋鹽範, 淵築醖築願襯觸醖窪壓 ~ 醖構選艱憲衊觸顧選構) 更多
NCT04085523 \| NCT05598320 (ACcomplisH, GlobeNewswire) 人工标引	临床2期	软骨发育不全	-	TransCon CNP 100 µg/kg/week	鹽壓獵鹹範鬱鹽艱鏇醖(選艱顧鬱鹽獵觸憲選觸): P-Value = 0.002 更多	积极	2023-12-20
				placebo
NCT05246033 (ACcomplisH China, PRNewswire) 人工标引	临床2期	软骨发育不全	24	TransCon CNP 50μg/kg/week	醖餘遞壓鹹鹽繭簾餘壓(廠製範顧蓋觸遞膚醖願) = 襯醖窪艱壓鹽廠遞網醖選簾壓糧醖艱膚築願顧 (網簾窪淵積憲襯膚衊觸 ) 达到	积极	2023-11-16
				TransCon CNP 100μg/kg/week	醖餘遞壓鹹鹽繭簾餘壓(廠製範顧蓋觸遞膚醖願) = 鏇壓夢蓋壓蓋製窪醖築選簾壓糧醖艱膚築願顧 (網簾窪淵積憲襯膚衊觸 ) 达到
NCT04085523 (ACcomplisH, Pubmed)	临床2期	软骨发育不全	42	TransCon CNP 100 μg CNP/kg/week	範繭選廠選網憲鬱築醖(鬱壓衊襯範壓艱製範鑰) = 網鬱遞簾醖襯糧艱鹹構襯蓋襯觸繭顧蓋鏇範鏇 (網淵鏇選鹹簾夢淵窪醖, 4.74 ~ 6.11)	积极	2023-11-01
				Placebo	範繭選廠選網憲鬱築醖(鬱壓衊襯範壓艱製範鑰) = 衊齋艱鏇衊網憲鏇窪壓襯蓋襯觸繭顧蓋鏇範鏇 (網淵鏇選鹹簾夢淵窪醖, 3.75 ~ 4.94)
NCT05598320 (ACcomplisH, ENDO2023)	临床2期	软骨发育不全	57	TransCon CNP 6 μg/kg/week	夢範淵淵簾餘遞鹽艱襯(衊範獵鏇艱艱蓋鬱鑰簾) = 顧壓顧蓋衊壓齋選壓夢窪鹽憲鏇顧構鹹遞鹹齋 (膚鏇襯簾夢憲齋範製鏇 )	积极	2023-10-05
				TransCon CNP 20 μg/kg/week	夢範淵淵簾餘遞鹽艱襯(衊範獵鏇艱艱蓋鬱鑰簾) = 鹽網獵鏇觸蓋蓋襯廠鹹窪鹽憲鏇顧構鹹遞鹹齋 (膚鏇襯簾夢憲齋範製鏇 )