[Translation] An open-label study evaluating the safety, efficacy, and pharmacokinetics of Ripuga in treatment-naïve Chinese subjects with Fabry disease

主要目的：通过评价整个研究期间在治疗中出现的严重不良事件（TEAE）的发生率来评估瑞普佳在中国法布雷病初治受试者中的安全性。次要目的： 1.评价瑞普佳的其他安全性参数; 2.评价瑞普佳对肾功能参数（估算肾小球滤过率 [eGFR]）的疗效； 1）评价瑞普佳对其他肾功能变量、疼痛以及药效学（PD）标记物（血浆 lyso-Gb3）的疗效 2）在年龄 <18 岁的受试者中评价听力变化 3）评价瑞普佳在中国法布雷病初治受试者中的药代动力学（PK）。

[Translation]

Primary objective: To evaluate the safety of Ripuga in Chinese Fabry disease naïve subjects by evaluating the incidence of treatment-emergent serious adverse events (TEAEs) throughout the study. Secondary objectives: 1. To evaluate other safety parameters of Ripuga; 2. To evaluate the efficacy of Ripuga on renal function parameters (estimated glomerular filtration rate [eGFR]); 1) To evaluate the efficacy of Ripuga on other renal function variables, pain, and pharmacodynamic (PD) markers (plasma lyso-Gb3); 2) To evaluate hearing changes in subjects aged <18 years; 3) To evaluate the pharmacokinetics (PK) of Ripuga in Chinese Fabry disease naïve subjects.

Start Date01 May 2022

Sponsor / Collaborator

Dhl Supply Chain Netherlands BV

[+3]

CTRI/2021/07/034906 / RecruitingPhase 4

A Post Marketing Surveillance (PMS) Study for VPRIVÃ?Â® (velaglucerase alfa) in India - SHP669-406_VPRIVÃ?Â® (velaglucerase alfa) - India

Start Date15 Jul 2021

Sponsor / Collaborator

Shire Human Genetic Therapies, Inc.

LBCTR2019090265 / Unknown statusPhase 3

A Phase 3 Randomized, Double-blind, Placebo-controlled, Parallel-group Efficacy and Safety Study of SHP647 as Maintenance Therapy in Subjects With Moderate to Severe Crohn’s Disease (CARMEN CD 307) - CARMEN CD 307

Start Date30 Mar 2020

Sponsor / Collaborator

Shire Human Genetic Therapies, Inc.

100 Clinical Results associated with Shire Human Genetic Therapies, Inc.

0 Patents (Medical) associated with Shire Human Genetic Therapies, Inc.

Literatures (Medical) associated with Shire Human Genetic Therapies, Inc.

01 Mar 2023·Surgery Today

Efficacy, safety, and pharmacokinetics of teduglutide in adult Japanese patients with short bowel syndrome and intestinal failure: two phase III studies with an extension

Article

Author: Chen, Szu-Ta ; Udagawa, Eri ; Yoon, MinJung ; Mizushima, Tsunekazu ; Sugita, Akira ; Nakamura, Shiro ; Suzuki, Ryohsuke Ken ; Ohge, Hiroki ; Grimm, Andrew ; Tazuke, Yuko ; Ikeuchi, Hiroki ; Wada, Motoshi

AbstractPurposeThe short- and long-term efficacy, safety, and pharmacokinetics of teduglutide were analyzed in adult Japanese patients with short bowel syndrome and intestinal failure (SBS-IF).MethodsPatients received teduglutide 0.05 mg/kg/day in clinical trials (TED-C14-004, SHP633-306, and extension SHP633-307). Data were analyzed at 24 weeks and an interim data cut-off of 4.5 years.ResultsThe parenteral support (PS) volume decreased by ≥ 20% for 9/18 patients at 24 weeks and in all 11 patients by data cut-off in SHP633-307. The mean (standard deviation) PS volume decreased from baseline at 24 weeks in TED-C14-004 (−30.1 ± 25.9%) and SHP633-306 (−25.6 ± 25.5%), and at data cut-off in SHP633-307 (−57.08 ± 28.49%). Teduglutide was absorbed quickly. The adverse events were consistent with the underlying disease and known adverse drug reactions. Anti-teduglutide antibody titers declined with long-term treatment.ConclusionsIn Japanese adults with SBS-IF, teduglutide treatment was associated with clinically meaningful reductions in PS requirements, similar to findings in prior international studies. No new safety concerns specific to the Japanese SBS-IF patient population were identified with short- or long-term teduglutide treatment. Anti-teduglutide antibody titers disappeared in most Japanese adults with long-term treatment. These results constitute the longest evaluation of teduglutide treatment within clinical trials reported to date.

01 Dec 2022·BMC NephrologyQ4 · MEDICINE

An open-label phase 2 trial to assess the efficacy, safety and pharmacokinetics of lanthanum carbonate in hyperphosphatemic children and adolescents with chronic kidney disease undergoing dialysis

Q4 · MEDICINE

ArticleOA

Author: Szabó, Tamás ; Sundberg, Aimee ; Uddin, Sharif ; Umeh, Obi ; Wasilewska, Anna ; Shavkin, Aleksey ; Achenbach, Heinrich ; Vergani, Andrea ; Murray, Rose Ann

AbstractBackgroundThis study assessed the efficacy, tolerability and pharmacokinetics (PK) of lanthanum carbonate (LC) in hyperphosphatemic children and adolescents with chronic kidney disease (CKD) undergoing dialysis.MethodsThis was a three-part, multicenter, open-label study of LC (oral powder formulation) in patients 10 to < 18 years old with CKD undergoing dialysis. In part 1, the single-dose PK of LC (500 mg, ≤12 years old; 1000 mg, > 12 years old) were summarized. In part 2, patients received calcium carbonate (CC [chewable tablet formulation]) (1500–6500 mg [total daily dose]) followed by LC (powder formulation) (1500–3000 mg [total daily dose]), or LC only (1500–3000 mg [total daily dose]), each for 8 weeks. During part 3, patients received LC (1500–3000 mg [total daily dose]) for up to 6 months. The primary efficacy endpoint was the proportion of LC-treated patients achieving serum phosphorus control after 8 weeks during parts 2 and/or 3, defined as: ≤1.94 mmol/L, < 12 years old; ≤1.78 mmol/L, ≥12 years old. Secondary efficacy endpoints included: the proportion of patients who achieved serum phosphorus control after 8 weeks of treatment with CC followed by 8 weeks of treatment with LC (with a washout period between treatments). The safety of LC and CC was also evaluated.ResultsIn part 1, 20 patients received a single dose of LC. In part 2, 53 and 51 patients were treated with CC and LC for 8 weeks, respectively. During part 3, 42 patients received LC for up to 6 months. Most patients were white and male. For the primary efficacy endpoint, 50% (17/34) of patients who received LC for 8 weeks during parts 2 and/or 3 achieved serum phosphorus control. After 8 weeks of treatment with CC, 58.8% of patients achieved serum phosphorus control; after a subsequent washout period and 8 weeks of treatment with LC, 70.6% of patients achieved serum phosphorus control. Tmax and t1/2 occurred within 3–8 h and ~ 19 h, respectively; however, variability was observed. LC and CC were generally well tolerated.ConclusionsThese data support the use of LC to manage hyperphosphatemia in pediatric patients with CKD undergoing dialysis.Trial registrationClinicalTrials.gov identifier: NCT01696279; EudraCT identifier: 2012–000171-17.Date of registration: 01/10/2012.

04 Sep 2022·The Journal of Infectious DiseasesQ2 · MEDICINE

Drug Resistance Mutations and Associated Phenotypes Detected in Clinical Trials of Maribavir for Treatment of Cytomegalovirus Infection

Q2 · MEDICINE

Article

Author: Wu, Jingyang ; Bo, Tien ; Crumpacker, Clyde ; Chou, Sunwen ; Song, Kening

AbstractBackgroundIn separate phase 2 trials, 120 patients received maribavir for cytomegalovirus (CMV) infection failing conventional therapy (trial 202) and 119 received maribavir for asymptomatic infection (trial 203). Overall, 172 cleared their CMV infection (CMV DNA <200 copies/mL) within 6 weeks.MethodsBaseline and posttreatment plasma samples were tested for mutations in viral genes UL97, UL54, and/or UL27. Selected viral mutants were phenotyped for drug susceptibility.ResultsBaseline samples revealed UL54 mutations newly phenotyped as conferring resistance to standard DNA polymerase inhibitor(s), including K493N, P497S, K513T, L565V, V823A, A987V, and E989D. Of 29 patients (including 25 from trial 202) who cleared but later experienced recurrent CMV infection while on maribavir, 23 had available UL97 genotyping data; 17 had known resistance mutations (T409M or H411Y) and 5 additional had UL97 C480F alone. The newly phenotyped mutation C480F conferred high-grade maribavir resistance and low-grade ganciclovir resistance. Among 25 who did not respond to >14 days of therapy, 9 showed T409M or H411Y and 4 others showed C480F alone.ConclusionsAfter maribavir therapy (400–1200 mg twice daily), UL97 mutations T409M, H411Y, or C480F emerge to confer maribavir resistance in patients with recurrent CMV infection while on therapy or no response to therapy.Clinical Trials RegistrationNCT01611974 and EudraCT 2010-024247-32.

News (Medical) associated with Shire Human Genetic Therapies, Inc.

30 Aug 2016

·www.biospace.com

Pliant Therapeutics Appoints Suzanne Bruhn, Ph.D., To Board Of Directors

REDWOOD CITY, Calif.--(BUSINESS WIRE)--Pliant Therapeutics, Inc., a company focused on discovering, developing and commercializing breakthrough treatments for fibrotic diseases, today announced the appointment of Suzanne Bruhn, Ph.D., to its board of directors. “With a career spanning almost two decades of success in the development, commercialization and partnering of new therapies for patients, her breadth of experience will bring significant value to Pliant Therapeutics as we advance our pipeline of innovative fibrosis therapies.” “Suzanne is a distinguished biopharmaceutical leader who brings a wealth of drug development expertise, and in particular, a deep knowledge of the fibrosis field,” said Bernard Coulie, M.D., Ph.D., CEO of Pliant Therapeutics. “With a career spanning almost two decades of success in the development, commercialization and partnering of new therapies for patients, her breadth of experience will bring significant value to Pliant Therapeutics as we advance our pipeline of innovative fibrosis therapies.” Dr. Bruhn most recently served as president and CEO of Promedior, Inc., a privately held, clinical-stage immunotherapy company pioneering the development of targeted therapeutics to treat diseases involving fibrosis. There, she successfully negotiated an exclusive agreement with Bristol Myers Squibb for the right to acquire Promedior for a total deal potential of $1.25 billion. Prior to Promedior, Dr. Bruhn spent 13 years at Shire Human Genetic Therapies (HGT), a division of Shire PLC, where she held a series of positions of increasing responsibility before serving as senior vice president of strategic planning and program management. Currently, Dr. Bruhn serves on the board of directors of Raptor Pharmaceuticals (NASDAQ: RPTP), a commercial-stage, biopharmaceutical company developing therapeutics for orphan diseases. Dr. Bruhn received a Bachelor’s degree from Iowa State University, a Ph.D. from the Massachusetts Institute of Technology and was a postdoctoral fellow at Harvard Medical School. “While significant medical and scientific progress has been made in recent years, there is still much work to be done for patients with fibrotic diseases,” said Dr. Bruhn. “Pliant Therapeutics truly embraces the challenge of developing new therapies for fibrosis, and I am looking forward to contributing my understanding of clinical development as the company advances its therapeutic pipeline.” About Pliant Therapeutics Pliant Therapeutics is an early-stage biotechnology company harnessing the therapeutic capabilities of integrin biology and TGF-ß signaling to develop breakthrough treatments for fibrotic diseases. By leveraging its powerful product engine, Pliant’s mission is to prevent or even reverse fibrosis, restoring organ function. The company also aims to build a patient registry for certain areas of fibrotic disease to both increase understanding of natural disease progression and fuel biomarker discovery for efficient clinical trial design. Founded by a group of seasoned experts in fibrosis biology and medicinal chemistry, Pliant Therapeutics was launched in 2016 by Third Rock Ventures and is headquartered in Redwood City, California. For more information, please visit . Contacts Pure CommunicationsKatie Engleman, 910-509-3977Katie@purecommunicationsinc.com

CollaborateImmunotherapy

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Drug(Targets)	Indications	Global Highest Phase

Idursulfase ( IDS )	Mucopolysaccharidosis II More	Approved
Agalsidase alfa ( GALA )	-	Phase 3
Epoetin delta(Sanofi) ( EPO receptor )	Retinal Diseases More	Pending
Recombinant human lysosomal acid lipase(Shire Human Genetic Therapies Inc) ( Lipase )	Wolman Disease More	Pending
Mesalamine ( PP2A )	Irritable Bowel Syndrome More	Pending

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