NSTL国家科技图书文献中心

1. PRX004 in variant amyloid transthyretin (ATTRv) amyloidosis: results of a phase 1, open-label, dose-escalation study NSTL国家科技图书文献中心

Suhr, Ole B. | Grogan, Martha ... - 《Amyloid》 - 2025,32(1) - 14～21 - 共8页 - 被引量：3

摘要：, phase 1 trial comprised a 3 + 3 dose-escalation phase | and a long-term extension (LTE) phase* (NCT03336580* | ). In the dose-escalation phase, patients received | PRX004 (0.1, 0.3, 1, 3, 10 or 30 mg/kg), administered | amyloidosis completed the dose-escalation phase; 17

关键词： Amyloidosis | ATTR | cardiomyopathy | monoclonal antibody | phase 1

2. Population pharmacokinetic analyses for sulbactam-durlobactam using Phase 1, 2, and 3 data NSTL国家科技图书文献中心

Cammarata, Anthony P... | Safir, M. Courtney ... - 《Antimicrobial agents and chemotherapy.》 - 2025,69(1) - e0048524～e0048524 - 共16页

摘要： using data from eight Phase* 1-3 studies. A total of* | (AUC(0-24)) when HD was started 1 hour after the end | Sulbactam-durlobactam is a beta-lactam/beta | -lactamase inhibitor combination approved in the United | States for the treatment of hospital-acquired and

关键词： sulbactam-durlobactam | sulbactam | durlobactam | population pharmacokinetics | Phase 1 | Phase 2 | Phase 3

3. Intrathecal methotrexate in progressive multiple sclerosis: a phase 1 open-label study with long-term follow-up NSTL国家科技图书文献中心

Kolb, Hadar | Shachaf, Yuval ... - 《Journal of neurology》 - 2025,272(5) - Article: 374～Article: 374 - 共9页

摘要：, open-label, phase* 1 study was conducted* | patients over short- and long-term periods. A 1-year | baseline to 1 year. Secondary endpoints encompassed 6 | Progressive multiple sclerosis (PMS) remains | challenging to treat effectively. Intrathecal methotrexate

关键词： Methotrexate | Intrathecal treatment | Progressive multiple sclerosis | Phase 1 study

4. Phase 1 study of the safety, tolerability, and pharmacokinetics of a synthetic macrocyclic peptide antibiotic (BRII-693) in healthy adult participants NSTL国家科技图书文献中心

Watkins, Michael | Zhu, Yali ... - 《Antimicrobial agents and chemotherapy.》 - 2025,69(1) - e0128824～e0128824 - 共10页

摘要：-controlled phase* 1 study investigated the safety* | and Chinese participants. This phase* 1 study* | range of single- and repeat-dosing. Mean t 1/2 was | BRII-693 is a next-generation intravenous (IV | )-administered synthetic macrocyclic peptide antibiotic for

关键词： antibiotic | phase 1 | pharmacokinetics | safety

5. A phase 1/2 safety and efficacy study of TAK-754 gene therapy: The challenge of achieving durable factor VIII expression in haemophilia A clinical trials NSTL国家科技图书文献中心

Chapin, John | alvarez Roman, Maria...... - 《Haemophilia》 - 2025,31(1) - 108～117 - 共10页

摘要：-754 phase* 1/2 AAV gene therapy study for the* | treatment of haemophilia A.MethodsA phase* 1/2 single arm* | IntroductionHaemophilia A is an X-linked | bleeding disorder resulting from a deficiency of factor | VIII (FVIII). To date, multiple gene therapies have

关键词： adeno-associated virus | gene therapy | haemophilia | hypophosphatemia | phase 1-2 | adeno‐associated virus | phase 1–2

6. Results From a Phase 1 Study Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of ANX005, a C1q Inhibitor, in Patients With Guillain-Barré Syndrome NSTL国家科技图书文献中心

Mohammad, Quazi Deen | Islam, Zhahirul ... - 《Journal of the peripheral nervous system》 - 2025,30(1) - e70009～e70009 - 共11页

摘要：, placebo-controlled Phase* 1 trial evaluated the safety* | >= 1 week and provided C1q blockade in the CSF | pathway. Further investigation in a larger Phase* 3 trial* | Background and Aims: Guillain-Barr & eacute | ; syndrome (GBS) is an acute autoimmune peripheral

关键词： ANX005 | C1q | complement | Guillain-Barr & eacute; syndrome | Phase 1

7. Anlotinib in combination with docetaxel for advanced nonsmall cell lung cancer after failure of platinum-based treatment: A phase 1/2 trial NSTL国家科技图书文献中心

Jiawei Shou | Jun Chen ... - 《Cancer.》 - 2025,131(10) - e35822～e35822 - 共12页

摘要： objective of this phase* 1/2 trial was to establish the* | the phase* 1 trial, eight patients were enrolled to* | docetaxel. In the phase* 2 trial, in total, 88 patients* | was evaluated in 88 patients.Results: In the phase* 2* | Background: The combination of anlotinib with

关键词： advanced cancer; anlotinib; docetaxel; nonsmall cell lung cancer; phase 1/2.

8. Phase 1 study of HP518, a PROTAC AR degrader in patients with mCRPC: results on safety, pharmacokinetics, and anti-tumor activity NSTL国家科技图书文献中心

Azad, Arun A. | Gurney, Howard ... - 《Investigational new drugs.》 - 2025,43(2) - 435～445 - 共11页

摘要：-label Phase* 1 dose escalation study was conducted in* | PSA50 response. In this initial Phase* 1 study, HP518* | 1 novel androgen receptor pathway inhibitor (ARPI | ) and <= 1 line of chemotherapy received HP518 once | ) were Grade 1 or 2. The most common AEs were nausea

关键词： Castration-resistant prostate cancer | HP-518 | Androgen receptor | PROTAC | Phase 1 clinical study

9. Results of Cancer and Leukemia Group B 10102 (Alliance), a Phase 1/2 Study NSTL国家科技图书文献中心

Marlise R. Luskin | Jun Yin ... - 《Cancer.》 - 2025,131(4) - e35750～e35750 - 共13页

摘要： increasing toxicity. Methods In this phase* 1/2 study* | were CD52+ at diagnosis. Phase* 1 tested three dose* | patients (115 in phase* 1, 180 in phase 2); 206 (69.8* | that 30 mg was tolerable. Phase* 2 established* | Abstract Background Acute lymphoblastic

关键词： alemtuzumab | ALL | antibody | chemotherapy | monoclonal | older

10. Pharmacokinetics, safety, and efficacy of daridorexant in Japanese subjects: Results from phase 1 and 2 studies NSTL国家科技图书文献中心

Uchiyama, Makoto | Mishima, Kazuo ... - 《Journal of sleep research.》 - 2025,34(1) - e14302～e14302 - 共14页 - 被引量：1

摘要： Phase 1 study, daridorexant (10, 25, 50 mg) or placebo | similar to 8 h. In the Phase* 2, four-period, four-way* | endpoint) from baseline to days 1/2. Statistically | days 1/2 (sWASO, latency to sleep onset). All | Daridorexant is a dual orexin receptor

摘要：, phase 1 trial comprised a 3 + 3 dose-escalation phase | and a long-term extension (LTE) phase* (NCT03336580* | ). In the dose-escalation phase, patients received | PRX004 (0.1, 0.3, 1, 3, 10 or 30 mg/kg), administered | amyloidosis completed the dose-escalation phase; 17

关键词： Amyloidosis | ATTR | cardiomyopathy | monoclonal antibody | phase 1

摘要： using data from eight Phase* 1-3 studies. A total of* | (AUC(0-24)) when HD was started 1 hour after the end | Sulbactam-durlobactam is a beta-lactam/beta | -lactamase inhibitor combination approved in the United | States for the treatment of hospital-acquired and

关键词： sulbactam-durlobactam | sulbactam | durlobactam | population pharmacokinetics | Phase 1 | Phase 2 | Phase 3

摘要：, open-label, phase* 1 study was conducted* | patients over short- and long-term periods. A 1-year | baseline to 1 year. Secondary endpoints encompassed 6 | Progressive multiple sclerosis (PMS) remains | challenging to treat effectively. Intrathecal methotrexate

关键词： Methotrexate | Intrathecal treatment | Progressive multiple sclerosis | Phase 1 study

摘要：-controlled phase* 1 study investigated the safety* | and Chinese participants. This phase* 1 study* | range of single- and repeat-dosing. Mean t 1/2 was | BRII-693 is a next-generation intravenous (IV | )-administered synthetic macrocyclic peptide antibiotic for

关键词： antibiotic | phase 1 | pharmacokinetics | safety

摘要：-754 phase* 1/2 AAV gene therapy study for the* | treatment of haemophilia A.MethodsA phase* 1/2 single arm* | IntroductionHaemophilia A is an X-linked | bleeding disorder resulting from a deficiency of factor | VIII (FVIII). To date, multiple gene therapies have

关键词： adeno-associated virus | gene therapy | haemophilia | hypophosphatemia | phase 1-2 | adeno‐associated virus | phase 1–2

摘要：, placebo-controlled Phase* 1 trial evaluated the safety* | >= 1 week and provided C1q blockade in the CSF | pathway. Further investigation in a larger Phase* 3 trial* | Background and Aims: Guillain-Barr & eacute | ; syndrome (GBS) is an acute autoimmune peripheral

关键词： ANX005 | C1q | complement | Guillain-Barr & eacute; syndrome | Phase 1

摘要： objective of this phase* 1/2 trial was to establish the* | the phase* 1 trial, eight patients were enrolled to* | docetaxel. In the phase* 2 trial, in total, 88 patients* | was evaluated in 88 patients.Results: In the phase* 2* | Background: The combination of anlotinib with

关键词： advanced cancer; anlotinib; docetaxel; nonsmall cell lung cancer; phase 1/2.

摘要：-label Phase* 1 dose escalation study was conducted in* | PSA50 response. In this initial Phase* 1 study, HP518* | 1 novel androgen receptor pathway inhibitor (ARPI | ) and <= 1 line of chemotherapy received HP518 once | ) were Grade 1 or 2. The most common AEs were nausea

关键词： Castration-resistant prostate cancer | HP-518 | Androgen receptor | PROTAC | Phase 1 clinical study

摘要： increasing toxicity. Methods In this phase* 1/2 study* | were CD52+ at diagnosis. Phase* 1 tested three dose* | patients (115 in phase* 1, 180 in phase 2); 206 (69.8* | that 30 mg was tolerable. Phase* 2 established* | Abstract Background Acute lymphoblastic

关键词： alemtuzumab | ALL | antibody | chemotherapy | monoclonal | older

摘要： Phase 1 study, daridorexant (10, 25, 50 mg) or placebo | similar to 8 h. In the Phase* 2, four-period, four-way* | endpoint) from baseline to days 1/2. Statistically | days 1/2 (sWASO, latency to sleep onset). All | Daridorexant is a dual orexin receptor

关键词： daridorexant | elderly | insomnia | Japanese | orexin receptor antagonist | pharmacokinetics

4008-161-200 800-990-8900

国家科技图书文献中心

摘要：, phase 1 trial comprised a 3 + 3 dose-escalation phase | and a long-term extension (LTE) phase (NCT03336580 | ). In the dose-escalation phase, patients received | PRX004 (0.1, 0.3, 1, 3, 10 or 30 mg/kg), administered | amyloidosis completed the dose-escalation phase; 17

关键词： Amyloidosis | ATTR | cardiomyopathy | monoclonal antibody | phase 1

摘要： using data from eight Phase 1-3 studies. A total of | (AUC(0-24)) when HD was started 1 hour after the end | Sulbactam-durlobactam is a beta-lactam/beta | -lactamase inhibitor combination approved in the United | States for the treatment of hospital-acquired and

关键词： sulbactam-durlobactam | sulbactam | durlobactam | population pharmacokinetics | Phase 1 | Phase 2 | Phase 3

摘要：, open-label, phase 1 study was conducted | patients over short- and long-term periods. A 1-year | baseline to 1 year. Secondary endpoints encompassed 6 | Progressive multiple sclerosis (PMS) remains | challenging to treat effectively. Intrathecal methotrexate

关键词： Methotrexate | Intrathecal treatment | Progressive multiple sclerosis | Phase 1 study

摘要：-controlled phase 1 study investigated the safety | and Chinese participants. This phase 1 study | range of single- and repeat-dosing. Mean t 1/2 was | BRII-693 is a next-generation intravenous (IV | )-administered synthetic macrocyclic peptide antibiotic for

关键词： antibiotic | phase 1 | pharmacokinetics | safety

摘要：-754 phase 1/2 AAV gene therapy study for the | treatment of haemophilia A.MethodsA phase 1/2 single arm | IntroductionHaemophilia A is an X-linked | bleeding disorder resulting from a deficiency of factor | VIII (FVIII). To date, multiple gene therapies have

关键词： adeno-associated virus | gene therapy | haemophilia | hypophosphatemia | phase 1-2 | adeno‐associated virus | phase 1–2

摘要：, placebo-controlled Phase 1 trial evaluated the safety | >= 1 week and provided C1q blockade in the CSF | pathway. Further investigation in a larger Phase 3 trial | Background and Aims: Guillain-Barr & eacute | ; syndrome (GBS) is an acute autoimmune peripheral

关键词： ANX005 | C1q | complement | Guillain-Barr & eacute; syndrome | Phase 1

摘要： objective of this phase 1/2 trial was to establish the | the phase 1 trial, eight patients were enrolled to | docetaxel. In the phase 2 trial, in total, 88 patients | was evaluated in 88 patients.Results: In the phase 2 | Background: The combination of anlotinib with

关键词： advanced cancer; anlotinib; docetaxel; nonsmall cell lung cancer; phase 1/2.

摘要：-label Phase 1 dose escalation study was conducted in | PSA50 response. In this initial Phase 1 study, HP518 | 1 novel androgen receptor pathway inhibitor (ARPI | ) and <= 1 line of chemotherapy received HP518 once | ) were Grade 1 or 2. The most common AEs were nausea

关键词： Castration-resistant prostate cancer | HP-518 | Androgen receptor | PROTAC | Phase 1 clinical study

摘要： increasing toxicity. Methods In this phase 1/2 study | were CD52+ at diagnosis. Phase 1 tested three dose | patients (115 in phase 1, 180 in phase 2); 206 (69.8 | that 30 mg was tolerable. Phase 2 established | Abstract Background Acute lymphoblastic

关键词： alemtuzumab | ALL | antibody | chemotherapy | monoclonal | older

摘要： Phase 1 study, daridorexant (10, 25, 50 mg) or placebo | similar to 8 h. In the Phase 2, four-period, four-way | endpoint) from baseline to days 1/2. Statistically | days 1/2 (sWASO, latency to sleep onset). All | Daridorexant is a dual orexin receptor

关键词： daridorexant | elderly | insomnia | Japanese | orexin receptor antagonist | pharmacokinetics

4008-161-200 800-990-8900

国家科技图书文献中心

摘要：, phase 1 trial comprised a 3 + 3 dose-escalation phase | and a long-term extension (LTE) phase* (NCT03336580* | ). In the dose-escalation phase, patients received | PRX004 (0.1, 0.3, 1, 3, 10 or 30 mg/kg), administered | amyloidosis completed the dose-escalation phase; 17

摘要： using data from eight Phase* 1-3 studies. A total of* | (AUC(0-24)) when HD was started 1 hour after the end | Sulbactam-durlobactam is a beta-lactam/beta | -lactamase inhibitor combination approved in the United | States for the treatment of hospital-acquired and

摘要：, open-label, phase* 1 study was conducted* | patients over short- and long-term periods. A 1-year | baseline to 1 year. Secondary endpoints encompassed 6 | Progressive multiple sclerosis (PMS) remains | challenging to treat effectively. Intrathecal methotrexate

摘要：-controlled phase* 1 study investigated the safety* | and Chinese participants. This phase* 1 study* | range of single- and repeat-dosing. Mean t 1/2 was | BRII-693 is a next-generation intravenous (IV | )-administered synthetic macrocyclic peptide antibiotic for

摘要：-754 phase* 1/2 AAV gene therapy study for the* | treatment of haemophilia A.MethodsA phase* 1/2 single arm* | IntroductionHaemophilia A is an X-linked | bleeding disorder resulting from a deficiency of factor | VIII (FVIII). To date, multiple gene therapies have

摘要：, placebo-controlled Phase* 1 trial evaluated the safety* | >= 1 week and provided C1q blockade in the CSF | pathway. Further investigation in a larger Phase* 3 trial* | Background and Aims: Guillain-Barr & eacute | ; syndrome (GBS) is an acute autoimmune peripheral

摘要： objective of this phase* 1/2 trial was to establish the* | the phase* 1 trial, eight patients were enrolled to* | docetaxel. In the phase* 2 trial, in total, 88 patients* | was evaluated in 88 patients.Results: In the phase* 2* | Background: The combination of anlotinib with

摘要：-label Phase* 1 dose escalation study was conducted in* | PSA50 response. In this initial Phase* 1 study, HP518* | 1 novel androgen receptor pathway inhibitor (ARPI | ) and <= 1 line of chemotherapy received HP518 once | ) were Grade 1 or 2. The most common AEs were nausea

摘要： increasing toxicity. Methods In this phase* 1/2 study* | were CD52+ at diagnosis. Phase* 1 tested three dose* | patients (115 in phase* 1, 180 in phase 2); 206 (69.8* | that 30 mg was tolerable. Phase* 2 established* | Abstract Background Acute lymphoblastic

摘要： Phase 1 study, daridorexant (10, 25, 50 mg) or placebo | similar to 8 h. In the Phase* 2, four-period, four-way* | endpoint) from baseline to days 1/2. Statistically | days 1/2 (sWASO, latency to sleep onset). All | Daridorexant is a dual orexin receptor