|
|
|
|
|
Inactive Indication- |
Drug Highest PhasePhase 1/2 |
First Approval Ctry. / Loc.- |
First Approval Date- |
|
MechanismCYP4V2 modulators |
|
|
|
Inactive Indication- |
Drug Highest PhasePhase 1/2 |
First Approval Ctry. / Loc.- |
First Approval Date- |
|
|
|
|
|
Inactive Indication- |
Drug Highest PhaseEarly Phase 1 |
First Approval Ctry. / Loc.- |
First Approval Date- |
An Open-Label, Multiple-Center, Phase I/II Dose Escalation Study for the Safety and Efficacy of NGGT002 in Adults With Classic Phenylketonuria
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects with classic Phenylketonuria (PKU). NGGT002 is an rAAV8 based vector carrying a functional copy of the human PAH gene.
Participants will receive a single administration of NGGT002 and will be followed for safety and efficacy for 5 years.
NGGT002静脉滴注治疗成人苯丙酮尿症的安全性和有效性的 I/II期临床研究
[Translation] Phase I/II clinical study on the safety and efficacy of intravenous infusion of NGGT002 in the treatment of phenylketonuria in adults
主要目的评估NGGT002注射液在成人苯丙酮尿症(PKU)患者中的安全性和耐受性。评估NGGT002注射液在成人苯丙酮尿症患者中降低血浆苯丙氨酸(Phe)浓度的有效性。
次要目的监测PKU疾病在饮食自由化、神经和心理症状以及患者生活质量方面的变化。评价治疗后,NGGT002在人体内的药代动力学(PK)、载体脱落和免疫原性特征。
[Translation] The primary objective is to evaluate the safety and tolerability of NGGT002 injection in adult patients with phenylketonuria (PKU). To evaluate the effectiveness of NGGT002 injection in reducing plasma phenylalanine (Phe) concentrations in adult patients with phenylketonuria.
The secondary objectives are to monitor changes in PKU disease in terms of dietary liberalization, neurological and psychological symptoms, and quality of life of patients. To evaluate the pharmacokinetic (PK), carrier shedding, and immunogenicity characteristics of NGGT002 in humans after treatment.
NGGT001视网膜下腔注射治疗结晶样视网膜变性的I/II期临床研究
[Translation] Phase I/II clinical study of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration
主要目的评价NGGT001视网膜下腔注射治疗结晶样视网膜变性的安全性和耐受性,推荐后期临床研究的给药剂量。初步探索NGGT001视网膜下腔注射治疗结晶样视网膜变性的有效性。次要目的评价NGGT001视网膜下腔注射后的药物代谢特性。评价NGGT001视网膜下腔注射后机体的免疫反应。
[Translation] The primary objective is to evaluate the safety and tolerability of NGGT001 subretinal injection in the treatment of crystalline retinal degeneration and recommend the dosage for later clinical studies. To preliminarily explore the effectiveness of NGGT001 subretinal injection in the treatment of crystalline retinal degeneration. The secondary objective is to evaluate the drug metabolism characteristics of NGGT001 after subretinal injection. To evaluate the body's immune response after subretinal injection of NGGT001.
100 Clinical Results associated with Suzhou Nuojiebei Biotechnology Co., Ltd.
0 Patents (Medical) associated with Suzhou Nuojiebei Biotechnology Co., Ltd.
100 Deals associated with Suzhou Nuojiebei Biotechnology Co., Ltd.
100 Translational Medicine associated with Suzhou Nuojiebei Biotechnology Co., Ltd.