A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)

Официальное название

A Study to Evaluate the Safety and Tolerability of Multiple Ascending Doses of GS3-007a Dry Suspension in Patients With PGHD and to Evaluate the Efficacy and Safety of 52-week of Treatment With GS3-007a Dry Suspension: a Two-part, Multicenter, Randomized, Phase Ib/II Clinical Trial

Обзор

This is a two-part clinical study for children with growth hormone deficiency. In the first part, participants will be randomly assigned to receive different doses of an oral treatment (GS3-007a dry suspension) or a placebo for 14 days. This part is double-blinded, meaning neither the participants nor the doctors will know who is receiving the treatment or placebo. The goal is to find a safe and well-tolerated dose. In the second part, participants will be randomly assigned to receive either the selected dose of GS3-007a or another approved treatment for 52 weeks. This part is open-label, so everyone will know which treatment is being given. After that, all participants may continue taking GS3-007a for another 156 weeks in an extension phase to study long-term effects.

Вмешательства

• Препарат GS3-007a dry suspension
  GS3-007a
• Препарат Placebo for GS3-007a dry suspension
  Placebo for GS3-007a dry suspension
• Препарат rhGH injection
  rhGH injection

Первичные конечные точки

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) [Срок оценки: up to 21 days (Ib phase)]
• Annualized height velocity (AHV) at Week 26 of treatment [Срок оценки: 26 weeks (II phase)]
• Number of Participants With TEAEs [Срок оценки: up to 160 weeks (II phase extension)]

Вторичные конечные точки (6)

• PK concentrations of GS3-007a and metabolite GS3-017 [Срок оценки: up to 7 days (Ib phase)]
• Concentrations of serum growth hormone (GH), insulin-like growth factor-1 (IGF-1), and insulin-like growth factor-binding protein 3 (IGFBP-3) [Срок оценки: up to 14 days (Ib phase)]
• AHVs at each evaluation point [Срок оценки: 13 weeks, 39 weeks, 52 weeks (II phase)]
• Changes from baseline (ΔIGF-1 SDS) in the standard deviation score of the PD indicator insulin-like growth factor-1 (IGF-1 SDS) at each evaluation point [Срок оценки: 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase)]
• AHVs at each evaluation point [Срок оценки: up to 156 weeks (II phase extension)]
• Change from extension baseline in the height standard deviation score at each evaluation point (ΔHT SDS) [Срок оценки: up to 156 weeks (II phase extension)]

Критерии участия

Критерии включения

• chronological age (CA) ≥3 years at Screening
• Prepubertal girls or boys
• Height at Screening lower than the reference height for normal children of the same chronological age and sex minus 2 standard deviations (-2 SD)
• A confirmed diagnosis of growth hormone deficiency (GHD)
• Having not been treated with any growth-promoting drugs
• BA delayed by ≥6 months compared with the CA at Screening Subjects who meet all of the following criteria are eligible to enroll in the extension study (applicable for Part II extension period)
• Subjects who have completed the 52-week treatment period of phase II
• Subjects who do not permanently discontinue the investigational Medicinal Product (IMP) during the 52-week treatment period of phase II

Критерии исключения

• A highly allergic constitution
• Suspected or confirmed total pituitary deficiency, including patients previously confirmed with deficiency of ≥2 pituitary hormones other than GH
• Being confirmed with other chromosomal abnormalities or growth abnormalities affecting growth
• Congenital skeletal dysplasia or serious spinal anomalies
• Cognitive hypofunction, neurodevelopmental disorders, or psychiatric/psychological disorders that, in the investigator's opinion, may interfere with evaluation of study endpoints
• Any clinically significant abnormality that may affect growth or evaluation of the IMP
• Screening magnetic resonance imaging (MRI) scan of the sellar region confirming prior or current intracranial tumor growth
• Concurrent use of any medications that may affect growth or response to growth hormone therapy
• Epiphyseal closure
• Electrocardiogram (ECG) QTcF interval abnormal, with a history of QT/QTc interval prolonged
• Hepatic function indicators abnormal at Screening Patients meeting any of the following criteria may not be enrolled in this extension study (applicable for Part II extension period)
• Subjects with closed epiphyses
• Any clinically significant abnormality that may affect growth or evaluation of the IMP
• Known or suspected allergy to the IMP
• Women with positive blood human chorionic gonadotropin (hCG) at the pre-treatment visit

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Последовательный дизайн

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology — Ухань

Идентификаторы

NCT: NCT07264595 · GenSci073-201

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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