Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency

Официальное название

A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children With Growth Hormone Deficiency (GHD)

Обзор

The OraGrowtH Phase 3 Trial is a multi-national trial. The goals of the trial are to study LUM-201 as a treatment for Pediatric Growth Hormone Deficiency (PGHD) in naive to treatment children and validate the LUM-201 predictive enrichment marker (LUM-201 PEM) strategy to select subjects likely to respond to therapy with daily oral LUM-201.

Вмешательства

• Препарат LUM-201
  1.6 mg/kg/day, administered orally once daily
• Другое Matched Placebo (Capsules)
  Administered orally once daily

Первичные конечные точки

• AHV after 12 months on LUM-201 compared to placebo [Срок оценки: Day 1 to Month 12]

Критерии участия

Критерии включения

• Subjects must be naïve to treatment and prepubertal
• Subjects must have a maximal GH response of < 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months
• Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex
• Morning or random cortisol level of ≥ 7.0 μg/dL
• ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys
• Baseline height velocity (HV) based on ≥ 6 months of growth assessments < 25th percentile for age and sex
• Bone Age delay of ≥ 12 months compared to the chronological age
• In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative.
• Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 3 months prior to Day 1
• Baseline IGF-1 standard deviation score (SDS) ≤ -1.0

Критерии исключения

• Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment.
• Arm span to height ratio > 2 SDs below the mean for age and sex
• A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201
• Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors
• Current inflammatory diseases requiring systemic corticosteroid treatment for > 2 consecutive weeks within the last 3 months prior to the Screening Visit
• Use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency
• Any ECG at the Screening Visit noted to have a clinically significant abnormality, as confirmed by the MM
• Any subjects suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the Screening or Day 1 Visit
• Any subject suspected of having intracranial hypertension (IH) as confirmed by fundoscopy and other assessments
• Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), or total bilirubin > upper limit of normal (ULN)
• Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0 ng/mL on any prior standard of care GH stimulation test completed within 12 months
• Body weight ≤ 14.0 kg
• BMI < -2 or > +2 SDs for age and sex based on WHO standards
• Birth weight for gestational age < 3rd percentile based on WHO standards
• Treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome P450 (CYP) 3A/4
• History of spinal, cranial, or total body irradiation
• Attention deficit hyperactivity disorder (ADHD) diagnosis

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Тройное слепое

Основная цель

Лечение

Центры проведения

США · 29 центров

• Lumos Pharma Investigational Site — Birmingham
• Lumos Pharma Investigational Site — Madera
• Lumos Pharma Investigational Site — Orange
• Lumos Pharma Investigational Site — Sacramento
• Lumos Pharma Investigational Site — San Diego
• Lumos Pharma Investigational Site — Centennial
• Lumos Pharma Investigational Site — Greenwood Village
• Lumos Pharma Investigational Site — Washington D.C.
• … и ещё 21 центр

Таиланд · 5 центров

• Lumos Pharma Investigational Site — Bangkok Noi
• Lumos Pharma Investigational Site — Pathumwan
• Lumos Pharma Investigational Site — Muang
• Lumos Pharma Investigational Site — Hat Yai
• Lumos Pharma Investigational Site — Muang

Великобритания · 4 центра

• Lumos Pharma Investigational Site — Liverpool
• Lumos Pharma Investigational Site — London
• Lumos Pharma Investigational Site — London
• Lumos Pharma Investigational Site — Sheffield

Австралия · 3 центра

• Lumos Pharma Investigational Site — South Brisbane
• Lumos Pharma Investigational Site — Clayton
• Lumos Pharma Investigational Site — Parkville

Новая Зеландия · 2 центра

• Lumos Pharma Investigational Site — Auckland
• Lumos Pharma Investigational Site — Wellington

Аргентина · 1 центр

• Lumos Pharma Investigational Site — San Miguel de Tucumán

Чили · 1 центр

• Lumos Pharma Investigational Site — Santiago

Индия · 1 центр

• Lumos Pharma Investigational Site — Jaipur

Идентификаторы

NCT: NCT06948214 · LUM-201-10

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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