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Идёт набор NCT05734196

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

Фаза I С лечением Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency Autosomal Recessive Hypophosphatemic Rickets Generalized Arterial Calcification of Infancy ATP-Binding Cassette Subfamily C Member 6 Deficiency

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: INZ-701.
Кому может быть актуально
Состояния в реестре: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency. Базовые параметры: до 1 год · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
США, Испания, Великобритания
Следующий шаг
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Официальное название

The ENERGY Study: An Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 (ENPP1) Deficiency or ATP-binding Cassette Sub-family C Member 6 (ABCC6) Deficiency

Обзор

The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.

Подробное описание

INZ-701 is an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy in development for the treatment of the ultra-rare genetic disorder, ENPP1 Deficiency or with ABCC6 Deficiency.

Study INZ701-104 (the ENERGY study) is a Phase 1b, open-label study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infant study participants with ENPP1 Deficiency or ABCC6 Deficiency.

The study will consist of up to a 60-day Screening Period, a 52-week Treatment Period during which study participants will receive INZ-701, an Extension Period during which participants may continue to receive INZ-701 until it is commercially available in the country where the participant resides, or until an alternative study of INZ-701 is available, and an End of Treatment (EOT) visit 30 days after the last dose of INZ-701. Upon treatment discontinuation, participants will continue to be followed for their ongoing disposition for survival outcome at least quarterly through the end of the study.

Вмешательства

  • Препарат INZ-701
    Recombinant fusion protein that contains the extracellular domains of human ENPP1 coupled with an Fc fragment from an immunoglobulin gamma-1 (IgG1) antibody.

Первичные конечные точки

  • Number of Treatment Emergent Adverse Events (TEAEs) [Срок оценки: 52 weeks (Treatment Period)]
  • Incidence of Anti-Drug Antibodies (ADA) [Срок оценки: 52 weeks (Treatment Period)]
  • Left Ventricular Ejection Fraction [Срок оценки: 52 weeks (Treatment Period)]
Вторичные конечные точки (4)
  • Change from Baseline in Plasma Inorganic Pyrophosphate (PPi) Levels [Срок оценки: 52 weeks (Treatment Period)]
  • Area under the Plasma Concentration versus Time Curve (AUC) of INZ-701 [Срок оценки: 52 weeks (Treatment Period)]
  • Maximum Plasma Concentration (Cmax) of INZ-701 [Срок оценки: 52 weeks (Treatment Period)]
  • ENPP1 Activity [Срок оценки: 52 weeks (Treatment Period)]

Критерии участия

Критерии включения

  • Infant aged ≤ 1 year at the time of enrollment
  • Study participant must have a confirmed post-natal molecular genetic diagnosis of ENPP1 Deficiency or ABCC6 Deficiency
  • Study participants must have clinical manifestations of generalized arterial calcification of infancy (GACI) or GACI-2, which must include at least one of the following: ectopic calcification, heart failure, respiratory distress, edema, cyanosis, hypertension, and cardiomegaly.
  • Study participant must weigh ≥0.5 kg at the time of the first dose of INZ-701 in this study
  • Written informed consent provided by a parent or legal guardian

Критерии исключения

  • In the opinion of the Investigator, presence of any clinically significant disease or laboratory abnormality that precludes study participation or may confound interpretation of study result
  • Receiving end of life or hospice care
  • Known malignancy
  • Concurrent participation in another non-Inozyme interventional study
  • Treatment with any non-Inozyme product or investigational device during study participation

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Не применимо
Модель
Одна группа
Маскирование
Открытое
Основная цель
Лечение

Центры проведения

США · 5 центров
  • Rady Children's Hospital — San Diego
  • Boston Children's Hospital — Boston
  • Nationwide Children's Hospital — Columbus
  • The Children's Hospital of Philadelphia — Philadelphia
  • The University of Utah — Salt Lake City
Испания · 1 центр
  • Hospital Sant Joan de Déu — Barcelona
Великобритания · 1 центр
  • Royal Manchester Children's Hospital — Manchester

Идентификаторы

NCT: NCT05734196 · INZ701-104

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗