A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

Официальное название

A Phase 2, Multicenter, Long-Term, Open Label Extension Trial Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children and Adolescents With Achondroplasia

Обзор

TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial. Participants who complete a prior TransCon CNP trial and meet all eligibility criteria will be invited to continue into the long-term open label extension trial to receive 100 µg CNP/kg/week of TransCon CNP. Trial treatment will be completed when the participant reaches 16 years of age for females and 18 years of age for males and have femur and tibial epiphyseal closure. TransCon CNP treatment will continue if femur and tibial epiphyseal closure is not confirmed at the age of 16 years for females, and 18 years for males. Treatment with TransCon CNP will be completed once femur and tibial epiphyseal closure is confirmed by radiographic imaging. The trial duration is individual for each trial participant. Visits will occur every 12-14 weeks throughout the trial.

Вмешательства

• Препарат TransCon CNP
  TransCon CNP drug product is a lyophilized powder in a single-use vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

Первичные конечные точки

• Safety and Tolerability [Срок оценки: Through trial completion, an average of 10 years]
• Height Z-scores [Срок оценки: Through trial completion, an average of 10 years]

Вторичные конечные точки (1)

• Annualized Growth Velocity [Срок оценки: Through trial completion, an average of 10 years]

Критерии участия

Критерии включения

• Written, signed informed consent of the parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC). For participants who are below the age of consent, a written assent will be obtained in accordance with applicable requirements as required by IRB/HREC/IEC. Upon reaching the legal age of consent, depending on applicable requirements, these participants will be asked to give their own written consent.
• Participants with achondroplasia who have completed a clinical trial with TransCon CNP.
• Parent(s)/legal guardian(s) willing and able to administer weekly SC injections of TransCon CNP and to follow the protocol.
• Considered eligible based on the safety evaluations performed for evaluating stopping/holding rule criteria during the prior TransCon CNP clinical trial.

Критерии исключения

• Known or suspected hypersensitivity to the investigational product or related products (trehalose, tris\[hydroxymethyl\]aminomethane, succinate, and methoxy polyethylene glycol \[mPEG\]).
• Have received any dose of prescription medications, investigational medicinal product (other than TransCon CNP).
• Sexually active female participants and female partners of male participants of childbearing potential not using a highly effective form of contraceptive (including oral, injectable, or implantable contraception, or intrauterine device (IUD)) for the entire trial period and for 90 days post end of the trial.
• Participants with serum 25-hydroxy-vitamin D (25OHD) levels of <50 nmol/L (<20 ng/mL) at Visit 1 must be on treatment regimen of Vitamin D supplementation.
• Any disease or condition that, in the opinion of the investigator, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or may present undue risk from receiving trial treatment. This could include family situations, complications or manifestations, or medications that might impact safety or be considered confounding.

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 · 8 центров

• Ascendis Pharma Investigational Site — Little Rock
• Ascendis Investigational Site — Aurora
• Ascendis Investigational Site — Wilmington
• Ascendis Investigational Site — Saint Paul
• Ascendis Pharma Investigational Site — Columbia Falls
• Ascendis Pharma Investigational Site — Buffalo
• Ascendis Investigational Site — Houston
• Ascendis Pharma Investigational Site — Madison

Австралия · 1 центр

• Ascendis Pharma Investigational Site — Parkville

Австрия · 1 центр

• Ascendis Investigational Site — Linz

Канада · 1 центр

• Ascendis Investigational Site — Montreal

Дания · 1 центр

• Ascendis Pharma Investigational Site — Copenhagen

Германия · 1 центр

• Ascendis Investigational Site — Berlin

Ирландия · 1 центр

• Ascendis Pharma Investigational Site — Dublin

Новая Зеландия · 1 центр

• Ascendis Investigational Site — Auckland

Португалия · 1 центр

• Ascendis Investigational Site — Coimbra

Испания · 1 центр

• Ascendis Investigational Site — Vitoria-Gasteiz

Идентификаторы

NCT: NCT05929807 · ASND0039

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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