A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Официальное название

A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Обзор

This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease. The principal aim of this study is to obtain safety and tolerability data across varous dose levels of DNL952 in participants with late-onset Pompe disease (LOPD).

Вмешательства

• Препарат DNL952
  Intravenous repeating dose

Первичные конечные точки

• Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs) [Срок оценки: 48 weeks]
• Incidence and severity of infusion-related reacations (IRRs) [Срок оценки: 48 weeks]

Вторичные конечные точки (6)

• PK parameter: Maximum concentration (Cmax) of DNL952 in serum [Срок оценки: 48 weeks]
• PK Parameter: Time to reach maximum concentration (tmax) of DNL952 in serum [Срок оценки: 48 weeks]
• PK Parameter: Area under the concentration-time curve (AUC) from time zero to time of last measurable concentration (AUClast) of DNL952 in serum [Срок оценки: 48 weeks]
• PK Parameter: AUC from time 0 to infinity (AUC∞) of DNL952 in serum [Срок оценки: 48 weeks]
• PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum [Срок оценки: 48 weeks]
• PK Parameter: terminal elimination half-life (t1/2) of DNL952 in serum [Срок оценки: 48 weeks]

Критерии участия

Критерии включения

• Body weight ≥40 kg
• Diagnosis of LOPD
• Upright FVC ≥ 30% of predicted normal value
• Able to ambulate ≥ 40 meters (use of assistive devices is acceptable)
• \[Cohorts A1-A4 only\] Have received avalglucosidase alfa or cipaglucosidase alfa at a dose of 20 mg/kg every 2 weeks for at least 12 months prior to screening
• \[Cohorts B1-B2 only\] Must not have received any enzyme-replacement therapy for Pompe disease in the 12 months prior to screening

Критерии исключения

• Any ongoing, clinically significant, unstable, or poorly controlled neurological, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematological, immunological, allergic, or ophthalmic disease not related to Pompe disease, or other major disorders. Well-controlled conditions are permitted if investigator and Sponsor agree.
• Wheelchair-dependent
• Require noninvasive ventilation for an average of more than 6 hours per day while awake or any invasive ventilation. Use of noninvasive ventilation during sleep is acceptable.
• Received an experimental gene therapy at any time or participation in any other investigational drug trial or use of investigational drug within 60 days or 5 half-lives, whichever is longer, before screening

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Последовательный дизайн

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 3 центра

• University of California-Irvine — Irvine
• Duke University School of Medicine - Early Phase Research Unit — Durham
• The Lysosomal & Rare Disorders Research & Treatment Center — Fairfax

Идентификаторы

NCT: NCT07354724 · DNLI-J-0001 · 2025-524082-25-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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