NOT_YET_RECRUITING
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are: The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP. This is an open-label study: all participants will receive treatment with molgramostim. Patients will: * Take molgramostim once daily via nebulizer every day for 12 months. * Visit the clinic approximately every 12 weeks for checkups and tests. * Keep a diary of any oxygen use.
Conditions:
🦠 Autoimmune Pulmonary Alveolar Proteinosis
🗓️ Study Start (Actual) August 2024
🗓️ Primary Completion (Estimated) January 2027
✅ Study Completion (Estimated) August 2027
👥 Enrollment (Estimated) 5
🔬 Study Type INTERVENTIONAL
📊 Phase PHASE3
Locations:
📍 Cincinnati, Ohio, United States

📋 Eligibility Criteria

Description

    Inclusion Criteria:

    • * Be ≥6 and \<18 years of age, at the time of signing the informed consent and informed assent (if applicable).
    • * Have a history of pulmonary alveolar proteinosis, based on examination of a lung biopsy, bronchoalveolar lavage cytology, or a high-resolution computed tomogram of the chest.
    • * Have a positive serum anti-GM-CSF autoantibody test result confirming aPAP.
    • * Have a hemoglobin (Hb)-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) ≤70% predicted at Screening.

    Exclusion Criteria:

    • * Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production.
    • * Have undergone treatment with Lung Lavage (WLL) within 1 month of Baseline
Ages Eligible for Study: 6 Years to 18 Years (CHILD, ADULT)
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No

🗓️ Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

  • First Submitted 22 May 2024
  • First Submitted that Met QC Criteria 22 May 2024
  • First Posted 29 May 2024

Study Record Updates

  • Last Update Submitted that Met QC Criteria 22 May 2024
  • Last Update Posted 29 May 2024
  • Last Verified May 2024