Clinical trial NCT06431776: Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
Official title: An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Autoimmune Pulmonary Alveolar Proteinosis
Keywords: alveolar proteinosis
autoimmune
lung lavage
GM-CSF
children
Lead sponsor: Savara Inc.
Sponsor class: INDUSTRY
First posted: 2024-05-29
Enrollment: 5
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-label study
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years, who are diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP).

The diagnosis of aPAP should be confirmed by an anti-GM-CSF antibody test and a history compatible with PAP based on patient symptoms, high resolution computed tomography of the lung, lung biopsy or bronchoalveolar lavage cytology.

The study consists of a 4-week screening period followed by a 48-week open-label treatment period. After completing the 48-week treatment or early withdrawal, subjects will enter a 4-week safety follow up period. The maximum treatment duration is 48-weeks, and the maximum study period will be 56 weeks. During the trial, lung lavage will be allowed as a rescue treatment in case of worsening of aPAP.

Eligibility criteria: 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

Primary outcomes:
measure: DLCO
timeFrame: 24 weeks
Secondary outcomes:
measure: DLCO
timeFrame: 48-weeks
measure: 6-minute walk distance
timeFrame: 24-weeks
measure: 6-minute walk distance
timeFrame: 48-weeks
measure: PedsQL
timeFrame: 24-weeks
measure: PedsQL
timeFrame: 48-weeks
measure: Oxygen Saturation (SpO2)
timeFrame: 24 weeks
measure: Oxygen Saturation (SpO2)
timeFrame: 48 weeks
Other outcomes:
measure: Adverse Events
timeFrame: 48 weeks
measure: Anti-GM-CSF Ab titer
timeFrame: 0, 4, 12,24,48 and 52 weeks
measure: FEV1
timeFrame: 24 and 48-weeks
measure: FVC
timeFrame: 24 and 48-weeks
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Savara Inc.
name: Yasmine Wasfi, MD, Ph.D.
role: STUDY_DIRECTOR
References: