Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis
Status:
Completed
Trial end date:
2021-06-07
Target enrollment:
Participant gender:
Summary
Autoimmune PAP is a rare lung disease affecting less than 5,000 individuals in US with no
FDA-approved pharmacologic therapy. Results from "off-label" use in case reports and clinical
studies completed outside of the US indicate that inhaled rhGM-CSF may be a safe and
effective thera-py for autoimmune PAP. Preliminary clinical trials of inhaled rhGM-CSF in
autoimmune PAP patients show promising results, 62%-96% therapeutic response rate without any
identifiable drug-related adverse effects in at least 73 autoimmune PAP patients. However,
the pharmacokinetics (PK), pharmacodynamics (PD), optimal dose, and treatment duration to
maximize efficacy are unknown.
The goal is to begin to address these knowledge gaps for inhaled sargramostim for autoimmune
PAP patients with a pilot safety and PK/PD study (TPSC-110). TPSC-110, PharmPAP, which is a
self-controlled open-label, phase I study to evaluate the safety, PK, and PD of inhaled
sargra-mostim in autoimmune PAP patients. These results will impact the field by 1)
confirming existing published data, 2) monitoring the local effects of inhaled sargramostim
in autoimmune PAP patients, 3) potentially demonstrating a safe starting dose for a later
trial to evaluate the therapeutic efficacy of inhaled sargramostim for autoimmune PAP.
Phase:
Phase 1
Details
Lead Sponsor:
Children's Hospital Medical Center, Cincinnati
Collaborators:
National Center for Advancing Translational Science (NCATS) Rare Diseases Clinical Research Network