The purpose of this open-label phase IIa clinical trial study is to evaluate the safety and preliminary efficacy of hBMMSC intravenous treatment in patients with aPAP.
Autoimmune pulmonary alveolar proteinosis (aPAP) is a rare lung disease for which there is no specific drug treatment. Currently, the standard treatment strategy for PAP is whole-lung lavage (WLL), which is invasive and has limited therapeutic efficacy. The purpose of this study is to evaluate the safety and preliminary efficacy of hBMMSC intravenous treatment in patients with aPAP. The clinical trial intends to involve 10 participants. The trial is expected to last approximately 2 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Group 1: 1.0×10\^6 cells/kg (participant's body weight) single dose (n=3) Group 2: 2.0×10\^6 cells/kg (participant's body weight) single dose (n=3) Group 3: 2.0×10\^6 cells/kg (participant's body weight) administered twice (n=4)
JiuzhitangMakerBeijingCellTech
Beijing, Daxing, China
Alveolar-arterial difference in oxygen tension (A-aDO2)
The changes of alveolar-arterial difference in oxygen tension (A-aDO2) relative to baseline were assessed in the hBMMSC treated group after 24 weeks of treatment.
Time frame: 24 weeks
Safety and tolerability
Number of participants with serious and non-serious adverse events
Time frame: 48 weeks
Difference in pulmonary function
Pulmonary function tests include diffusing capacity of the lung for carbon monoxide(DLCO),forced vital capacity(FVC), forced expiratory volume in one second(FEV1).Change from Baseline in the DLCO、FVC、 FEV1 as measured
Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Alveolar-arterial oxygen difference (A-aDO2)
Change from Baseline in the A-aDO2 as measured
Time frame: 48 weeks
Difference in 6-minute walk distance (6MWD)
Change from Baseline in the 6MWD as measured
Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Percutaneous arterial oxygen saturation (SpO2)
Change from Baseline in the SpO2 as measured
Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Difference in modified Medical Research Council(mMRC)
Change from Baseline in the mMRC as tested
Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Difference in St. George's Respiratory Questionnaire(SGRQ)
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Change from Baseline in the SGRQ as tested
Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Imaging(Chest CT score )
Parenchymal lung density determined by quantitative computed tomography (CT) densitometry. Change from Baseline (Day 0) in the parenchymal densitometry as measured at 24 and 48 weeks
Time frame: 24 weeks, 48 weeks
Difference in Disease severity score (DSS)
Change from Baseline in the DSS score
Time frame: 24 weeks, 48 weeks
Difference in arterial partial pressure of oxygen (PaO2)
Change from Baseline in the PaO2 as measured
Time frame: 24 weeks, 48 weeks
Difference in Krebs Von den Lungen-6(KL-6)
Change from Baseline in the KL-6 as measured
Time frame: 2 weeks,4 weeks, 12 weeks, 24 weeks
Difference in carcinoembryonic antigen(CEA)
Change from Baseline in the CEA as measured
Time frame: 2 weeks,4 weeks, 12 weeks, 24 weeks
Difference in lactate dehydrogenase (LDH)
Change from Baseline in the LDH as measured
Time frame: 2 weeks,4 weeks, 12 weeks, 24 weeks
Number of Rescue whole lung lavage (WLL) in 108 weeks
Criteria for performing WLL were based on symptoms, decreased exercise capacity, and/or worsening of clinical symptoms of aPAP as judged by the investigator that the participant developed hypoxemia or decreased oxygen saturation
Time frame: 108 weeks