Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| The First Affiliated Hospital of Guangzhou Medical University | OTHER |
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental: hBMMSC | Experimental | 1.0×10^6/kg hBMMSC (participant's body weight) single dose 2.0×10^6/kg hBMMSC (participant's body weight) single dose 2.0×10^6/kg hBMMSC (participant's body weight) administered twice |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| human Bone Marrow Mesenchymal Stem Cell (hBMMSC) | Drug | 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) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability | Number of participants with serious and non-serious adverse events | 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 |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| JiuzhitangMakerBeijingCellTech | Beijing | Daxing | 100021 | China |
Not provided
| ID | Term |
|---|---|
| C567049 | Pulmonary Alveolar Proteinosis, Acquired |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
|
| 4 weeks, 12 weeks, 24 weeks, 48 weeks |
| Alveolar-arterial oxygen difference (A-aDO2) | Change from Baseline in the A-aDO2 as measured | 48 weeks |
| Difference in 6-minute walk distance (6MWD) | Change from Baseline in the 6MWD as measured | 4 weeks, 12 weeks, 24 weeks, 48 weeks |
| Percutaneous arterial oxygen saturation (SpO2) | Change from Baseline in the SpO2 as measured | 4 weeks, 12 weeks, 24 weeks, 48 weeks |
| Difference in modified Medical Research Council（mMRC） | Change from Baseline in the mMRC as tested | 4 weeks, 12 weeks, 24 weeks, 48 weeks |
| Difference in St. George's Respiratory Questionnaire（SGRQ） | Change from Baseline in the SGRQ as tested | 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 | 24 weeks, 48 weeks |
| Difference in Disease severity score (DSS) | Change from Baseline in the DSS score | 24 weeks, 48 weeks |
| Difference in arterial partial pressure of oxygen (PaO2) | Change from Baseline in the PaO2 as measured | 24 weeks, 48 weeks |
| Difference in Krebs Von den Lungen-6（KL-6） | Change from Baseline in the KL-6 as measured | 2 weeks，4 weeks, 12 weeks, 24 weeks |
| Difference in carcinoembryonic antigen（CEA） | Change from Baseline in the CEA as measured | 2 weeks，4 weeks, 12 weeks, 24 weeks |
| Difference in lactate dehydrogenase (LDH) | Change from Baseline in the LDH as measured | 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 | 108 weeks |