An Open-label Phase IIa Study to Evaluate the Safety and Preliminary Efficacy of HBMMSC Injection in the Treatment of APAP

Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.1 site in 1 country10 target enrollmentDecember 25, 2023

ConditionsAutoimmune Pulmonary Alveolar Proteinosis

Interventionshuman Bone Marrow Mesenchymal Stem Cell (hBMMSC)

Drugshuman Bone Marrow Mesenchymal Stem Cell (hBMMSC)

Overview

Phase: Phase 2
Intervention: human Bone Marrow Mesenchymal Stem Cell (hBMMSC)
Conditions: Autoimmune Pulmonary Alveolar Proteinosis
Sponsor: Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.
Enrollment: 10
Locations: 1
Primary Endpoint: Alveolar-arterial difference in oxygen tension (A-aDO2)
Status: Active, not recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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.

Detailed Description

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.

Registry

clinicaltrials.gov

Start Date

December 25, 2023

End Date

August 30, 2026

Last Updated

last year

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Male or female aged 18 years or older
•Patients diagnosed with aPAP by both CT and biopsy (TBLB, TBCB, or surgical lung biopsy) or bronchoalveolar perfusion (BAL) as well as GM-CSF autoantibodies in serum positive
•No remission was demonstrated by CT, pulmonary function test results,or blood gas analysis at least 2 times (at least 3 months apart) before enrollment
•At rest PaO2≤70 mmHg
•Fertile participants must receive effective medical contraception (for both male and female participants, up to one year after the last study dosing)
•Voluntary signed informed consent

Exclusion Criteria

•Diagnosed with hereditary PAP, secondary PAP, or another type of PAP
•Received whole lung lavage（WLL） therapy within 6 months before enrollment
•Received previous GM-CSF therapy within 6 months before enrollment
•Received other clinical trial treatment within 3 months before enrollment
•Participated in other stem cell studies within 1 year before enrollment
•Inflammatory disease or autoimmune disorder requiring treatment associated with significant immunosuppression (e.g. more than 10 mg/day systemic prednisolone)
•Active infection (viral, bacterial, fungal or mycobacterial), which may affect the assessment of efficacy in the trial
•History of malignant tumors
•Known allergic reactions to any of the ingredients in the study drug
•Participants who, in the opinion of the investigator, would aggravate any other serious pre-existing medical condition are not suitable for this trial

Arms & Interventions

Experimental: hBMMSC

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

Intervention: human Bone Marrow Mesenchymal Stem Cell (hBMMSC)

Outcomes

Primary Outcomes

Alveolar-arterial difference in oxygen tension (A-aDO2)

Time Frame: 24 weeks

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.

Secondary Outcomes

Imaging（Chest CT score ）(24 weeks, 48 weeks)
Safety and tolerability(48 weeks)
Difference in pulmonary function(4 weeks, 12 weeks, 24 weeks, 48 weeks)
Alveolar-arterial oxygen difference (A-aDO2)(48 weeks)
Difference in 6-minute walk distance (6MWD)(4 weeks, 12 weeks, 24 weeks, 48 weeks)
Percutaneous arterial oxygen saturation (SpO2)(4 weeks, 12 weeks, 24 weeks, 48 weeks)
Difference in modified Medical Research Council（mMRC）(4 weeks, 12 weeks, 24 weeks, 48 weeks)
Difference in St. George's Respiratory Questionnaire（SGRQ）(4 weeks, 12 weeks, 24 weeks, 48 weeks)
Difference in Disease severity score (DSS)(24 weeks, 48 weeks)
Difference in arterial partial pressure of oxygen (PaO2)(24 weeks, 48 weeks)
Difference in Krebs Von den Lungen-6（KL-6）(2 weeks，4 weeks, 12 weeks, 24 weeks)
Difference in carcinoembryonic antigen（CEA）(2 weeks，4 weeks, 12 weeks, 24 weeks)
Difference in lactate dehydrogenase (LDH)(2 weeks，4 weeks, 12 weeks, 24 weeks)
Number of Rescue whole lung lavage (WLL) in 108 weeks(108 weeks)