Study of Human Bone Marrow Mesenchymal Stem Cells in aPAP

• Status: Not yet recruiting
• Trial end date: 2026-08-30
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.
• Collaborator: The First Affiliated Hospital of Guangzhou Medical University

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

• Women who are known to be pregnant, breastfeeding, have a positive pregnancy test (which will be detected during the screening process), or plan to become pregnant during the trial