Study of Human Bone Marrow Mesenchymal Stem Cells in APAP

Jiuzhitang

Status and phase: Active, not recruiting; Phase 2

Conditions: Autoimmune Pulmonary Alveolar Proteinosis

Treatments: Drug: human Bone Marrow Mesenchymal Stem Cell (hBMMSC)

Study type: Interventional

Funder types: Other

Identifiers

NCT06111846

MKMSC-CT-001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 10 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Experimental: hBMMSC

Experimental group

Description:

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

Treatment:

Drug: human Bone Marrow Mesenchymal Stem Cell (hBMMSC)