Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT06111846

Study of Human Bone Marrow Mesenchymal Stem Cells in APAP

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Autoimmune Pulmonary Alveolar Proteinosis Pulmonary Alveolar Proteinosis, Acquired

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

JiuzhitangMakerBeijingCellTech

Beijing, Daxing, 100021, China

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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)

Other names: hBMMSC

Primary outcomes

  1. 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

  1. Safety and tolerability

    Time frame: 48 weeks

    Number of participants with serious and non-serious adverse events

  2. Difference in pulmonary function

    Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks

    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

  3. Alveolar-arterial oxygen difference (A-aDO2)

    Time frame: 48 weeks

    Change from Baseline in the A-aDO2 as measured

  4. Difference in 6-minute walk distance (6MWD)

    Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks

    Change from Baseline in the 6MWD as measured

  5. Percutaneous arterial oxygen saturation (SpO2)

    Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks

    Change from Baseline in the SpO2 as measured

  6. Difference in modified Medical Research Council（mMRC）

    Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks

    Change from Baseline in the mMRC as tested

  7. Difference in St. George's Respiratory Questionnaire（SGRQ）

    Time frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks

    Change from Baseline in the SGRQ as tested

  8. Imaging（Chest CT score ）

    Time frame: 24 weeks, 48 weeks

    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

  9. Difference in Disease severity score (DSS)

    Time frame: 24 weeks, 48 weeks

    Change from Baseline in the DSS score

  10. Difference in arterial partial pressure of oxygen (PaO2)

    Time frame: 24 weeks, 48 weeks

    Change from Baseline in the PaO2 as measured

  11. Difference in Krebs Von den Lungen-6（KL-6）

    Time frame: 2 weeks，4 weeks, 12 weeks, 24 weeks

    Change from Baseline in the KL-6 as measured

  12. Difference in carcinoembryonic antigen（CEA）

    Time frame: 2 weeks，4 weeks, 12 weeks, 24 weeks

    Change from Baseline in the CEA as measured

  13. Difference in lactate dehydrogenase (LDH)

    Time frame: 2 weeks，4 weeks, 12 weeks, 24 weeks

    Change from Baseline in the LDH as measured

  14. Number of Rescue whole lung lavage (WLL) in 108 weeks

    Time frame: 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

Sponsors and collaborators

Lead sponsor

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

Other

Collaborators

  • The First Affiliated Hospital of Guangzhou Medical University

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2025
Study completion
2026
First posted
Nov 1, 2023
Registry last updated
Mar 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.