Study of Human Bone Marrow Mesenchymal Stem Cells in APAP

March 19, 2025 updated by: Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
10

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • daxing
      • Beijing, daxing, China, 100021
        • JiuzhitangMakerBeijingCellTech

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: 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
Drug: human Bone Marrow Mesenchymal Stem Cell (hBMMSC)
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
24 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Safety and tolerability
Time Frame: 48 weeks
Number of participants with serious and non-serious adverse events
48 weeks
Alveolar-arterial oxygen difference (A-aDO2)
Time Frame: 48 weeks
Change from Baseline in the A-aDO2 as measured
48 weeks
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
4 weeks, 12 weeks, 24 weeks, 48 weeks
Percutaneous arterial oxygen saturation (SpO2)
Time Frame: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Change from Baseline in the SpO2 as measured
4 weeks, 12 weeks, 24 weeks, 48 weeks
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
4 weeks, 12 weeks, 24 weeks, 48 weeks
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
4 weeks, 12 weeks, 24 weeks, 48 weeks
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
24 weeks, 48 weeks
Difference in Disease severity score (DSS)
Time Frame: 24 weeks, 48 weeks
Change from Baseline in the DSS score
24 weeks, 48 weeks
Difference in arterial partial pressure of oxygen (PaO2)
Time Frame: 24 weeks, 48 weeks
Change from Baseline in the PaO2 as measured
24 weeks, 48 weeks
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
2 weeks，4 weeks, 12 weeks, 24 weeks
Difference in carcinoembryonic antigen（CEA）
Time Frame: 2 weeks，4 weeks, 12 weeks, 24 weeks
Change from Baseline in the CEA as measured
2 weeks，4 weeks, 12 weeks, 24 weeks
Difference in lactate dehydrogenase (LDH)
Time Frame: 2 weeks，4 weeks, 12 weeks, 24 weeks
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
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
108 weeks
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
4 weeks, 12 weeks, 24 weeks, 48 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
The First Affiliated Hospital of Guangzhou Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 25, 2023

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 22, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 30, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 19, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 26, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 1, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 19, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • MKMSC-CT-001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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