Autoimmune Pulmonary Alveolar ProteinosisPhase 2NCT06111846
Study of Human Bone Marrow Mesenchymal Stem Cells in APAP
A Phase 2 study testing human Bone Marrow Mesenchymal Stem Cell (hBMMSC) in people with Autoimmune Pulmonary Alveolar Proteinosis, run by Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd. at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Autoimmune Pulmonary Alveolar Proteinosis
- What is being tested?
- human Bone Marrow Mesenchymal Stem Cell (hBMMSC)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 25 Dec 2023to 30 Aug 2026
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
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- 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 locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Being tested
- human Bone Marrow Mesenchymal Stem Cell (hBMMSC)
- Medical subject headings
- Pulmonary Alveolar Proteinosis, Acquired
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
human Bone Marrow Mesenchymal Stem Cell (hBMMSC)Drug
Also called 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)
| Study group | Role | What they receive |
|---|---|---|
| Experimental: hBMMSC | Experimental | 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 Receives: Drug: human Bone Marrow Mesenchymal Stem Cell (hBMMSC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Alveolar-arterial difference in oxygen tension (A-aDO2)
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.
Measured: 24 weeks
Secondary outcomes (14)
Safety and tolerability
Number of participants with serious and non-serious adverse events
Measured: 48 weeks
Difference in pulmonary function
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
Measured: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Alveolar-arterial oxygen difference (A-aDO2)
Change from Baseline in the A-aDO2 as measured
Measured: 48 weeks
Difference in 6-minute walk distance (6MWD)
Change from Baseline in the 6MWD as measured
Measured: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Percutaneous arterial oxygen saturation (SpO2)
Change from Baseline in the SpO2 as measured
Measured: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Difference in modified Medical Research Council（mMRC）
Change from Baseline in the mMRC as tested
Measured: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Difference in St. George's Respiratory Questionnaire（SGRQ）
Change from Baseline in the SGRQ as tested
Measured: 4 weeks, 12 weeks, 24 weeks, 48 weeks
Imaging（Chest CT score ）
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
Measured: 24 weeks, 48 weeks
Difference in Disease severity score (DSS)
Change from Baseline in the DSS score
Measured: 24 weeks, 48 weeks
Difference in arterial partial pressure of oxygen (PaO2)
Change from Baseline in the PaO2 as measured
Measured: 24 weeks, 48 weeks
Difference in Krebs Von den Lungen-6（KL-6）
Change from Baseline in the KL-6 as measured
Measured: 2 weeks，4 weeks, 12 weeks, 24 weeks
Difference in carcinoembryonic antigen（CEA）
Change from Baseline in the CEA as measured
Measured: 2 weeks，4 weeks, 12 weeks, 24 weeks
Difference in lactate dehydrogenase (LDH)
Change from Baseline in the LDH as measured
Measured: 2 weeks，4 weeks, 12 weeks, 24 weeks
Number of Rescue whole lung lavage (WLL) in 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
Measured: 108 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2023
- Study start25 Dec 2023
- Primary completion22 Jul 2025 (estimated)
- Study completion30 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune Pulmonary Alveolar ProteinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.
- Sponsor type
- University, hospital or other
- Collaborators
- The First Affiliated Hospital of Guangzhou Medical University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06111846
- Sponsor's protocol ID
- MKMSC-CT-001
- Status last verified
- Mar 2025
- First submitted
- 19 Oct 2023
- Last updated
- 24 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No