Use of hUC-MSC Product (BX-U001) for the Treatment of COVID-19 With ARDS

Phase 1

Not yet recruiting

• Conditions: COVID-19; Acute Respiratory Distress Syndrome; ARDS
• Interventions: Biological: Human umbilical cord mesenchymal stem cells + best supportive care; Other: Placebo control + best supportive care

• Registration Number: NCT04452097
• Lead Sponsor: Baylx Inc.

Brief Summary

This is a phase 1/2a study including 2 parts, phase 1 and phase 2a. The phase 1 part is an open-label, single-arm, dose-escalating study to evaluate the safety and explore the dose limiting toxicity and maximum tolerated dose of a human umbilical cord derived mesenchymal stem cell product (BX-U001) in severe COVID-19 pneumonia patients with acute respiratory distress syndrome (ARDS). Qualified subjects after the screening will be divided into low, medium, or high dose groups to receive a single intravenous infusion of BX-U001 at the dose of 0.5×10\^6, 1.0×10\^6, or 1.5×10\^6 cells/kg of body weight, respectively. The Phase 2a part is a randomized, placebo-controlled, double-blind clinical trial examining the safety and biological effects of BX-U001 at the appropriate dose selected from phase 1 for severe COVID-19 pneumonia patients with the same inclusion/exclusion criteria as the phase 1 part.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-06-28
• Study First Submitted QC: 2020-06-28
• Study First Posted: 2020-06-30
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-11
• Last Update Posted: 2025-02-12
• Study Start: 2025-12-01
• Primary Completion: 2026-12-31
• Study Completion: 2027-02-28

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 39

Inclusion Criteria

1. Male or female, aged between 18 and 80;
2. Laboratory (RT-PCR, Gene Sequencing or Antibody) confirmed cases of severe COVID-19 pneumonia with mild to moderate ARDS (Berlin definition);
3. Patients are intubated;
4. Patients who voluntarily adhere to the research procedures and ensure good compliance during the research period;
5. Patients who fully understand the research nature of this study and sign written informed consent.

Exclusion Criteria

1. Subjects who have received investigational drug (except for Remdesivir) for the treatment of COVID-19 within 30 days before screening;
2. Subjects who are pregnant, breastfeeding or whose urinary pregnancy test is positive before participation in the study; subjects who are pregnant, breastfeeding, have a birth plan, or are unwillingness to use contraception during the study period and within 12 months of infusion; except for subjects who have sterilization surgery or menopause during the study period;
3. Within 3 days before screening/randomization, subjects who have used high-dose corticosteroids that is equivalent to methylprednisolone >240 mg/day or irregular use of systemic corticosteroids to treat other diseases that could affect the efficacy evaluated by the investigator;
4. Subjects receiving extracorporeal membrane oxygenation (ECMO) support.
5. Subjects who are allergic to low-molecular-weight heparin calcium or human albumin;
6. Subjects with ongoing malignant tumors.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Phase 1 Low-dose Group	Human umbilical cord mesenchymal stem cells + best supportive care	Eligible subjects will receive a single infusion of hUC-MCS product at the dose of 0.5 million cells/kg in addition to standard of care treatment.
Phase 1 High-dose Group	Human umbilical cord mesenchymal stem cells + best supportive care	Eligible subjects will receive a single infusion of hUC-MCS product at the dose of 1.5 million cells/kg in addition to standard of care treatment.
Phase 2a Treatment Group	Human umbilical cord mesenchymal stem cells + best supportive care	Eligible subjects will receive a single infusion of hUC-MCS product at the selected dose from phase 1 in addition to standard of care treatment.
Phase 1 Middle-dose Group	Human umbilical cord mesenchymal stem cells + best supportive care	Eligible subjects will receive a single infusion of hUC-MCS product at the dose of 1 million cells/kg in addition to standard of care treatment.
Phase 2a Control Group	Placebo control + best supportive care	Eligible subjects will receive a single infusion of placebo control and standard of care treatment.

Primary Outcome Measures

Name	Time	Method
Incidence of any treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)	Day 28	Safety will be defined by the incidence of TEAEs and TESAEs as assessed by the treating physician
Incidence of infusion-related adverse events	Day 3	Safety will be defined by the incidence of infusion-related adverse events as assessed by the treating physician

Secondary Outcome Measures

Name	Time	Method
Duration of ICU stay	Day 28
Changes in blood cytokine levels	Day 28
Selection of an appropriate dose of BX-U001 for the following Phase 2 study	Day 28	The dose will be selected based on the assessment of dose-limiting toxicity and maximum tolerated dose.
All-cause mortality	Day 28
Duration of hospital stay	Day 28
Proportion of patients achieving clinical response by the improvement of at least 2 points on the 7-point ordinal scale.	Day 14	The scale is as follows: 1. Death 2. Hospitalized, on invasive mechanical ventilation or extracorporeal membrane oxygenation (ECMO) 3. Hospitalized, on non-invasive ventilation or high flow oxygen devices 4. Hospitalized, requiring low flow supplemental oxygen 5. Hospitalized, not requiring supplemental oxygen - requiring ongoing medical care (COVID-19 related or otherwise) 6. Hospitalized, not requiring supplemental oxygen - no longer required ongoing medical care 7. Not hospitalized.