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

NCT04452097 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: BXU001-COVID19
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

COVID-19; ARDS; Acute Respiratory Distress Syndrome

Outcome Measures

Primary Outcomes (2)

• Incidence of infusion-related adverse events

  Safety will be defined by the incidence of infusion-related adverse events as assessed by the treating physician

  Day 3

• Incidence of any treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)

  Safety will be defined by the incidence of TEAEs and TESAEs as assessed by the treating physician

  Day 28

Secondary Outcomes (6)

• Selection of an appropriate dose of BX-U001 for the following Phase 2 study

  Day 28

• All-cause mortality

  Day 28

• Proportion of patients achieving clinical response by the improvement of at least 2 points on the 7-point ordinal scale.

  Day 14

• Duration of ICU stay

  Day 28

• Duration of hospital stay

  Day 28

Study Arms (5)

Phase 1 Low-dose Group

EXPERIMENTAL

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.

Biological: Human umbilical cord mesenchymal stem cells + best supportive care

Phase 1 Middle-dose Group

EXPERIMENTAL

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.

Biological: Human umbilical cord mesenchymal stem cells + best supportive care

Phase 1 High-dose Group

EXPERIMENTAL

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.

Biological: Human umbilical cord mesenchymal stem cells + best supportive care

Phase 2a Treatment Group

EXPERIMENTAL

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.

Biological: Human umbilical cord mesenchymal stem cells + best supportive care

Phase 2a Control Group

PLACEBO COMPARATOR

Eligible subjects will receive a single infusion of placebo control and standard of care treatment.

Other: Placebo control + best supportive care

Interventions

Human umbilical cord mesenchymal stem cells + best supportive care BIOLOGICAL

hUC-MSC product will be administered intravenously in addition to the standard of care treatment.

Phase 1 High-dose Group; Phase 1 Low-dose Group; Phase 1 Middle-dose Group; Phase 2a Treatment Group

Placebo control + best supportive care OTHER

Placebo control will be administered intravenously in addition to the standard of care treatment.

Phase 2a Control Group

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Subjects who have received investigational drug (except for Remdesivir) for the treatment of COVID-19 within 30 days before screening;
• 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;
• 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;
• Subjects receiving extracorporeal membrane oxygenation (ECMO) support.
• Subjects who are allergic to low-molecular-weight heparin calcium or human albumin;
• Subjects with ongoing malignant tumors.

Sponsors & Collaborators

• Baylx Inc.: lead

MeSH Terms

Conditions

COVID-19; Respiratory Distress Syndrome

Condition Hierarchy (Ancestors)

Pneumonia, Viral; Pneumonia; Respiratory Tract Infections; Infections; Virus Diseases; Coronavirus Infections; Coronaviridae Infections; Nidovirales Infections; RNA Virus Infections; Lung Diseases; Respiratory Tract Diseases; Respiration Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: In phase 1, eligible subjects will be divided into low, medium and high-dose groups with 3 patients/group. If there is no safety concerns for each group, the dose will be escalated from lower dose to the next higher dose. In phase 2a, a total of 30 subjects will be randomized into the treatment and control groups with a ratio of 3:2.

Study Record Dates

• First Submitted: June 28, 2020
• First Posted: June 30, 2020
• Study Start: June 1, 2026
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): September 28, 2027
• Last Updated: December 29, 2025

Record last verified: 2025-12