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

• ClinicalTrials.gov Identifier: NCT04452097
• Recruitment Status: Not yet recruiting
• First Posted: June 30, 2020
• Last Update Posted: November 27, 2020
• Sponsor: Baylx Inc.
• Information provided by (Responsible Party): Baylx Inc.

Tracking Information

• First Submitted Date: June 28, 2020
• First Posted Date: June 30, 2020
• Last Update Posted Date: November 27, 2020
• Estimated Study Start Date: February 1, 2021
• Estimated Primary Completion Date: November 30, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 28, 2020)

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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 24, 2020)

• Selection of an appropriate dose of BX-U001 for the following Phase 2 study [ Time Frame: Day 28 ]
  The dose will be selected based on the assessment of dose-limiting toxicity and maximum tolerated dose.
• All-cause mortality [ Time Frame: Day 28 ]
• Proportion of patients achieving clinical response by the improvement of at least 2 points on the 7-point ordinal scale. [ Time Frame: 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.
• Duration of ICU stay [ Time Frame: Day 28 ]
• Duration of hospital stay [ Time Frame: Day 28 ]
• Changes in blood cytokine levels [ Time Frame: Day 28 ]

Original Secondary Outcome Measures (submitted: June 28, 2020)

Selection of an appropriate dose of the hUC-MSC product for the following Phase 2 study [ Time Frame: Day 28 ]

The dose will be selected based on the assessment of dose-limiting toxicity and maximum tolerated dose.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Use of hUC-MSC Product (BX-U001) for the Treatment of COVID-19 With ARDS
• Official Title: A Phase 1/2a Study of the Safety and Efficacy of BX-U001 for the Treatment of Severe COVID-19 Pneumonia With Moderate to Severe Acute Respiratory Distress Syndrome (ARDS).
• 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 Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2

Study Design

Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

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.

Masking: None (Open Label)
Primary Purpose: Treatment

Condition

• COVID-19
• ARDS
• Acute Respiratory Distress Syndrome

Intervention

• Biological: Human umbilical cord mesenchymal stem cells + best supportive care
  hUC-MSC product will be administered intravenously in addition to the standard of care treatment.
• Other: Placebo control + best supportive care
  Placebo control will be administered intravenously in addition to the standard of care treatment.

Study Arms

• Experimental: Phase 1 Low-dose Group
  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.
  Intervention: Biological: Human umbilical cord mesenchymal stem cells + best supportive care
• Experimental: Phase 1 Middle-dose Group
  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.
  Intervention: Biological: Human umbilical cord mesenchymal stem cells + best supportive care
• Experimental: Phase 1 High-dose Group
  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.
  Intervention: Biological: Human umbilical cord mesenchymal stem cells + best supportive care
• Experimental: Phase 2a Treatment Group
  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.
  Intervention: Biological: Human umbilical cord mesenchymal stem cells + best supportive care
• Placebo Comparator: Phase 2a Control Group
  Eligible subjects will receive a single infusion of placebo control and standard of care treatment.
  Intervention: Other: Placebo control + best supportive care

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Not yet recruiting
• Estimated Enrollment (submitted: November 24, 2020): 39
• Original Estimated Enrollment (submitted: June 28, 2020): 9
• Estimated Study Completion Date: December 30, 2021
• Estimated Primary Completion Date: November 30, 2021 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 80 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Vincent Liao, MD. Ph.D.; 949-308-1952; baylx@baylxinc.com

• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT04452097
• Other Study ID Numbers: BXU001-COVID19
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No

• IPD Sharing Statement: Not Provided
• Responsible Party: Baylx Inc.
• Study Sponsor: Baylx Inc.
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Baylx Inc.
• Verification Date: November 2020