Clinical trial NCT04452097: 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).
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
PHASE2
Conditions: COVID-19
ARDS
Acute Respiratory Distress Syndrome
Lead sponsor: Baylx Inc.
Sponsor class: INDUSTRY
First posted: 2020-06-30
Enrollment: 39
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
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.
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Incidence of infusion-related adverse events
timeFrame: Day 3
measure: Incidence of any treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)
timeFrame: Day 28
Secondary outcomes:
measure: Selection of an appropriate dose of BX-U001 for the following Phase 2 study
timeFrame: Day 28
measure: All-cause mortality
timeFrame: Day 28
measure: Proportion of patients achieving clinical response by the improvement of at least 2 points on the 7-point ordinal scale.
timeFrame: Day 14
measure: Duration of ICU stay
timeFrame: Day 28
measure: Duration of hospital stay
timeFrame: Day 28
measure: Changes in blood cytokine levels
timeFrame: Day 28
Other outcomes:
Locations:
Study officials:
References: