ULSC-CV-01 is a clinical trial that comprises both Phase 1 and Phase 2a, which will be conducted sequentially. This trial will evaluate the safety and potential efficacy of allogeneic Umbilical Cord Lining Stem Cells (ULSC), which are a type of umbilical cord tissue derived mesenchymal stem cells (MSC), with intravenous (IV) administration in hospitalized patients with acute respiratory distress syndrome (ARDS) due to COVID-19.
The Phase 1, open-label, non-controlled trial in this study will investigate the safety of
intravenous (IV) infusion of ULSC in a total of 20 patients with COVID-19-related ARDS that
will include patients that are not intubated and not on a ventilator (NV) and patients that
are intubated and on a ventilator (V) for respiratory support. Separate cohorts of each group
(NV and V) will receive either a single dose (one infusion) or repeat dose (two infusions
separated by 48-hour interval).
The Phase 2a randomized and placebo-controlled trial in this study will investigate the
potential efficacy of IV infusion of ULSC in a total of 40 patients with COVID-19-related
ARDS that will all be EITHER NV or V; the determination of that eligibility criterion and the
ULSC dosing regimen will be based on Phase 1 data of safety and tolerability. Phase 2a will
evaluate EITHER single dose (one infusion) or repeat dose (two infusions separated by 48-hour
interval). The randomization will be 3:1 with 30 patients receiving investigational product
(ULSC) and 10 patients receiving placebo (carrier control).
Biological: Umbilical Cord Lining Stem Cells (ULSC)
IV infusion of allogeneic ULSC (100 million cells per dose) in sterile saline for injection
Other Name: Umbilical Cord Lining Stem Cells (ULSC), type of mesenchymal stem cells (MSC) derived from single donor umbilical cord tissue for allogeneic use
Other: Placebo (carrier control)
IV infusion of carrier control consisting of sterile saline for injection
Inclusion Criteria:
1. Adult, male or female, age ≥18 years old
2. Diagnosis of the presence of the COVID-19 agent with confirmation of COVID-19 by
standard reverse transcriptase polymerase chain reaction (RT-PCR) or equivalent test.
3. Patient with diagnosis of COVID-related ARDS, classified as either:
- Not requiring mechanical ventilation (NV) or
- Requiring mechanical ventilation (V).
According to Berlin Definition of Acute Respiratory Distress Syndrome (ARDS), patients
will be categorized based on degrees of hypoxemia [arterial partial pressure of oxygen
(PaO2)/oxygen concentration (FiO2)]:
- Mild ARDS: 200 mm Hg < PaO2/FIO2 ≤ 300 mm Hg
- Moderate ARDS: 100 mm Hg < PaO2/FIO2 ≤ 200 mm Hg
- Severe ARDS: PaO2/FIO2 ≤ 100 mm Hg
4. Patient who has exhibited deterioration in condition during the past 72 hours prior to
the informed consent.
5. Patient receiving standard of care in-hospital therapy, including appropriate critical
oxygenation, fluid, and hemodynamic support as indicated clinically.
6. Patient or responsible family member or surrogate signs informed consent.
Exclusion Criteria
1. Hypersensitivity to study product components. History of hypersensitivity to dimethyl
sulfoxide (DMSO).
2. Active cancer or prior diagnosis of cancer within the past year; however, patients
with basal and squamous cell cancer of skin will not be excluded.
3. Organ transplant recipient.
4. Chronic renal failure being treated by renal replacement therapy (dialysis) before
development of COVID-19.
5. Any other condition that, in the judgment of the Investigator or Sponsor, would be a
contraindication to enrollment, study product administration, or follow-up.
6. Pregnancy or lactation; a negative pregnancy test between screening and day 1 (before
administration of treatment) will be required of women with childbearing potential,
and they will be advised of the requirement to use an effective means of
contraception. A woman is considered to be of childbearing potential unless she is
postmenopausal and without menses for 12 months or without a uterus and/or both
ovaries.
Miami Baptist Hospital
Miami, Florida, United States
Sanford Research
Sioux Falls, South Dakota, United States