ARDS, HumanPhase 1/2EXIT-ARDSNCT05127122
Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles Infusion Treatment for ARDS
A Phase 1/2 study testing Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles and Saline in people with ARDS, Human, run by Direct Biologics, LLC.
Trial summary
To evaluate the safety and efficacy of intravenous(IV) administration of bone marrow mesenchymal stem cell derived extracellular vesicles(EV), ExoFlo, as treatment for Acute Respiratory Distress Syndrome. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- ARDS, Human
- What is being tested?
- Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles and Saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Dec 2024to Dec 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Provision of signed and dated informed consent form (either by the individual or by the individual's healthcare proxy).
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female aged 18-85.
- Moderate to severe ARDS as defined by modified Berlin definition (See Section 2.2.1), which includes timing within 1 week of known clinical insult or new or worsening respiratory symptoms; bilateral opacities not fully explained by effusions, or lung collapse; respiratory failure not fully explained by cardiac failure or fluid overload; PaO2/FiO2 ≤ 200 mm Hg.
- Hypoxia requiring noninvasive oxygen support such as Nasal Cannula (NC), Nonrebreather (NRB), Bilevel Positive Airway Pressure (BIPAP), Continuous Positive Airway Pressure (CPAP), high flow nasal cannula oxygen (HFNC O2) or mechanical ventilation (MV) despite initiating standard of care.
- If the candidate is either a male or female of reproductive potential, he or she must agree to use of double barrier method of highly effective birth control contraception such as condoms with oral contraceptive pill or choose to remain abstinent if already practicing abstinence during the screening period. The required duration of usage of double barrier method OR maintenance of abstinence must include the time from the beginning of the screening period until 90 days following the last dose of the study treatment.
Who cannot join
- Vulnerable populations such as pregnant patients, children, individuals with severe physical or mental disabilities who cannot provide meaningful consent.
- Active malignancy requiring treatment within the last five years.
- Major physical trauma in the last 2 days, including motor vehicle accidents, assaults, mechanical falls with sequelae of significant bleeding or craniofacial bruising, and surgeries, such that not one or more injury may be undiagnosed at time of screening.
- Diagnosis or suspected diagnosis of sepsis without basic microbiology cultures (urinalysis, urine culture, two sets of blood cultures, respiratory culture, at least a standard respiratory viral panel PCR) being collected prior to screening. (Note: Standard respiratory viral panel is defined by at least PCR testing for Adenovirus, Influenza A, Influenza B, Parainfluenza 1-3, Rhinovirus/Enterovirus, and RSV.)
- Duration of mechanical ventilation exceeds 5 days or 120 hours.
- Severe pre-existing organ dysfunction prior to admission, as evidenced by being currently listed on one or more organ transplant list, or intermittent Hemodialysis (HD) or Peritoneal Dialysis.
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- Presence of severe hematologic disorder or coagulopathy, such as Disseminated Intravascular Coagulopathy, as evidenced by the activation of a Massive Transfusion Protocols (as defined by the rapid administration of packed Red Blood Cells, Fresh Frozen Plasma and Platelets in fixed ratios, including at least 6 packed Red Blood Cells, within a 24 hour period) within 72 hours or frequent thrombosis of catheters (as specified by replacing 2 catheters or more within a 72 hour period of new placement) within 72 hours.
- Severe anemia or myelodysplastic syndrome requiring more than 10 units of packed Red Blood Cells transfused within the last 12 months.
- Patient is currently connected to Extracorporal Membrane Oxygenation.
About this study
Official title: Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles Infusion Treatment for Acute Respiratory Distress Syndrome: A Phase I/II Clinical Trial
To evaluate the safety and efficacy of intravenous(IV) administration of bone marrow mesenchymal stem cell derived extracellular vesicles(EV), ExoFlo, as treatment for Acute Respiratory Distress Syndrome.
Read the detailed description
To evaluate the safety and efficacy of intravenous(IV) administration of bone marrow mesenchymal stem cell-derived extracellular vesicles (EV), ExoFlo, as treatment for Acute Respiratory Distress Syndrome. ExoFlo is also referred to as Investigational Product (IP) throughout the protocol. The duration of the study is 60 days, and the endpoints are as follows:
Primary Endpoint:
- The incidence of serious adverse events.
- All-Cause Mortality at 28 days.
Secondary Endpoints:
- Ventilator-free days at 28days.
- Improvement in partial pressure of arterial oxygen to fraction of inspired oxygen (PaO2/FiO2) ratio from pre-infusion baseline(Day 0)to Day 7.
The study population will consist of 81 inpatient adult patients between 18 and 85 years of age with moderate to severe ARDS as defined by modified Berlin's criteria. Patients will be randomized via Interactive Response Technology to one of the following 3 treatment arms:
- PLACEBO: Normal saline 100 mL
- EXPERIMENTAL: IP 10 mL mixed with Normal Saline 90 mL
- EXPERIMENTAL: IP 15 mL mixed with Normal Saline 85 mL
- Conditions
- ARDS, Human
- Being tested
- Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles, Saline
- Keywords
- ExoFlo, EXIT-ARDS, ARDS, Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles, Direct Biologics
- Medical subject headings
- Respiratory Distress Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bone Marrow Mesenchymal Stem Cell Derived Extracellular VesiclesDrug
Also called ExoFlo
Injectable
SalineOther
Placebo Saline
| Study group | Role | What they receive |
|---|---|---|
| Placebo Saline | Placebo comparator | Saline Receives: Other: Saline |
| 10mL Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles | Experimental | ExoFlo (Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles) Receives: Drug: Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles |
| 15mL Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles | Experimental | ExoFlo (Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles) Receives: Drug: Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of serious adverse events.
Measured: 60 days
Number to patients with All-Cause Mortality at 28 days
Measured: 28 days
Secondary outcomes (2)
Ventilator-free days at 28days
Measured: 28 days
Change in partial pressure of arterial oxygen to fraction of inspired oxygen (PaO2/FiO2) ratio from pre-infusion baseline(Day 0)to Day 7.PaO2 may be calculated from arterial blood gas (ABG)or imputed from the SpO2daily.
Measured: 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2021
- Study startDec 2024 (estimated)
- Primary completionDec 2024 (estimated)
- Study completionDec 2024 (estimated)
Why it stopped: Change in development plan
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ARDS, HumanStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Direct Biologics, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bill Arana, Study director, Direct Biologics, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05127122
- Sponsor's protocol ID
- DB-EF-EXITARDS-0003
- Acronym
- EXIT-ARDS
- Status last verified
- Jul 2024
- First submitted
- 9 Nov 2021
- Last updated
- 29 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles Infusion Treatment for Acute Respiratory Distress Syndrome: A Phase I/II Clinical Trial
- ClinicalTrials.gov ID
- NCT05127122
- Acronym / sponsor ID
- EXIT-ARDS / DB-EF-EXITARDS-0003
- Conditions
- ARDS, Human
- Keywords
- ExoFlo, EXIT-ARDS, ARDS, Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles, Direct Biologics
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Change in development plan
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Direct Biologics, LLCIndustry (pharma, biotech, devices)
- Study start
- Dec 2024 (estimated)
- Primary completion
- Dec 2024 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 19 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the safety and efficacy of intravenous(IV) administration of bone marrow mesenchymal stem cell derived extracellular vesicles(EV), ExoFlo, as treatment for Acute Respiratory Distress Syndrome.
Detailed description
To evaluate the safety and efficacy of intravenous(IV) administration of bone marrow mesenchymal stem cell-derived extracellular vesicles (EV), ExoFlo, as treatment for Acute Respiratory Distress Syndrome. ExoFlo is also referred to as Investigational Product (IP) throughout the protocol. The duration of the study is 60 days, and the endpoints are as follows:
Primary Endpoint:
- The incidence of serious adverse events.
- All-Cause Mortality at 28 days.
Secondary Endpoints:
- Ventilator-free days at 28days.
- Improvement in partial pressure of arterial oxygen to fraction of inspired oxygen (PaO2/FiO2) ratio from pre-infusion baseline(Day 0)to Day 7.
The study population will consist of 81 inpatient adult patients between 18 and 85 years of age with moderate to severe ARDS as defined by modified Berlin's criteria. Patients will be randomized via Interactive Response Technology to one of the following 3 treatment arms:
- PLACEBO: Normal saline 100 mL
- EXPERIMENTAL: IP 10 mL mixed with Normal Saline 90 mL
- EXPERIMENTAL: IP 15 mL mixed with Normal Saline 85 mL
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo Saline | Placebo comparator | Other: SalineSaline |
| 10mL Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles | Experimental | Drug: Bone Marrow Mesenchymal Stem Cell Derived Extracellular VesiclesExoFlo (Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles) |
| 15mL Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles | Experimental | Drug: Bone Marrow Mesenchymal Stem Cell Derived Extracellular VesiclesExoFlo (Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles) |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Provision of signed and dated informed consent form (either by the individual or by the individual's healthcare proxy).
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female aged 18-85.
- Moderate to severe ARDS as defined by modified Berlin definition (See Section 2.2.1), which includes timing within 1 week of known clinical insult or new or worsening respiratory symptoms; bilateral opacities not fully explained by effusions, or lung collapse; respiratory failure not fully explained by cardiac failure or fluid overload; PaO2/FiO2 ≤ 200 mm Hg.
- Hypoxia requiring noninvasive oxygen support such as Nasal Cannula (NC), Nonrebreather (NRB), Bilevel Positive Airway Pressure (BIPAP), Continuous Positive Airway Pressure (CPAP), high flow nasal cannula oxygen (HFNC O2) or mechanical ventilation (MV) despite initiating standard of care.
Key exclusion criteria (9 listed)
- Vulnerable populations such as pregnant patients, children, individuals with severe physical or mental disabilities who cannot provide meaningful consent.
- Active malignancy requiring treatment within the last five years.
- Major physical trauma in the last 2 days, including motor vehicle accidents, assaults, mechanical falls with sequelae of significant bleeding or craniofacial bruising, and surgeries, such that not one or more injury may be undiagnosed at time of screening.
- Diagnosis or suspected diagnosis of sepsis without basic microbiology cultures (urinalysis, urine culture, two sets of blood cultures, respiratory culture, at least a standard respiratory viral panel PCR) being collected prior to screening. (Note: Standard respiratory viral panel is defined by at least PCR testing for Adenovirus, Influenza A, Influenza B, Parainfluenza 1-3, Rhinovirus/Enterovirus, and RSV.)
- Duration of mechanical ventilation exceeds 5 days or 120 hours.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bone Marrow Mesenchymal Stem Cell Derived Extracellular VesiclesAlso: ExoFloInjectable | Drug | 10mL Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles, 15mL Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles |
| SalinePlacebo Saline | Other | Placebo Saline |
Primary outcome
The incidence of serious adverse events.
Time frame: 60 days
Number to patients with All-Cause Mortality at 28 days
Time frame: 28 days
2 secondary outcomes
Ventilator-free days at 28days
Time frame: 28 days
Change in partial pressure of arterial oxygen to fraction of inspired oxygen (PaO2/FiO2) ratio from pre-infusion baseline(Day 0)to Day 7.PaO2 may be calculated from arterial blood gas (ABG)or imputed from the SpO2daily.
Time frame: 7 days
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bill Arana study director
Direct Biologics, LLC
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles and Saline in people with ARDS, Human, run by Direct Biologics, LLC.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 29 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of serious adverse events. (time frame: 60 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2024.