Study of Cord Blood Derived Mesenchymal Stem Cells for Treatment of Moderate, Severe or Critical Pneumonia

Plain English Summary

Study of Cord Blood Derived Mesenchymal Stem Cells for Treatment of Moderate, Severe or Critical Pneumonia is a Phase 2 clinical trial sponsored by M.D. Anderson Cancer Center studying COVID-19 Infection, COVID-19-Associated Acute Respiratory Distress Syndrome, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm, Symptomatic COVID-19 Infection Laboratory-Confirmed. This trial tests if stem cells from umbilical cords can help patients with moderate to critical pneumonia, including that caused by COVID-19. It is for adults (18+) with pneumonia showing signs of moderate to severe illness, confirmed by chest X-ray and oxygen levels. Participants will receive either standard care or standard care plus an infusion of stem cells, and will be closely monitored. Alternative treatments include standard medical care for pneumonia and COVID-19, and other investigational therapies. The trial aims to enroll 70 participants.

Official Summary

This is a phase I trial followed by a phase II randomized trial. The purpose of phase I study is the feasibility of treating patients with acute respiratory distress syndrome (ARDS) related to COVID-19 infection (COVID-19) with cord blood-derived mesenchymal stem cells (MSC). The purpose of the phase II trial is to compare the effect of MSC with standard of care in these patients. MSCs are a type of stem cells that can be taken from umbilical cord blood and grown into many different cell types that can be used to treat cancer and other diseases. The MSCs being used for infusion in this trial are collected from healthy, unrelated donors and are stored and grown in a laboratory. Giving MSC infusions may help control the symptoms of COVID-19 related ARDS.

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18 or older with pneumonia and breathing difficulties (fast breathing or low oxygen). Patients with COVID-19 pneumonia must meet specific severity criteria. Individuals with severe liver disease, pregnant or breastfeeding women, and those on life support (ECMO) cannot participate. Patients not expected to survive more than 48 hours are also excluded. This trial is studying COVID-19 Infection, COVID-19-Associated Acute Respiratory Distress Syndrome, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm, Symptomatic COVID-19 Infection Laboratory-Confirmed, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The main goal is to see if patients receiving stem cells are alive and free from severe side effects within 30 days, indicating the treatment is safe and potentially effective. The specific primary outcome measures are: Incidence of composite serious adverse events (Phase I) (Within 30 days of the first mesenchymal stem cell (MSC) infusion); Patients alive without grade 3, 4 infusional toxicity (Phase II) (At day 30 post MSC infusion); Patients alive with grade 3 or 4 infusional toxicity (Phase II) (At day 30 post MSC infusion); Patients not alive (Phase II) (At day 30 post MSC infusion). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 2, which tests whether the treatment actually works against the target condition. Phase 2 trials involve 100-300 patients and continue to monitor safety while evaluating effectiveness. This phase often tests different dosages to find the optimal amount. About 33% of Phase 2 drugs advance to Phase 3. If successful, the treatment will move to large-scale Phase 3 trials needed for FDA approval.

Why This Trial Matters

This trial addresses the need for new treatments for severe pneumonia, particularly ARDS caused by COVID-19, by investigating the potential of stem cells to reduce inflammation and improve lung functi Phase 2 success would typically lead to larger Phase 3 trials needed for regulatory approval. This research targets COVID-19 Infection, COVID-19-Associated Acute Respiratory Distress Syndrome, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm, Symptomatic COVID-19 Infection Laboratory-Confirmed, where improved treatment options are needed.

Investor Insight

This trial explores a novel cell-based therapy for a significant unmet need in respiratory illness, with potential applications beyond COVID-19, suggesting a future market for regenerative medicine in Phase 2 trials have approximately a 15-20% chance of eventually gaining FDA approval.

Is This Trial Right for Me?

Ask your doctor about the potential benefits and risks of stem cell therapy compared to standard treatments. Understand that you will receive either standard care or standard care plus stem cell infusion, and will be monitored for side effects. Participation involves regular check-ups and medical assessments to track your progress and any adverse events. This trial is currently recruiting participants. The trial is being conducted at 1 site. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: RANDOMIZED
• Model: PARALLEL
• Masking: NONE
• Enrollment: 70 participants

Interventions

• OTHER: Best Practice — Receive standard of care
• BIOLOGICAL: Mesenchymal Stem Cell — Given IV

Primary Outcomes

• Incidence of composite serious adverse events (Phase I) (Within 30 days of the first mesenchymal stem cell (MSC) infusion)
• Patients alive without grade 3, 4 infusional toxicity (Phase II) (At day 30 post MSC infusion)
• Patients alive with grade 3 or 4 infusional toxicity (Phase II) (At day 30 post MSC infusion)
• Patients not alive (Phase II) (At day 30 post MSC infusion)

Secondary Outcomes

• Proportion of successfully extubated patients who present intubated on ventilator support (Phase I) (Up to day 30 post MSC infusion)
• Rate of successful progression to intubation in patients who require supplemental oxygen but who are otherwise able to breathe without assistance (Phase I) (Up to day 30 post MSC infusion)
• Overall survival rate (Phase I) (At day 30 post MSC infusion)
• Survival rate in patients who present intubated on ventilator support (Phase I) (At day 30 post MSC infusion)
• Survival rate in patients who require supplemental oxygen but who are otherwise able to breathe without assistance (Phase I) (At day 30 post MSC infusion)

Full Eligibility Criteria

Inclusion Criteria:

1. Age greater than or equal 18 years.
2. Participants with chest x-ray findings concerning for pneumonia from any cause, with clinical signs suggestive of at least moderate illness such as respiratory rate \>20 breaths per minute or with oxygen saturation less than 93% on room air\*\*
3. Participants with COVID-19 associated pneumonia must meet baseline categorization of Moderate, Severe or Critical COVID-19 per FDA Guidance for Industry COVID-19: Developing Drugs and Biologics Products for Treatment or Prevention, February 2021.
4. Negative pregnancy test in a woman with childbearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.
5. Participant or legally authorized representative consent. Participants with diminished mental capacity may be allowed on to enroll on the study.
6. Because of the nature of COVID-19, participants enrolled on this study with COVID-19 associated pneumonia may have been previously enrolled in other IND trials for their cancer diagnosis or for COVID-19. These enrollments will not exclude them from enrollment to this study.

Exclusion Criteria:

* Moribund participants not expected to survive up to 48 hours
* Participants with severe chronic liver disease (Childs-Pugh score \> 10)
* Pregnant and/or lactating women
* Participants on extracorporeal membrane oxygenation

Trial Locations

• M D Anderson Cancer Center, Houston, Texas, United States

Frequently Asked Questions

Related Conditions

• COVID-19 Infection
• COVID-19-Associated Acute Respiratory Distress Syndrome
• Pneumonia
• Acute Respiratory Distress Syndrome (ARDS)
• Respiratory Failure
• Lung Inflammation
• Sepsis
• Critical Illness
• Hematopoietic and Lymphoid Cell Neoplasm
• Malignant Solid Neoplasm

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