Microbiome Clinical Trials & Research Studies
Clinical Trial Landscape: As of 2024, there are over 800 active clinical trials investigating microbiome interventions worldwide. These studies span multiple therapeutic areas and represent a $2.3 billion investment in microbiome-based medicine development.
Current Phase III Clinical Trials
Phase III trials represent the most advanced microbiome interventions, testing safety and efficacy in large patient populations before potential FDA approval:
| Study Name | Sponsor | Indication | Intervention | Enrollment | Status |
|---|---|---|---|---|---|
| CORAL Study | Seres Therapeutics | Recurrent C. difficile | SER-109 (spore-based) | 320 patients | Completed |
| VOWELS Study | Vedanta Biosciences | C. difficile Prevention | VE303 (defined consortium) | 374 patients | Recruiting |
| GUARDIAN Study | Rebiotix (Ferring) | C. difficile Recurrence | RBX2660 (microbiota) | 267 patients | Completed |
| PRISM-UC Study | Finch Therapeutics | Ulcerative Colitis | CP101 (microbiome therapy) | 206 patients | Active |
| MELODY Study | Enterome | Crohn's Disease | EB8018 (FimH antagonist) | 412 patients | Recruiting |
Phase II Clinical Trials by Therapeutic Area
Inflammatory Bowel Disease
Leading Studies:
- MODIFY Study: 4D Pharma's MRx0518 in Crohn's disease (180 patients)
- HARMONY Study: Evelo Biosciences' EDP1815 in atopic dermatitis (289 patients)
- RESTORE Study: Assembly Biosciences' ABI-M201 in ulcerative colitis (156 patients)
Approach: Most trials use rationally-designed bacterial consortiums or single-strain probiotics targeting specific inflammatory pathways.
Metabolic Disorders
Key Trials:
- SWEET Study: Akkermansia muciniphila in metabolic syndrome (134 subjects)
- ENGAGE Study: Synlogic's SYNB1618 for phenylketonuria (PKU) (85 patients)
- PRECISION Study: Kaleido Biosciences' KB195 in diabetes (198 patients)
Mechanism: Targeting metabolic pathways through engineered bacteria or beneficial microbes that modulate glucose metabolism and inflammation.
Cancer Immunotherapy
Notable Studies:
- OPTIMIZE Study: Microbiome modulation plus checkpoint inhibitors (225 patients)
- ENHANCE Study: Evelo's EDP1503 in solid tumors (76 patients)
- KEYNOTE-958: FMT plus pembrolizumab in melanoma (40 patients)
Rationale: Enhancing anti-tumor immune responses by optimizing the gut microbiome composition to improve immunotherapy efficacy.
Innovative Phase I/II Studies
Next-Generation Microbiome Therapies
Emerging approaches represent the cutting edge of microbiome therapeutic development:
| Technology | Example Study | Institution | Innovation |
|---|---|---|---|
| Engineered Bacteria | Synlogic SYNB1020 | Yale University | Bacteria engineered to consume ammonia in liver disease |
| Phage Therapy | PhagoBiotics Trial | University of Geneva | Targeted elimination of pathogenic bacteria |
| Synthetic Biology | Lodo Therapeutics LDO-1 | Stanford University | Programmed bacteria producing therapeutic compounds |
| Precision Prebiotics | Kaleido KB109 | Harvard Medical School | Targeted feeding of beneficial bacteria |
| Microbiome Editing | Eligo Bioscience ELG-001 | Rockefeller University | CRISPR-based microbiome modification |
Pediatric Microbiome Trials
Pediatric populations require specialized approaches due to developing microbiomes and unique safety considerations:
Early Life Interventions
BABY STEPS Study (University of Calgary)
- Population: 1,654 infants
- Intervention: Bifidobacterium longum supplementation
- Primary Endpoint: Reduced allergies and asthma at age 5
- Duration: 5-year follow-up
Childhood Obesity Prevention
GROW HEALTHY Study (Boston Children's Hospital)
- Population: 342 children (ages 2-5)
- Approach: Family-based microbiome intervention
- Methods: Diet modification plus targeted probiotics
- Outcomes: BMI trajectories and metabolic markers
Autism Spectrum Disorders
MICROBIOTA Study (Arizona State University)
- Phase: II randomized controlled trial
- Participants: 84 children with ASD
- Treatment: Standardized fecal microbiota transplant
- Assessment: Behavioral and GI symptom improvements
Global Clinical Trial Landscape
Regional Distribution and Focus Areas
Geographic Distribution: North America leads with 45% of global microbiome trials, followed by Europe (32%), Asia-Pacific (18%), and other regions (5%). Different regions show distinct therapeutic focuses reflecting local health priorities and regulatory environments.
| Region | Active Trials | Primary Focus | Leading Institutions | Regulatory Status |
|---|---|---|---|---|
| United States | 285 | Cancer, IBD, C. difficile | NIH, Mayo Clinic, Johns Hopkins | FDA fast-track designations |
| European Union | 156 | Metabolic disorders, allergies | Karolinska, University of Copenhagen | EMA novel therapy pathways |
| China | 89 | Hepatitis, diabetes | Chinese Academy of Sciences | NMPA accelerated approval |
| Japan | 43 | Aging, neurodegeneration | RIKEN, University of Tokyo | PMDA consultation program |
| Australia | 31 | Mental health, autoimmune | University of Melbourne | TGA provisional approval |
Regulatory Pathways and Approval Status
FDA-Approved Microbiome Therapies
- REBYOTA (Ferring): First FDA-approved fecal microbiota product for recurrent C. difficile (2022)
- VOWST (Seres Therapeutics): Oral spore-based microbiome therapeutic for C. difficile (2023)
- Pending Approvals: 12 microbiome therapies in FDA review for various indications
Fast Track Designations
FDA has granted Fast Track status to multiple microbiome therapies:
- VE303 for C. difficile prevention
- EDP1815 for atopic dermatitis
- SYNB1618 for phenylketonuria
- KB195 for hepatic encephalopathy
Orphan Drug Designations
Several microbiome therapies have received orphan drug status for rare diseases:
- SYNB1020 for hyperammonemia
- AG019 for oral mucositis
- MET-2 for homocystinuria
- ELX-02 for genetic hearing loss
Clinical Trial Design Considerations
Unique Challenges in Microbiome Clinical Trials
Methodological Innovations
Microbiome clinical trials require specialized approaches to address unique challenges:
- Baseline Microbiome Profiling: Stratification based on pre-treatment microbiome composition
- Adaptive Trial Designs: Modification of interventions based on interim microbiome analysis
- Precision Endpoints: Biomarker-driven outcomes rather than traditional clinical measures
- Longitudinal Sampling: Multiple timepoints to capture microbiome dynamics
- Multi-omics Integration: Combining microbiome, metabolome, and host response data
Standardization Efforts
The field is moving toward standardized protocols to improve reproducibility and comparison between studies:
Sample Collection Standards
- Standardized collection protocols
- Cold-chain maintenance requirements
- Quality control metrics
- Contamination prevention measures
Analytical Harmonization
- Common sequencing platforms
- Standardized bioinformatics pipelines
- Reference databases
- Statistical analysis frameworks
Patient Recruitment and Participation
Current Recruitment Challenges
Microbiome clinical trials face unique recruitment challenges that researchers are actively addressing:
Recruitment Barriers: Many microbiome trials struggle with recruitment due to dietary restrictions, frequent sampling requirements, and patient concerns about microbiome interventions. Novel recruitment strategies are being developed to address these challenges.
Digital Recruitment Platforms
- Microbiome-specific patient registries
- Mobile apps for screening and enrollment
- Social media recruitment campaigns
- Electronic health record integration
Patient Engagement Strategies
- Home-based sample collection kits
- Real-time microbiome feedback to participants
- Patient education programs
- Flexible visit scheduling
Diversity and Inclusion Initiatives
The field recognizes the critical need for diverse participant populations to ensure microbiome therapies work across different genetic and cultural backgrounds:
- Global Microbiome Diversity Project: Ensuring representation from underrepresented populations
- Cultural Competency Training: Research staff training on cultural factors affecting microbiome studies
- Community Partnership Programs: Collaborations with community health centers
- Multilingual Study Materials: Translation of consent forms and patient materials
Future Clinical Trial Trends
Combination Therapies
Future trials increasingly test microbiome interventions combined with standard treatments:
- Microbiome therapy + immunotherapy
- Probiotics + antibiotics
- FMT + dietary interventions
- Microbiome editing + gene therapy
Personalized Approaches
Precision microbiome medicine trials are emerging:
- Individualized bacterial consortiums
- Genotype-guided microbiome interventions
- AI-powered treatment selection
- Real-time microbiome monitoring
Prevention Studies
Shift toward preventive microbiome interventions:
- Early-life microbiome optimization
- Antibiotic-associated dysbiosis prevention
- Cancer recurrence prevention
- Healthy aging microbiome maintenance
How to Participate in Microbiome Research
Finding Clinical Trials
Patients interested in participating in microbiome clinical trials can find opportunities through multiple resources:
- ClinicalTrials.gov: Search for "microbiome," "probiotics," or "fecal transplant"
- Institutional Research Centers: Contact major academic medical centers
- Patient Advocacy Groups: Disease-specific organizations often maintain trial listings
- Microbiome Companies: Many sponsor patient registries for future studies
- Healthcare Providers: Physicians can help identify relevant trials
Considerations Before Participating
Important Questions to Ask
- What are the potential risks and benefits?
- How long is the study duration?
- What samples or tests are required?
- Are there dietary or lifestyle restrictions?
- Will I receive results from my microbiome analysis?
- What happens to my samples after the study?
- Is there a placebo group?
- What are the study visit requirements?
Clinical Trial Disclaimer: This information is for educational purposes only. Clinical trial participation should always be discussed with healthcare providers. Trial designs, enrollment status, and results may change. Always verify current information through official study sources and clinical trial databases.
Medical Disclaimer: The information provided on this website is for educational purposes only and should not be considered medical advice. Always consult with healthcare professionals for medical concerns and before making changes to your health regimen.