ClinicalMetric tracks all active clinical trials for rare genetic disorders sourced from ClinicalTrials.gov. Research in this area spans early-phase safety investigations through large Phase 3 confirmatory trials, conducted by NIH-funded academic centers, independent investigators, and pharmaceutical sponsors worldwide. Each listing is updated daily as new studies open enrollment and existing ones reach capacity or complete follow-up.
Trial listings include the NCT identifier, current recruitment status, phase classification, primary and secondary endpoints, estimated enrollment size, participating countries, and direct links to the full protocol on ClinicalTrials.gov. Eligibility criteria — including age range, disease stage, and prior treatment requirements — are documented for every study to help patients and clinicians assess suitability before contacting a trial site.
Frequently Asked Questions — rare genetic disorders Clinical Trials
How many clinical trials are currently recruiting for rare genetic disorders?
ClinicalMetric currently tracks 1 actively recruiting clinical trials for rare genetic disorders, sourced in real time from ClinicalTrials.gov. The total number of registered studies—including those not yet enrolling or in active follow-up—is 1. Trial availability changes daily as new studies open enrollment and existing ones reach capacity.
What trial phases are available for rare genetic disorders?
rare genetic disorders research spans multiple clinical trial phases. Phase 1 studies evaluate safety and dosing in small groups, Phase 2 studies assess preliminary efficacy in 100–300 participants, and Phase 3 trials compare the new treatment against the standard of care in 300–3,000+ patients. Phase 4 post-approval studies monitor long-term outcomes in real-world populations.
How do I find out if I qualify for a rare genetic disorders clinical trial?
Eligibility criteria for rare genetic disorders trials vary by study and typically specify age range, disease stage or severity, prior treatment history, and specific diagnostic or laboratory parameters. Each listing on ClinicalMetric links to the full protocol on ClinicalTrials.gov, where inclusion and exclusion criteria are documented. Contact the sponsoring site's research coordinator directly to confirm your eligibility—your treating physician or specialist can also help identify the most appropriate trial based on your medical history and current treatment status.
Top Sponsors
Bahçeşehir University 1 trial
Recruiting Clinical Trials
ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional.
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Last Reviewed: April 2026 ·
Data Methodology