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Clinical trials

Completed Phase 2 Rare Diseases trials

Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.

Filters: Condition: Rare Diseases Phase: Phase 2 Status: Completed Clear all

70 trials match Source: ClinicalTrials.gov registrations

Trials started per year

By phase, 2000 to 2026.

Trials started per year
CategoryTrials
20003
20012
20061
20131
20141
20151
20161
20191
20221

Status

Trials by registry status. Select a bar to filter.

Trials by status
CategoryTrials
Completed70

Top lead sponsors

A sponsor with a page on Pharma Observatory links to it.

Top lead sponsors
CategoryTrials
National Center for Research Resources (NCRR)33
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)2
Johns Hopkins University2
National Institute of Allergy and Infectious Diseases (NIAID)2
National Institute of Neurological Disorders and Stroke (NINDS)2
Boston University1
Charles Drew University of Medicine and Science1
Children's Hospital Medical Center, Cincinnati1
Children's Hospital of Philadelphia1
Emory University1

Top conditions

MeSH conditions the trials were registered with, not their broader categories. A condition with a page on Pharma Observatory links to it.

Top conditions
CategoryTrials
Rare Diseases70
Genetic Diseases, Inborn11
Mental Disorders11
Neurologic Manifestations11
Hematologic Diseases10
Communicable Diseases9
Connective Tissue Diseases9
Immune System Diseases9
Arthritis8
Metabolism, Inborn Errors7

Trials

Showing 1 to 25 of 70. Each trial links to its page on Pharma Observatory.

Recently changed trials

Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.

No changes to these trials in the last 7 days.

Go deeper on Pharma Observatory

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