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Table 7 Number of trials by conditions for females and males only and phase of study

From: Does the low prevalence affect the sample size of interventional clinical trials of rare diseases? An analysis of data from the aggregate analysis of clinicaltrials.gov

Gender

Diseases/conditions

Phase 2

Phase 3

Females only

 
 

Addison Disease

1

0

Congenital Toxoplasmosis

0

1

Gastroschisis

0

2

Hereditary Breast and Ovarian Cancer Syndrome

1

0

Lymphangioleiomyomatosis

3

0

Ondine Syndrome

0

1

Ovarian Cancer

48

11

Peripartum Cardiomyopathy

1

0

Placental Insufficiency

1

0

Preeclampsia

1

1

Rett Syndrome

5

0

Systemic Sclerosis

1

0

Turner Syndrome

0

1

Males only

 
 

Allan-Herndon-Dudley Syndrome

2

0

Barth Syndrome

1

0

Becker Muscular Dystrophy

1

0

Duchenne Muscular Dystrophy

6

2

Fabry Disease

2

0

Fragile X Syndrome

4

0

Hemophilia

0

1

Hemophilia A

1

2

Severe Hemophilia A

1

1

X-Linked Hypohidrotic Ectodermal Dysplasia

1

0