A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

PHASE2RecruitingINTERVENTIONAL
Enrollment

72

Participants

Timeline

Start Date

November 22, 2024

Primary Completion Date

March 31, 2027

Study Completion Date

September 30, 2041

Conditions
Short Stature Homeobox- Containing Gene SHOX DeficiencyNoonan SyndromeTurner Syndrome
Interventions
DRUG

Vosoritide Injection

Modified recombinant human C-type natriuretic peptide Vosoritide

DRUG

Human Growth Hormone

Commercial product containing somatotropin.

Trial Locations (20)

10029

RECRUITING

Icahn School of Medicine at Mount Sinai, New York

10467

RECRUITING

Children's Hospital at Montefiore, The Bronx

13385

RECRUITING

Hôpital de la Timone, Marseille

16147

NOT_YET_RECRUITING

IRCCS Istituto Giannina Gaslini, Genova

19803

RECRUITING

Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children), Wilmington

20010

RECRUITING

Children's National Medical Center, Washington D.C.

28203

RECRUITING

Atrium Health Carolinas Medical Center, Charlotte

33011

NOT_YET_RECRUITING

Central University Hospital of Asturias, Oviedo

33155

NOT_YET_RECRUITING

Nicklaus Children's Hospital, Miami

40506

RECRUITING

Kentucky Children's Hospital, Lexington

55454

RECRUITING

M Health Fairview Pediatric Specialty Clinic - Explorer, Minneapolis

75019

RECRUITING

Hôpital Robert-Debré, Paris

75235

RECRUITING

Children's Medical Center Dallas, Dallas

77030

RECRUITING

University of Texas Health Science Center at Houston (UT Health), Houston

80045

RECRUITING

Children's Hospital Colorado, Aurora

83712

RECRUITING

St. Luke's Children's Endocrinology and Diabetes, Boise

94270

RECRUITING

South Paris University Hospitals - Bicetre Hospital, Le Kremlin-Bicêtre

02115

RECRUITING

New York Medical College, Boston

49 933

RECRUITING

Angers University Hospital Center, Angers

Unknown

RECRUITING

CHU de Toulouse - Hôpital des Enfants, Toulouse

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY