VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

PHASE2/PHASE3CompletedINTERVENTIONAL
Enrollment

56

Participants

Timeline

Start Date

October 31, 2015

Primary Completion Date

March 28, 2018

Study Completion Date

March 28, 2018

Conditions
Niemann-Pick Disease, Type C
Interventions
DRUG

Parts A/B: Adrabetadex

900 - 1800 milligram (mg) of adrabetadex administered every 2 weeks via lumbar intrathecal infusion

OTHER

Parts A/B: Sham Control

No experimental drug is administered to participants - intrathecal administrations are simulated by skin prick

Trial Locations (29)

2031

The Prince of Wales Hospital, Sydney

3050

Royal Melbourne Hospital, Parkville

3168

Monash Medical Centre, Clayton

3204

Waikato Hospital, Hamilton West

6000

Royal Perth Hospital, Perth

10467

Montefiore Medical Center, The Bronx

18101

Lehigh Valley Health Network, Allentown

19104

The Children's Hospital of Philadelphia, Philadelphia

27514

University of North Carolina, Chapel Hill

32608

Shands Children's Hospital, Gainesville

35223

University of Alabama at Birmingham, Birmingham

44791

Katholisches Klinikim Bochum gGmbH, Bochum

48149

Universitaetsklinikum Muenster, Münster

55131

Universitaetsklinikum Mainz, Mainz

60612

Rush University Medical Center, Chicago

78723

Dell Children's Medical Center of Central Texas, Austin

80045

University of Colorado Denver, Aurora

92867

Children's Hospital of Orange County, Orange

98405

Multicare Institute for Research and Innovation, Tacoma

119074

National University Hospital (Singapore) Pte, Ltd, Singapore

94143-0780

University of California San Francisco, San Francisco

20892-2425

Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda

02115

Boston Children's Hospital, Boston

75 571

CHU Paris Est - Hospital d'Enfants Armand-Trousseau, Paris

08035

Hospital Universitario del Valle Hebron, Barcelona

06230

Hacettepe University Medical Faculty, Altındağ

06570

Gazi University Medical Faculty, Çankaya

B4 6NH

Birmingham Women's and Children's NHS Trust, Birmingham

WC1N 3JH

Great Ormond Street Hospital, London

All Listed Sponsors
lead

Mandos LLC

INDUSTRY

NCT02534844 - VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease | Biotech Hunter | Biotech Hunter