Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
NCT ID: NCT04958642
Last Updated: 2023-08-29
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2/PHASE3
66 participants
INTERVENTIONAL
2015-12-23
2022-04-11
Brief Summary
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This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords).
In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control).
In Part C, all participants will receive study drug.
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Adrabetadex
All participants receive their prescribed dose of adrabetadex. Dose is allowed to be adjusted down to a minimum of 400 milligrams (mg) or up to a maximum of 900 mg, at the investigator's discretion.
Adrabetadex
Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.
Interventions
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Adrabetadex
Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Has agreed to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2 weeks dosing regimen
* The investigator has received prior written authorization from the sponsor for the participant to enter VTS301 Part C on an amended dose and/or regimen
* Has received prior written authorization from Vtesse to enroll directly into Part C
4 Years
21 Years
ALL
No
Sponsors
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Mandos LLC
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Study Lead
Role: STUDY_DIRECTOR
Mandos LLC
Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Children's Hospital of Orange County: CHOC Children's
Orange, California, United States
University of California San Francisco
San Francisco, California, United States
University of Colorado Denver
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Shands Children's Hospital
Gainesville, Florida, United States
Rush University Medical Center
Chicago, Illinois, United States
Eunice Kennedy Shriver National Institute of Child Health and Human Development
Bethesda, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Montefiore Medical Center
The Bronx, New York, United States
University of North Carolina
Chapel Hill, North Carolina, United States
Lehigh Valley Health Network
Allentown, Pennsylvania, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Dell Children's Medical Center of Central Texas
Austin, Texas, United States
Multicare Institute for Research and Innovation
Tacoma, Washington, United States
The Prince of Wales Hospital
Randwick, New South Wales, Australia
Monash Medical Centre
Clayton, Victoria, Australia
Royal Melbourne Hospital
Melbourne, Victria, Australia
Royal Perth Hospital
Perth, Western Australia, Australia
CHU Paris Est - Hopital d'Enfants Armand-Trousseau
Paris, Cedex 12, France
Katholisches Klinikim Bochum gGmbH
Bochum, , Germany
Universitaetsklinikum Mainz
Mainz, , Germany
Universitaetsklinikum Muenster
Münster, , Germany
Waikato Hospital
Hamilton, , New Zealand
National University Hospital (Singapore) Pte, Ltd
Singapore, , Singapore
INSELSPITAL, Universitätsspital Bern
Bern, , Switzerland
Gazi University Medical Faculty
Ankara, , Turkey (Türkiye)
Hacettepe University Medical Faculty
Ankara, , Turkey (Türkiye)
Birmingham Children's Hospital NHS Foundation Trust
Birmingham, , United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2015-002548-15
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VTS301 (Part C)
Identifier Type: -
Identifier Source: org_study_id
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