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

Primary Purpose

Niemann-Pick Disease, Type C

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Parts A/B: Adrabetadex

Parts A/B: Sham Control

About this trial

This is an interventional treatment trial for Niemann-Pick Disease, Type C focused on measuring Niemann-Pick Type C1 (NPC1) Disease, neurologic disease, gross motor dysfunction, fine motor dysfunction, dysphagia, swallowing problems, cognitive dysfunction, gait abnormalities, pediatrics

Eligibility Criteria

4 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

Parts A/B:

Had onset of neurological symptoms prior to 15 years of age
Has confirmed diagnosis of NPC1 determined by either:
1. two NPC1 mutations
2. positive filipin staining and at least one NPC1 mutation
3. vertical supranuclear gaze palsy (VSNGP) in combination with either: one NPC1 mutation, OR positive filipin staining or oxysterol levels consistent with NPC disease and no Niemann-Pick Type C2 (NPC2) Disease mutations
Adult participant or parent/guardian has provided written informed consent, with assent collected from minors of appropriate age
Is able to undergo a lumbar puncture (LP) and IT drug administration under conscious sedation or general anesthesia
Has an NPC Clinical Severity Scale Score of 1 through 4, inclusive, in two or more of the following components: ambulation, fine motor skills, or swallowing; and has a score of 0 through 4 on the cognition component
Has a total NPC Clinical Severity Scale Score of 10 or greater
If taking miglustat, must have been on a stable dose for past 6-8 weeks and be willing to remain on a stable dose
If participant has seizures, they have been adequately controlled for 3 months without changing dose or regimen
Has agreed to discontinue all non-prescription supplements at least 1 month prior to first dose (Study Day 0)
Has agreed to discontinue any other investigational treatments for NPC including vorinostat or arimoclomol at least 3 months prior to first dose (Study Day 0)
If of child-bearing potential (not surgically sterile), agrees to use a medically acceptable method continuously, until at least 30 days after participation in the study

Key Exclusion Criteria:

Has exclusion criteria as assessed by NPC Clinical Severity Scale:
1. Unable to walk, wheelchair dependent (ambulation NPC score=5)
2. Has need for a nasogastric tube to overcome swallowing difficulties (swallowing NPC score=5) unless used for supplemental feeding or administering medication
3. severe dysmetria (fine motor score =5) or
4. minimal cognitive function (cognition NPC score=5)
Weighs less than 15 kg
Has had prior treatment with intravenous 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) for NPC1 disease, unless the subject has undergone a minimum 3-month washout period prior to Study Day 0, or has had any prior intrathecal (IT) administration of HP-β-CD
Is taking antipsychotics for treatment of psychosis; use of antipsychotic medication for treatment of other disorders (e.g., Attention Deficit Hyperactivity Disorder) will not exclude participation in this trial
Has a history of hypersensitivity reactions to any product containing HP-β-CD
Has a spinal deformity that could impact the ability to perform a lumbar puncture
Has had a skin infection in the lumbar region within 2 months of study entry
Has neutropenia, defined as an absolute neutrophil count (ANC) of less than 1.5 X 10^9/L
Has thrombocytopenia (platelet count of less than 75 X 10^9/L)
Has activated partial thromboplastin time (aPTT) or prothrombin time (PT) prolonged by > 1.5 times the upper limit of normal (ULN) or known history of a bleeding disorder
Has had status epilepticus occurring within 3 months of screening and/or seizure frequency that cannot be quantified
Has evidence of either obstructive hydrocephalus or normal pressure hydrocephalus
Has recently used anticoagulants [in past 2 weeks prior to first dose (Study Day 0); re: lumbar puncture safety].
Is unable to comply with the study procedures or has a clinical disease or laboratory abnormality that in the opinion of the investigator would potentially increase the risk of participation

Sites / Locations

University of Alabama at Birmingham
Children's Hospital of Orange County
University of California San Francisco
University of Colorado Denver
Shands Children's Hospital
Rush University Medical Center
Eunice Kennedy Shriver National Institute of Child Health and Human Development
Boston Children's Hospital
Montefiore Medical Center
University of North Carolina
Lehigh Valley Health Network
The Children's Hospital of Philadelphia
Dell Children's Medical Center of Central Texas
Multicare Institute for Research and Innovation
The Prince of Wales Hospital
Monash Medical Centre
Royal Melbourne Hospital
Royal Perth Hospital
CHU Paris Est - Hospital d'Enfants Armand-Trousseau
Katholisches Klinikim Bochum gGmbH
Universitaetsklinikum Mainz
Universitaetsklinikum Muenster
Waikato Hospital
National University Hospital (Singapore) Pte, Ltd
Hospital Universitario del Valle Hebron
Hacettepe University Medical Faculty
Gazi University Medical Faculty
Birmingham Women's and Children's NHS Trust
Great Ormond Street Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Other

Arm Label

Parts A/B: Sham Control

Parts A/B: Adrabetadex

Arm Description

Participants receive no study drug

Participants receive adrabetadex

Outcomes

Primary Outcome Measures

Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score

Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52

The Clinician CGIC is a 7-point Likert scale. The scale requires assessment of change from a baseline level of disease activity, with anchors ranging from markedly improved, moderately improved, and minimally improved to no change and corresponding worsening (minimally, moderately, markedly). The Investigator rates his/her impression of the change in each participant's condition at week 52 on a scale from marked improvement (1) to marked worsening (7).

Secondary Outcome Measures

Parts A/B: Change From Baseline to Week 52 in NPC-SS Total Score (Excluding Hearing and Auditory Brainstem Response [ABR])

The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. The hearing domain and auditory brainstem response modifiers are removed from the total NPC-SS total score for this measure. A Likert-like scale is used to assign the remaining 8 major domain scores of 0 to 5 (better to worse). The total score was the sum of individual component scores which ranges from 0 (best) to 40 (worst), with higher scores indicating more severe clinical impairment.

Parts A/ B: Number of Participants Classified as CGIC Responders at Week 52

Parts A/B: Number of Participants Classified as Responders on NPC-SS Total Score (Excluding Hearing and ABR) at Week 52

The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. The hearing domain and ABR modifiers are removed from the total NPC-SS total score for this measure. A Likert-like scale is used to assign the remaining 8 major domain scores of 0 to 5 (better to worse). The total score was the sum of individual components scores which ranges from 0 (best) to 40 (worst), with higher scores indicating more severe clinical impairment. Responders on NPC-SS Total Score are defined as participants with no change or improvement on NPC-SS total score from baseline to Week 52.

Parts A/B: EQ-5D-3L Questionnaire Visual Analog Scale (VAS) Score (for Health Status) at Baseline and at Week 52

The EQ-5D-3L assessment is a self-reported, simple, descriptive system measuring 5 dimensions including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. A vertical VAS allows the participants to indicate their health state that day, and ranges from 0 (worst imaginable) to 100 (best imaginable), with higher scores indicating better health state.

Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option

Participants who manifested significant disease progression according to predefined clinical criteria after treatment of 26 weeks or more had the option to rescue. Number of participants who qualified for the rescue option following a minimum of 26 weeks of treatment were analyzed.

Parts A/B: Change From Baseline to Week 52 in Each of the 9 Clinical Domains of the NPC-SS

The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. A Likert-like scale is used to assign to each domain score of 0 to 5 (better to worse) with higher scores indicating more severe clinical impairment.

Parts A/B: Change From Baseline to Week 52 in the Total NPC-SS (With Hearing Domain and ABR Modifier Included)

The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. A Likert-like scale is used to assign to each domain score of 0 to 5 (better to worse). The total score was the sum of individual components scores which ranges from 0 (best) to 45 (worst), with higher scores indicating more severe clinical impairment.

Parts A/B: Time to One Point Increase (Worsening) in NPC-SS Composite Score

The NPC-SS composite score is the sum of the ambulation, cognition, fine motor, and swallowing domains of the NPC-SS. Each of the four NPC-SS components (ambulation, cognition, fine motor, and swallowing) are rated on a scale from 0 (better) to 5 (worse). The total score was the sum of individual components scores which ranges from 0 (best) to 20 (worst), with higher scores indicating more severe clinical impairment. The product-limit survival analysis method is used to estimate the time to one point increase (worsening) in NPC-SS composite score. Time to worsening in NPC-SS Composite Score defined as the interval from study drug administration to a one point increase in the NPC-SS composite score. If a subject discontinued from the study prior to Week 52, then the subject was censored at time of discontinuation. If a subject completed the Week 52 visit, then the subject was censored at the time of last study visit.

Parts A/B: Change From Baseline in the Timed Up and Go (TUG) Test at Week 52

The TUG is a test of balance and risk for falls. This test measures the time taken by a participant to walk 3 meters starting from a sitting position and it ends when the participant is seated again.

Parts A/B: Change From Baseline in the 9-Hole Peg Test at Week 52

The 9-Hole Peg Test is a brief, standardized, quantitative test of upper extremity function. The participant picks up 9 pegs puts them in a block containing nine empty holes, and, once they are in the holes, removes them again as quickly as possible one at a time. The total time to complete the task is recorded.

Parts A/B: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

A TEAE is defined as an adverse event (AE) with onset on or after start of study Drug. An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.

Parts A/B: Change From Baseline to Week 52 in Mean Annualized Rate of Change (Slope) of NPC-SS Composite Score

Full Information

NCT ID

NCT02534844

First Posted

August 18, 2015

Last Updated

February 20, 2023

Sponsor

Mandos LLC

1. Study Identification

Unique Protocol Identification Number

NCT02534844

Brief Title

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

Official Title

A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled 3-Part Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease

Study Type

Interventional

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Completed

Study Start Date

October 2015 (Actual)

Primary Completion Date

March 28, 2018 (Actual)

Study Completion Date

March 28, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Mandos LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844. This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords). In Parts A/B, two out of every three patients will receive the study drug. The third patient will receive 1 to 2 small needle pricks at the location where the LP and IT injection is normally made (sham control). In Part C, all participants will receive study drug, as described in the Part C registration record. Start date for this record is the first day a participant was enrolled in Parts A/B. The trial is actually continuing until the last primary outcome measure of safety data are collected from Part C participants. The last primary outcome measure of safety, along with final adverse events results will be posted in the separate Part C registration record.

Detailed Description

Non-clinical studies and a Phase 1 clinical trial suggest that intrathecal administration of VTS-270 in patients with neurologic manifestations of Niemann-Pick Type C1 (NPC1) disease has the potential to slow the rate of progression of their neurologic disease. Niemann-Pick Type C1 (NPC1) disease is a rare, neurodegenerative, inherited, autosomal recessive lysosomal lipid storage disorder primarily in children and teenagers. The disease is characterized by the inability to properly metabolize cholesterol and other lipids within the cell due to mutations in the NPC1 gene, causing unesterified cholesterol to accumulate in the brain, liver and spleen. This study plans to enroll about 51 participants with NPC1 disease. It will be conducted in three parts: Parts A, B, and C. Part A will evaluate 3 different dose levels of VTS-270 in 12 participants to determine the dose level for Parts B and C. In Part B, 39 more participants will join the original 12 to evaluate the safety and effectiveness of the dose selected from Part A compared to sham control. Part C will be an open-label extension phase of the study for Part B participants who either complete Part B or have met rescue therapy criteria, as well as participants entering Part C from other trials. Participants in Part C will receive treatment with VTS-270 until the product is licensed or the program is terminated (anticipated within 5 years). Final results will be posted in the Part C registration record (NCT04958642).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Niemann-Pick Disease, Type C

Keywords

Niemann-Pick Type C1 (NPC1) Disease, neurologic disease, gross motor dysfunction, fine motor dysfunction, dysphagia, swallowing problems, cognitive dysfunction, gait abnormalities, pediatrics

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Parallel Assignment

Model Description

In Parts A/B (see other registration for Part C description)

Masking

Care ProviderInvestigator

Masking Description

While it is a double-blind trial, the participant and outcomes assessor will be blinded, as well as the Care Provider and Investigator.

Allocation

Randomized

Enrollment

56 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Parts A/B: Sham Control

Arm Type

Experimental

Arm Description

Participants receive no study drug

Arm Title

Parts A/B: Adrabetadex

Arm Type

Other

Arm Description

Participants receive adrabetadex

Intervention Type

Drug

Intervention Name(s)

Parts A/B: Adrabetadex

Other Intervention Name(s)

2-hydroxypropyl-β-cyclodextrin, Cyclodextrin, VTS-270, Adrabetadex

Intervention Description

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

Intervention Type

Other

Intervention Name(s)

Parts A/B: Sham Control

Other Intervention Name(s)

Procedure Control, Skin prick

Intervention Description

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

Primary Outcome Measure Information:

Title

Parts A/B: Change From Baseline to Week 52 in 4-Item Composite Score of Niemann Pick Type C Severity Scale (NPC-SS) Score

Description

Time Frame

Baseline, Week 52

Title

Parts A/B: Number of Participants Classified With Each Score on the Clinician Global Impression of Change (CGIC) at Week 52

Description

Time Frame

Week 52

Secondary Outcome Measure Information:

Title

Parts A/B: Change From Baseline to Week 52 in NPC-SS Total Score (Excluding Hearing and Auditory Brainstem Response [ABR])

Description

Time Frame

Baseline, Week 52

Title

Parts A/ B: Number of Participants Classified as CGIC Responders at Week 52

Description

Time Frame

Week 52

Title

Parts A/B: Number of Participants Classified as Responders on NPC-SS Total Score (Excluding Hearing and ABR) at Week 52

Description

The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. The hearing domain and ABR modifiers are removed from the total NPC-SS total score for this measure. A Likert-like scale is used to assign the remaining 8 major domain scores of 0 to 5 (better to worse). The total score was the sum of individual components scores which ranges from 0 (best) to 40 (worst), with higher scores indicating more severe clinical impairment. Responders on NPC-SS Total Score are defined as participants with no change or improvement on NPC-SS total score from baseline to Week 52.

Time Frame

Week 52

Title

Parts A/B: EQ-5D-3L Questionnaire Visual Analog Scale (VAS) Score (for Health Status) at Baseline and at Week 52

Description

Time Frame

Baseline, Week 52

Title

Parts A/B: Number of Participants Treated for at Least 6 Months Who Qualified for the Rescue Option

Description

Time Frame

Baseline up to Week 26

Title

Parts A/B: Change From Baseline to Week 52 in Each of the 9 Clinical Domains of the NPC-SS

Description

The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. A Likert-like scale is used to assign to each domain score of 0 to 5 (better to worse) with higher scores indicating more severe clinical impairment.

Time Frame

Baseline, Week 52

Title

Parts A/B: Change From Baseline to Week 52 in the Total NPC-SS (With Hearing Domain and ABR Modifier Included)

Description

The NPC-SS total score is based on 9 domains including ambulation, fine motor skills, cognition, swallowing, memory, speech, eye movement, hearing (sensorineural) and seizures. A Likert-like scale is used to assign to each domain score of 0 to 5 (better to worse). The total score was the sum of individual components scores which ranges from 0 (best) to 45 (worst), with higher scores indicating more severe clinical impairment.

Time Frame

Baseline, Week 52

Title

Parts A/B: Time to One Point Increase (Worsening) in NPC-SS Composite Score

Description

Time Frame

Baseline up to Week 52

Title

Parts A/B: Change From Baseline in the Timed Up and Go (TUG) Test at Week 52

Description

The TUG is a test of balance and risk for falls. This test measures the time taken by a participant to walk 3 meters starting from a sitting position and it ends when the participant is seated again.

Time Frame

Baseline, Week 52

Title

Parts A/B: Change From Baseline in the 9-Hole Peg Test at Week 52

Description

Time Frame

Baseline, Week 52

Title

Parts A/B: Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Description

Time Frame

Baseline up to Week 52

Title

Parts A/B: Change From Baseline to Week 52 in Mean Annualized Rate of Change (Slope) of NPC-SS Composite Score

Description

Time Frame

Baseline, Week 52

10. Eligibility

Sex

All

Minimum Age & Unit of Time

4 Years

Maximum Age & Unit of Time

21 Years

Accepts Healthy Volunteers

Eligibility Criteria

Key Inclusion Criteria: Parts A/B: Had onset of neurological symptoms prior to 15 years of age Has confirmed diagnosis of NPC1 determined by either: two NPC1 mutations positive filipin staining and at least one NPC1 mutation vertical supranuclear gaze palsy (VSNGP) in combination with either: one NPC1 mutation, OR positive filipin staining or oxysterol levels consistent with NPC disease and no Niemann-Pick Type C2 (NPC2) Disease mutations Adult participant or parent/guardian has provided written informed consent, with assent collected from minors of appropriate age Is able to undergo a lumbar puncture (LP) and IT drug administration under conscious sedation or general anesthesia Has an NPC Clinical Severity Scale Score of 1 through 4, inclusive, in two or more of the following components: ambulation, fine motor skills, or swallowing; and has a score of 0 through 4 on the cognition component Has a total NPC Clinical Severity Scale Score of 10 or greater If taking miglustat, must have been on a stable dose for past 6-8 weeks and be willing to remain on a stable dose If participant has seizures, they have been adequately controlled for 3 months without changing dose or regimen Has agreed to discontinue all non-prescription supplements at least 1 month prior to first dose (Study Day 0) Has agreed to discontinue any other investigational treatments for NPC including vorinostat or arimoclomol at least 3 months prior to first dose (Study Day 0) If of child-bearing potential (not surgically sterile), agrees to use a medically acceptable method continuously, until at least 30 days after participation in the study Key Exclusion Criteria: Has exclusion criteria as assessed by NPC Clinical Severity Scale: Unable to walk, wheelchair dependent (ambulation NPC score=5) Has need for a nasogastric tube to overcome swallowing difficulties (swallowing NPC score=5) unless used for supplemental feeding or administering medication severe dysmetria (fine motor score =5) or minimal cognitive function (cognition NPC score=5) Weighs less than 15 kg Has had prior treatment with intravenous 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) for NPC1 disease, unless the subject has undergone a minimum 3-month washout period prior to Study Day 0, or has had any prior intrathecal (IT) administration of HP-β-CD Is taking antipsychotics for treatment of psychosis; use of antipsychotic medication for treatment of other disorders (e.g., Attention Deficit Hyperactivity Disorder) will not exclude participation in this trial Has a history of hypersensitivity reactions to any product containing HP-β-CD Has a spinal deformity that could impact the ability to perform a lumbar puncture Has had a skin infection in the lumbar region within 2 months of study entry Has neutropenia, defined as an absolute neutrophil count (ANC) of less than 1.5 X 10^9/L Has thrombocytopenia (platelet count of less than 75 X 10^9/L) Has activated partial thromboplastin time (aPTT) or prothrombin time (PT) prolonged by > 1.5 times the upper limit of normal (ULN) or known history of a bleeding disorder Has had status epilepticus occurring within 3 months of screening and/or seizure frequency that cannot be quantified Has evidence of either obstructive hydrocephalus or normal pressure hydrocephalus Has recently used anticoagulants [in past 2 weeks prior to first dose (Study Day 0); re: lumbar puncture safety]. Is unable to comply with the study procedures or has a clinical disease or laboratory abnormality that in the opinion of the investigator would potentially increase the risk of participation

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Study Lead

Organizational Affiliation

Mandos LLC

Official's Role

Study Director

Facility Information:

Facility Name

University of Alabama at Birmingham

City

Birmingham

State/Province

Alabama

ZIP/Postal Code

35223

Country

United States

Facility Name

Children's Hospital of Orange County

City

Orange

State/Province

California

ZIP/Postal Code

92867

Country

United States

Facility Name

University of California San Francisco

City

San Francisco

State/Province

California

ZIP/Postal Code

94143-0780

Country

United States

Facility Name

University of Colorado Denver

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

Shands Children's Hospital

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32608

Country

United States

Facility Name

Rush University Medical Center

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60612

Country

United States

Facility Name

Eunice Kennedy Shriver National Institute of Child Health and Human Development

City

Bethesda

State/Province

Maryland

ZIP/Postal Code

20892-2425

Country

United States

Facility Name

Boston Children's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Montefiore Medical Center

City

Bronx

State/Province

New York

ZIP/Postal Code

10467

Country

United States

Facility Name

University of North Carolina

City

Chapel Hill

State/Province

North Carolina

ZIP/Postal Code

27514

Country

United States

Facility Name

Lehigh Valley Health Network

City

Allentown

State/Province

Pennsylvania

ZIP/Postal Code

18101

Country

United States

Facility Name

The Children's Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Dell Children's Medical Center of Central Texas

City

Austin

State/Province

Texas

ZIP/Postal Code

78723

Country

United States

Facility Name

Multicare Institute for Research and Innovation

City

Tacoma

State/Province

Washington

ZIP/Postal Code

98405

Country

United States

Facility Name

The Prince of Wales Hospital

City

Sydney

State/Province

New South Wales

ZIP/Postal Code

2031

Country

Australia

Facility Name

Monash Medical Centre

City

Clayton

State/Province

Victoria

ZIP/Postal Code

3168

Country

Australia

Facility Name

Royal Melbourne Hospital

City

Parkville

State/Province

Victoria

ZIP/Postal Code

3050

Country

Australia

Facility Name

Royal Perth Hospital

City

Perth

State/Province

Western Australia

ZIP/Postal Code

6000

Country

Australia

Facility Name

CHU Paris Est - Hospital d'Enfants Armand-Trousseau

City

Paris

State/Province

Cedex 12

ZIP/Postal Code

75 571

Country

France

Facility Name

Katholisches Klinikim Bochum gGmbH

City

Bochum

ZIP/Postal Code

44791

Country

Germany

Facility Name

Universitaetsklinikum Mainz

City

Mainz

ZIP/Postal Code

55131

Country

Germany

Facility Name

Universitaetsklinikum Muenster

City

Münster

ZIP/Postal Code

48149

Country

Germany

Facility Name

Waikato Hospital

City

Hamilton West

ZIP/Postal Code

3204

Country

New Zealand

Facility Name

National University Hospital (Singapore) Pte, Ltd

City

Singapore

ZIP/Postal Code

119074

Country

Singapore

Facility Name

Hospital Universitario del Valle Hebron

City

Barcelona

ZIP/Postal Code

08035

Country

Spain

Facility Name

Hacettepe University Medical Faculty

City

Altındağ

State/Province

Ankara

ZIP/Postal Code

06230

Country

Turkey

Facility Name

Gazi University Medical Faculty

City

Çankaya

State/Province

Ankara

ZIP/Postal Code

06570

Country

Turkey

Facility Name

Birmingham Women's and Children's NHS Trust

City

Birmingham

State/Province

West Midlands

ZIP/Postal Code

B4 6NH

Country

United Kingdom

Facility Name

Great Ormond Street Hospital

City

London

ZIP/Postal Code

WC1N 3JH

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing Plan Description

Summary aggregate (basic) results (including adverse events information) and the study protocol are made available on clinicaltrials.gov (NCT02534844) when required by regulation. Individual de-identified patient data will not be disclosed. Requests for additional information should be directed to the company at medinfo@mnk.com.

Learn more about this trial

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

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