Niemann-Pick Disease, Type CPhase 2/3NCT04958642
Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
A Phase 2/3 study testing Adrabetadex in people with Niemann-Pick Disease, Type C, run by Mandos LLC at 30 sites in 9 countries.
Trial summary
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. Read more about this study
- Who is it for?
- Ages 4 to 21 years
- Conditions the study is for
- Niemann-Pick Disease, Type C
- What is being tested?
- Adrabetadex
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 66 participants
- Where
- 30 sitesin 9 countries
- Study dates
- 23 Dec 2015to 11 Apr 2022
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- One of the following is required for inclusion into VTS301 Part C:
- 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
Who cannot join
- None of the inclusion criteria are applicable
Trial locations and contacts
30 sites in 9 countries.
30 sites in 9 countries
United States15 sites
Lehigh Valley Health Network
Allentown, Pennsylvania, 18101
University of Colorado Denver
Aurora, Colorado, 80045
Dell Children's Medical Center of Central Texas
Austin, Texas, 78723
Eunice Kennedy Shriver National Institute of Child Health and Human Development
Bethesda, Maryland, 20892-2425
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02115
University of North Carolina
Chapel Hill, North Carolina, 27514
Rush University Medical Center
Chicago, Illinois, 60612
Shands Children's Hospital
Gainesville, Florida, 32608
Children's Hospital of Orange County: CHOC Children's
Orange, California, 92867
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of California San Francisco
San Francisco, California, 94143-0780
Multicare Institute for Research and Innovation
Tacoma, Washington, 98405
Montefiore Medical Center
The Bronx, New York, 10467
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Australia4 sites
Monash Medical Centre
Clayton, Victoria, 3168
Royal Melbourne Hospital
Melbourne, Victria
Royal Perth Hospital
Perth, Western Australia, 6000
The Prince of Wales Hospital
Randwick, New South Wales, 2031
Germany3 sites
Katholisches Klinikim Bochum gGmbH
Bochum, 44791
Universitaetsklinikum Mainz
Mainz, 55131
Universitaetsklinikum Muenster
Münster, 48149
Turkey (Türkiye)2 sites
Gazi University Medical Faculty
Ankara
Hacettepe University Medical Faculty
Ankara
United Kingdom2 sites
Birmingham Children's Hospital NHS Foundation Trust
Birmingham, B4 6NH
Great Ormond Street Hospital
London, WC1N 3JH
France1 site
CHU Paris Est - Hopital d'Enfants Armand-Trousseau
Paris, Cedex 12, 75 571
Singapore1 site
National University Hospital (Singapore) Pte, Ltd
Singapore, 119074
About this study
Official title: A Phase 2B/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here.
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.
Read the detailed description
Participants in Part C will receive adrabetadex until the investigator considers adrabetadex to no longer be beneficial to the participant, or the development program is discontinued.
- Conditions
- Niemann-Pick Disease, Type C
- Being tested
- Adrabetadex
- Keywords
- Niemann-Pick Type C1 (NPC1) Disease, neurologic disease, gross motor dysfunction, fine motor dysfunction, dysphagia, swallowing problems, cognitive dysfunction, gait abnormalities, pediatrics
- Medical subject headings
- Niemann-Pick Disease, Type C, Disease, Deglutition Disorders, Cognitive Dysfunction, Mobility Limitation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AdrabetadexDrug
Also called VTS-270, 2-hydroxypropyl-β-cyclodextrin, Cyclodextrin
Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.
| Study group | Role | What they receive |
|---|---|---|
| Adrabetadex | Experimental | 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. Receives: Drug: Adrabetadex |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Measured: Baseline up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2021
- Study start23 Dec 2015
- Primary completion11 Apr 2022
- Study completion11 Apr 2022
Why it stopped: Terminated by previous Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Niemann-Pick Disease, Type CStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- In Part C, all participants receive adrabetadex
- Masking
- None (open label)
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mandos LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Study Lead, Study director, Mandos LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04958642
- Sponsor's protocol ID
- VTS301 (Part C)
- Other IDs
- 2015-002548-15, 2015
- Status last verified
- Aug 2023
- First submitted
- 29 Jun 2021
- Last updated
- 29 Aug 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2B/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
- ClinicalTrials.gov ID
- NCT04958642
- Acronym / sponsor ID
- VTS301 (Part C)
- Conditions
- 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
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated by previous Sponsor decision
- Enrolment
- 66 participants (actual)
- Lead sponsor
- Mandos LLCIndustry (pharma, biotech, devices)
- Study start
- 23 Dec 2015 (actual)
- Primary completion
- 11 Apr 2022 (actual)
- Study completion
- 11 Apr 2022 (actual)
- First posted
- 12 Jul 2021 (actual)
- Results
- Posted 29 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here.
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.
Detailed description
Participants in Part C will receive adrabetadex until the investigator considers adrabetadex to no longer be beneficial to the participant, or the development program is discontinued.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- In Part C, all participants receive adrabetadex
| Arm | Type | Receives |
|---|---|---|
| Adrabetadex | Experimental | Drug: AdrabetadexAll 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. |
Who may qualify
- Ages
- 4 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (1 listed)
- None of the inclusion criteria are applicable
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AdrabetadexAlso: VTS-270, 2-hydroxypropyl-β-cyclodextrin, CyclodextrinMallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion. | Drug | Adrabetadex |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to 5 years
An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Study Lead study director
Mandos LLC
30 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Adrabetadex in people with Niemann-Pick Disease, Type C, run by Mandos LLC at 30 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 29 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 30 sites in United States, Australia, Germany, Turkey (Türkiye), United Kingdom and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Aug 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: Baseline up to 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Aug 2023; AzTrials last refreshed its copy on 8 Nov 2026.