Niemann-Pick Type C DiseasePhase 3NCT03643562
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
A Phase 3 study testing Adrabetadex in people with Niemann-Pick Type C Disease, run by Mandos LLC at 17 sites in United States.
Trial summary
This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks. Participants who were already taking adrabetadex will receive their stable dose. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Niemann-Pick Type C Disease
- 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
- 27 participants
- Where
- 17 sitesUnited States
- Study dates
- 18 Jun 2018to 2 Nov 2021
- Phase
- Phase 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- To be included in the study, a participant must meet the following criteria:
- Is male or female and at least 4 years of age at time of screening.
- Has a confirmed diagnosis of NPC and exhibits neurologic symptoms.
- Has written informed consent/assent to participate.
- Has the ability to undergo LP and IT drug administration.
- If taking miglustat (Zavesca®), must have been on a stable dose for the past 6 weeks and be willing to remain on a stable dose for the duration of participation in the study, or discontinue miglustat use at least 6 weeks before entry into the study (Day 1).
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- If has a history of seizures, the condition is adequately controlled as per protocol requirements.
- Agrees to discontinue any investigational treatments (other than adrabetadex) for at least 1 month before first dose on Day 1.
- If engaging in heterosexual sex, agrees to use a protocol-defined method of contraception throughout the study, and until 30 days after completing the study.
- Has a responsible adult who the investigator determines is able and willing to comply with study requirements and a parent/guardian who will accompany the participant to study visits.
Who cannot join
- Participants will be excluded from the study if they meet any of the following criteria:
- Weighs less than 15 kg.
- Has a history of hypersensitivity reactions to any product containing 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) or has a history of hypersensitivity reactions or allergy to anesthesia/sedation.
- Has received treatment with any investigational product (other than adrabetadex) within 1 month prior to Day 1 of treatment.
- Is pregnant or nursing.
- Has systemic infection or uncontrolled psychosis.
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- Has known history of a bleeding disorder.
- Has used anticoagulants within 2 months of entry into the study.
- Per protocol, or in the opinion of the investigator:
- has laboratory values that would preclude participation
- has suspected infection of the central nervous system (CNS)
- has a spinal deformity that could impact performance of repeated LPs
- has a serious skin infection in the lumbar region or evidence of obstructive or normal pressure hydrocephalus
- is unable to comply with the study requirements
- has a medical condition that might increase the risk of participation
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
United States17 sites
Rare Disease Research, LLC
Atlanta, Georgia, 30318
Children's Hospital Colorado
Aurora, Colorado, 80045
Dell Children's Medical Center of Central Texas
Austin, Texas, 78723
Rush University Medical Center
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Children's Specialty Center of Nevada
Las Vegas, Nevada, 89109
University of Arkansas System
Little Rock, Arkansas, 72205
Loma Linda University Health System
Loma Linda, California, 92354
Children's Hospital Los Angeles
Los Angeles, California, 90027
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
NYU Langone Medical Center
New York, New York, 10017
Children's Hospital of Orange County
Orange, California, 92868
Oregon Health & Science University
Portland, Oregon, 97239
Carilion Medical Center, Carilion Clinic
Roanoke, Virginia, 24014
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
Official title: Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks.
Participants who were already taking adrabetadex will receive their stable dose. Participants who have not ever taken it will start by receiving 400 mg.
Participants will receive treatment every 2 weeks until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason. Participants will not receive additional study treatment after their participation in this protocol.
- Conditions
- Niemann-Pick Type C Disease
- Being tested
- Adrabetadex
- Keywords
- Niemann-Pick, NPC, NPC Type 1 (NPC1)
- Medical subject headings
- Niemann-Pick Disease, Type C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AdrabetadexDrug
Also called VTS-270
Administered via lumbar puncture (LP) and IT infusion
| Study group | Role | What they receive |
|---|---|---|
| Adrabetadex | Experimental | Participants will receive prescribed adrabetadex by intra-thecal (IT) injection every 2 weeks. 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 adverse event (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 Week 134
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2018
- Study start18 Jun 2018
- Primary completion2 Nov 2021
- Study completion2 Nov 2021
Why it stopped: Sponsor had determined that the benefit-risk profile of adrabetadex was not favorable and the clinical development program for adrabetadex was discontinued.
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 Type C DiseaseStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Continued treatment for children at least 4 years of age
- Masking
- None (open label)
- Enrolment
- 27 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
- NCT03643562
- Sponsor's protocol ID
- VTS-270-001
- Status last verified
- Dec 2023
- First submitted
- 19 Aug 2018
- Last updated
- 17 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- 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 17 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
- ClinicalTrials.gov ID
- NCT03643562
- Acronym / sponsor ID
- VTS-270-001
- Conditions
- Niemann-Pick Type C Disease
- Keywords
- Niemann-Pick, NPC, NPC Type 1 (NPC1)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor had determined that the benefit-risk profile of adrabetadex was not favorable and the clinical development program for adrabetadex was discontinued.
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Mandos LLCIndustry (pharma, biotech, devices)
- Study start
- 18 Jun 2018 (actual)
- Primary completion
- 2 Nov 2021 (actual)
- Study completion
- 2 Nov 2021 (actual)
- First posted
- 23 Aug 2018 (actual)
- Results
- Posted 17 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks.
Participants who were already taking adrabetadex will receive their stable dose. Participants who have not ever taken it will start by receiving 400 mg.
Participants will receive treatment every 2 weeks until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason. Participants will not receive additional study treatment after their participation in this protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Continued treatment for children at least 4 years of age
| Arm | Type | Receives |
|---|---|---|
| Adrabetadex | Experimental | Drug: AdrabetadexParticipants will receive prescribed adrabetadex by intra-thecal (IT) injection every 2 weeks. |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Is male or female and at least 4 years of age at time of screening.
- Has a confirmed diagnosis of NPC and exhibits neurologic symptoms.
- Has written informed consent/assent to participate.
- Has the ability to undergo LP and IT drug administration.
- If taking miglustat (Zavesca®), must have been on a stable dose for the past 6 weeks and be willing to remain on a stable dose for the duration of participation in the study, or discontinue miglustat use at least 6 weeks before entry into the study (Day 1).
Key exclusion criteria (15 listed)
- Weighs less than 15 kg.
- Has a history of hypersensitivity reactions to any product containing 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) or has a history of hypersensitivity reactions or allergy to anesthesia/sedation.
- Has received treatment with any investigational product (other than adrabetadex) within 1 month prior to Day 1 of treatment.
- Is pregnant or nursing.
- Has systemic infection or uncontrolled psychosis.
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-270Administered via lumbar puncture (LP) and IT infusion | Drug | Adrabetadex |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to Week 134
An adverse event (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 17 Jan 2024.
- 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
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Adrabetadex in people with Niemann-Pick Type C Disease, run by Mandos LLC at 17 sites in United States.
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 10 inclusion and 15 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 17 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States. 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 phase mean?
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 17 Jan 2024). 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 Week 134). 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 17 Jan 2024.