Smith-Magenis SyndromePhase 2/3NCT02231008
Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMS
A Phase 2/3 study testing tasimelteon in people with Smith-Magenis Syndrome and Circadian, run by Vanda Pharmaceuticals at 4 sites in United States.
Trial summary
The aim of this study is to investigate tasimelteon vs. placebo on sleep disturbances of individuals with Smith-Magenis Syndrome. Read more about this study
- Who is it for?
- Ages 3 to 65 years
- Conditions the study is for
- Smith-Magenis Syndrome, Circadian
- What is being tested?
- tasimelteon
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 49 participants
- Where
- 4 sitesUnited States
- Study dates
- Sep 2015to Jan 2022
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
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 3 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- A confirmed clinical diagnosis of SMS
- Informed consent from the patient or the legal guardian
- Male or female between the ages of 3- 65 years of age
- Recent history of sleep disturbances
- Have an appointed care-giver complete the required outpatient assessments
- Willing and able to comply with study requirements and restrictions
Who cannot join
- Unable to dose daily with medication
- Exposure to any investigational drug, including placebo, within 30 days or 5 half-lives (whichever was longer) of screening
- Any other sound medical reason as determined by the clinical investigator
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Study site
Baltimore, Maryland
Study site
Chevy Chase, Maryland
Study site
Houston, Texas
Study site
Santa Monica, California
About this study
Official title: A Double-blind, Randomized, Two-period Crossover Study Evaluating the Effects of Tasimelteon vs. Placebo on Sleep Disturbances of Individuals With Smith-Magenis Syndrome (SMS)
The aim of this study is to investigate tasimelteon vs. placebo on sleep disturbances of individuals with Smith-Magenis Syndrome.
- Conditions
- Smith-Magenis Syndrome, Circadian
- Being tested
- tasimelteon, placebo
- Keywords
- SMS
- Medical subject headings
- Smith-Magenis Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
tasimelteonDrug
placeboDrug
| Study group | Role | What they receive |
|---|---|---|
| Tasimelteon | Experimental | Single dose oral capsule or equivalent age-appropriate oral formulation, daily dosage Receives: Drug: tasimelteon |
| Placebo | Placebo comparator | Placebo comparator Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the efficacy of tasimelteon administered daily compared to placebo, as measured by improvement in sleep parameters
Measured: 9 Weeks
Secondary outcomes (1)
Safety and tolerability as measured by spontaneous reporting of adverse events (AEs).
Measured: up to 137 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2014 (estimated)
- Study startSep 2015
- Primary completion19 Nov 2018
- Study completionJan 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Smith-Magenis SyndromeStudies of the same condition, open and under way.
- More trials run by Vanda PharmaceuticalsThe sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Single group
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanda Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02231008
- Sponsor's protocol ID
- VP-VEC-162-2401
- Status last verified
- Nov 2022
- First submitted
- 29 Aug 2014
- Last updated
- 18 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Randomized, Two-period Crossover Study Evaluating the Effects of Tasimelteon vs. Placebo on Sleep Disturbances of Individuals With Smith-Magenis Syndrome (SMS)
- ClinicalTrials.gov ID
- NCT02231008
- Acronym / sponsor ID
- VP-VEC-162-2401
- Conditions
- Smith-Magenis Syndrome, Circadian
- Keywords
- SMS
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Vanda PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Sep 2015 (actual)
- Primary completion
- 19 Nov 2018 (actual)
- Study completion
- Jan 2022 (actual)
- First posted
- 3 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to investigate tasimelteon vs. placebo on sleep disturbances of individuals with Smith-Magenis Syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Tasimelteon | Experimental | Drug: tasimelteonSingle dose oral capsule or equivalent age-appropriate oral formulation, daily dosage |
| Placebo | Placebo comparator | Drug: placeboPlacebo comparator |
Who may qualify
- Ages
- 3 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- A confirmed clinical diagnosis of SMS
- Informed consent from the patient or the legal guardian
- Male or female between the ages of 3- 65 years of age
- Recent history of sleep disturbances
- Have an appointed care-giver complete the required outpatient assessments
Key exclusion criteria (3 listed)
- Unable to dose daily with medication
- Exposure to any investigational drug, including placebo, within 30 days or 5 half-lives (whichever was longer) of screening
- Any other sound medical reason as determined by the clinical investigator
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 |
|---|---|---|
| tasimelteon | Drug | Tasimelteon |
| placebo | Drug | Placebo |
Primary outcome
To determine the efficacy of tasimelteon administered daily compared to placebo, as measured by improvement in sleep parameters
Time frame: 9 Weeks
1 secondary outcome
Safety and tolerability as measured by spontaneous reporting of adverse events (AEs).
Time frame: up to 137 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2022.
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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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing tasimelteon in people with Smith-Magenis Syndrome and Circadian, run by Vanda Pharmaceuticals at 4 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 6 inclusion and 3 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: finished, as of the sponsor's last update on 18 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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. Some participants in this study may receive a placebo. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To determine the efficacy of tasimelteon administered daily compared to placebo, as measured by improvement in sleep parameters (time frame: 9 Weeks). 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 18 Nov 2022.