Huntington DiseasePhase 1NCT03787758
A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B
A Phase 1 study testing SAGE-718 in people with Huntington Disease, run by Supernus Pharmaceuticals, Inc. at 2 sites in United States.
Trial summary
This study is a phase 1, double-blind, placebo-controlled, multiple ascending dose study to determine the safety, tolerability, and pharmacokinetics of SAGE-718 oral solution in healthy adults (Part A) with an open-label cohort of patients with Huntington's disease (Part B) Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Huntington Disease
- What is being tested?
- SAGE-718
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 2 sitesUnited States
- Study dates
- 28 Feb 2019to 7 Oct 2019
- Phase
- Phase 1
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject is positive for mutant HTT (documented CAG repeats ≥ 40 units).
- Subject has a body weight ≥50 kg and body mass index ≥18.0 and ≤30.0 kg/m2 at screening.
Who cannot join
- Subject has any clinically significant abnormal finding on the physical exam at screening or admission.
- Subject has a history or presence of a neurologic disease or condition (other than Huntington's disease), including but not limited to severe chorea, epilepsy, closed head trauma with clinically significant sequelae, or a prior seizure.
- Subject has a family history of epilepsy.
- Subject has a positive screening test for alcohol or drugs of abuse (including marijuana) at screening or admission.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Sage Investigational Site
Long Beach, California, 90806
Sage Investigational Site
Marlton, New Jersey, 08053
About this study
Official title: A Phase 1, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Determine the Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Healthy Adults With an Open-label Cohort of Patients With Huntington's Disease
This study is a phase 1, double-blind, placebo-controlled, multiple ascending dose study to determine the safety, tolerability, and pharmacokinetics of SAGE-718 oral solution in healthy adults (Part A) with an open-label cohort of patients with Huntington's disease (Part B)
Read the detailed description
This posting addresses Part B
- Conditions
- Huntington Disease
- Being tested
- SAGE-718
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAGE-718Drug
SAGE-718
| Study group | Role | What they receive |
|---|---|---|
| SAGE-718 | Experimental | Not described Receives: Drug: SAGE-718 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.
Measured: 21 Days
Percentage of participants with change from baseline in vital signs.
Measured: 21 Days
Change from baseline in electrocardiograms (ECGs) including PR interval, QT interval, QTc interval, QTcF, and rhythm abnormalities
Measured: 21 Days
Percentage of participants with change from baseline in clinical laboratory parameters.
Measured: 21 Days
Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS).
Measured: 21 Days
Secondary outcomes (3)
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by area under the curve [AUC].
Measured: 17 Days
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by maximum observed concentration [Cmax].
Measured: 17 Days
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by time of occurrence of Cmax [tmax].
Measured: 17 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Dec 2018
- Study start28 Feb 2019
- Primary completion7 Oct 2019
- Study completion7 Oct 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Huntington 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Supernus Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03787758
- Sponsor's protocol ID
- 718-CLP-102 B
- Status last verified
- Jan 2022
- First submitted
- 17 Dec 2018
- Last updated
- 17 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Data sharing will be consistent with the results submission policy of ClinicalTrials.gov.
Publications
Koenig A, Lewis M, Wald J, Li S, Varoglu M, Dai J, Sankoh A, Paumier K, Doherty J, Quirk M. Dalzanemdor (SAGE-718), a novel, investigational N-methyl-D-aspartate receptor positive allosteric modulator: Safety, tolerability, and clinical pharmacology in randomized dose-finding studies in healthy participants and an open-label study in participants with Huntington's disease. Clin Transl Sci. 2024 Jul;17(7):e13852. doi: 10.1111/cts.13852.
PubMed 38988035
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Determine the Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Healthy Adults With an Open-label Cohort of Patients With Huntington's Disease
- ClinicalTrials.gov ID
- NCT03787758
- Acronym / sponsor ID
- 718-CLP-102 B
- Conditions
- Huntington Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Supernus Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Feb 2019 (actual)
- Primary completion
- 7 Oct 2019 (actual)
- Study completion
- 7 Oct 2019 (actual)
- First posted
- 26 Dec 2018 (actual)
- 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.
This study is a phase 1, double-blind, placebo-controlled, multiple ascending dose study to determine the safety, tolerability, and pharmacokinetics of SAGE-718 oral solution in healthy adults (Part A) with an open-label cohort of patients with Huntington's disease (Part B)
Detailed description
This posting addresses Part B
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SAGE-718 | Experimental | Drug: SAGE-718 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Subject is positive for mutant HTT (documented CAG repeats ≥ 40 units).
- Subject has a body weight ≥50 kg and body mass index ≥18.0 and ≤30.0 kg/m2 at screening.
Key exclusion criteria (4 listed)
- Subject has any clinically significant abnormal finding on the physical exam at screening or admission.
- Subject has a history or presence of a neurologic disease or condition (other than Huntington's disease), including but not limited to severe chorea, epilepsy, closed head trauma with clinically significant sequelae, or a prior seizure.
- Subject has a family history of epilepsy.
- Subject has a positive screening test for alcohol or drugs of abuse (including marijuana) at screening or admission.
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 |
|---|---|---|
| SAGE-718SAGE-718 | Drug | SAGE-718 |
Primary outcome
Number of Participants with the Incidence of Adverse Events and Serious Adverse Events.
Time frame: 21 Days
Percentage of participants with change from baseline in vital signs.
Time frame: 21 Days
Change from baseline in electrocardiograms (ECGs) including PR interval, QT interval, QTc interval, QTcF, and rhythm abnormalities
Time frame: 21 Days
Percentage of participants with change from baseline in clinical laboratory parameters.
Time frame: 21 Days
Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS).
Time frame: 21 Days
3 secondary outcomes
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by area under the curve [AUC].
Time frame: 17 Days
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by maximum observed concentration [Cmax].
Time frame: 17 Days
PK profile of SAGE-718 following administration of multiple doses of SAGE-718 Oral Solution as assessed by time of occurrence of Cmax [tmax].
Time frame: 17 Days
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 17 Sep 2025.
- Derived from this study: PubMed 38988035Koenig A, Lewis M, Wald J, Li S, Varoglu M, Dai J, Sankoh A, Paumier K, Doherty J, Quirk M. Dalzanemdor (SAGE-718), a novel, investigational N-methyl-D-aspartate receptor positive allosteric modulator: Safety, tolerability, and clinical pharmacology in randomized dose-finding studies in healthy participants and an open-label study in participants with Huntington's disease. Clin Transl Sci. 2024 Jul;17(7):e13852. doi: 10.1111/cts.13852.
- Individual participant dataSharing planned: No. Data sharing will be consistent with the results submission policy of ClinicalTrials.gov.
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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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SAGE-718 in people with Huntington Disease, run by Supernus Pharmaceuticals, Inc. at 2 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 2 inclusion and 4 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 17 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Number of Participants with the Incidence of Adverse Events and Serious Adverse Events. (time frame: 21 Days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03787758, which also lists 1 linked publication.
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 Sep 2025.