Prader-Willi SyndromePhase 1NCT05504395
A Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome
A Phase 1 study testing CSTI-500 in people with Prader-Willi Syndrome, run by ConSynance Therapeutics at 1 site in United States.
Trial summary
The purpose of this Phase 1 study is to evaluate the pharmacokinetics (PK) and safety of a single dose of CSTI-500 10 mg in subjects with Prader-Willi syndrome (PWS) between 13 and 50 years of age with a genetically confirmed diagnosis of PWS. Read more about this study
- Who is it for?
- Ages 13 to 50 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- CSTI-500
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Nov 2022to 21 Feb 2023
- 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 13 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male and female subjects (13-50 years of age at screening) with a documented medical record history of PWS confirmed by genetic testing.
- Subject must have a reliable caregiver/parent to bring the subject to the site for the visits, remain with the subject during visit times when allowed to be with the subject and respond to any questions during the visits.
- Female subjects must not be pregnant or lactating and be willing to use double barrier birth control method throughout the study.
- A normal supine systolic blood pressure must be ≤140 mmHg and ≥100 mmHg; diastolic blood pressure must be ≤80 mmHg and ≥60 mmHg at Screening. Pulse rate must be ≥50 bpm and ≤100 bpm and pulse rate increase on standing must be within acceptable range.
- All concomitant medications including blood pressure medications and type 2 diabetic medications must be stable for ≥3 months prior to screening (≤10% change). Supplements and vitamins are not considered concomitant medications for eligibility purposes.
Who cannot join
- Participation in any clinical study with an investigational drug/device within 3 months prior to screening or during the study.
- Recent use (within 3 months) of weight loss agents including prescription, herbal medications, and weight loss supplements.
- Major surgery within 6 months of screening or planned during the study or history of bariatric surgery.
- Any malignancy in the 2 years prior to screening (excluding basal cell carcinoma or squamous cell carcinoma of the skin or cervical carcinoma in situ that have been successfully treated).
- Current liver, pulmonary, cardiac, or GI disease that would be expected to adversely affect study participation. Stable disease, e.g., asthma or controlled hypertension is not excluded. Liver disease or liver injury as indicated by abnormal liver function tests, ALT, AST, alkaline phosphatase, or serum bilirubin (≥3X ULN for any of these tests).
- Unexplained history or presence of combination of unexplained symptoms e.g., dizziness, syncope, fatigue, palpitations/tachycardia, headaches, or exercise intolerance.
Show all 12Show fewer
- Heart failure classified per the New York Heart Association (NYHA) level II or greater.
- Myocardial infarction, stroke, or confirmed TIA within the last 5 years.
- Uncontrolled Type 2 diabetes as defined by HbA1c ≥ 9% at Screening.
- Insulin-dependent Type 1 diabetes.
- Subjects with a history of any suicidal behavior.
- Inability to swallow the oral capsule whole with water.
Trial locations and contacts
1 site in 1 country.
United States1 site
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
About this study
Official title: A Phase 1, Single Center, Open Label, Single Dose, Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome
The purpose of this Phase 1 study is to evaluate the pharmacokinetics (PK) and safety of a single dose of CSTI-500 10 mg in subjects with Prader-Willi syndrome (PWS) between 13 and 50 years of age with a genetically confirmed diagnosis of PWS.
Read the detailed description
This is an open-label, single center, Phase 1 study to evaluate the PK and safety of a 10 mg single oral dose of CSTI-500, a triple monoamine reuptake inhibitor (TRI), in patients with genetically confirmed PWS. The study will consist of a Screening Period of up to 1-3 days prior to the Baseline Visit (Visit 2). In addition to the Screening Visit (Visit 1), eligible subjects will attend five in-clinic site visits for PK blood draws and safety assessments over a 6-day period. At Visit 2 all subjects will receive one single oral dose of CSTI-500 10 mg. Approximately 14 patients aged 13 to 50 years who meet all eligibility criteria will receive one single dose of CSTI-500.
- Conditions
- Prader-Willi Syndrome
- Being tested
- CSTI-500
- Keywords
- PWS, Phase 1, Single Dose, Non randomized, PK, Safety
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CSTI-500Drug
Also called AMR-001181
Single 10 mg capsule
| Study group | Role | What they receive |
|---|---|---|
| CSTI-500 10mg | Experimental | All eligible subjects will be administered a single oral dose of CSTI-500 10 mg at Visit 2 Receives: Drug: CSTI-500 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Observed Plasma Concentration (Cmax)
Maximum observed plasma concentration following drug administration determined directly from the concentration-time profile.
Measured: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
AUC0-72
Area under the plasma-drug concentration-time curve from pre-dose (time 0) to 72 hours after drug administration
Measured: Pre-dose to 1, 2, 4, 8, 12, 24, 48, and 72 hours post-dose
AUC0-inf
Area under the plasma-drug concentration-time curve from pre-dose (time 0) extrapolated to infinite time
Measured: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
CSTI-500 plasma concentration
Measured: 1 hour post-dose
CSTI-500 plasma concentration
Measured: 2 hour post-dose
CSTI-500 plasma concentration
Measured: 4 hour post-dose
CSTI-500 plasma concentration
Measured: 8 hour post-dose
CSTI-500 plasma concentration
Measured: 12 hour post-dose
CSTI-500 plasma concentration
Measured: 24 hour post-dose
CSTI-500 plasma concentration
Measured: 48 hour post-dose
CSTI-500 plasma concentration
Measured: 72 hour post-dose
CSTI-500 plasma concentration
Measured: 144 hour post-dose
Secondary outcomes (8)
Incidence of treatment-emergent adverse events (TEAEs)
Number of participants with TEAEs, defined as an adverse event (AE) that is new or worsened in severity after the dose of study drug. AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA) and will be summarized by system organ class, preferred term, and treatment.
Measured: From pre-dose to 15 days post-dose
Incidence of clinically significant findings in physical examinations
Measured: Screening to 12, 24, 48, 72, and 144 hours post-dose
Incidence of clinically significant findings in vital signs
Participants will be assessed for any clinically significant changes in vital parameters (blood pressure, heart rate in supine and standing position, respiratory rate, and body temperature). This also includes evaluation of postural orthostatic tachycardic syndrome.
Measured: Screening and pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Incidence of clinically significant findings in laboratory values
Laboratory evaluations include hematology, thyroid function, and chemistry blood and urine laboratory tests.
Measured: Screening to 24, 48, 72, and 144 hours post-dose
Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs)
Measured: Screening and pre-dose to 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Comparison of CSTI-500 Cmax values obtained from venous blood draws with values obtained from finger prick samples in PWS subjects
Measured: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Comparison of CSTI-500 AUC0-72 values obtained from venous blood draws with values obtained from finger prick samples in PWS subjects
Measured: Pre-dose to 1, 2, 4, 8, 12, 24, 48, and 72 hours post-dose
Comparison of CSTI-500 AUC0-inf values obtained from venous blood draws with values obtained from finger prick samples in PWS subjects
Measured: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2022
- Study start14 Nov 2022
- Primary completion21 Feb 2023
- Study completion21 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- ConSynance Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Italo Biaggioni, MD, Principal investigator, Vanderbilt Autonomic Dysfunction Center, Vanderbilt University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05504395
- Sponsor's protocol ID
- CSTI-500-003
- Status last verified
- Aug 2023
- First submitted
- 4 Aug 2022
- Last updated
- 9 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- 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 9 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Single Center, Open Label, Single Dose, Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT05504395
- Acronym / sponsor ID
- CSTI-500-003
- Conditions
- Prader-Willi Syndrome
- Keywords
- PWS, Phase 1, Single Dose, Non randomized, PK, Safety
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- ConSynance TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 14 Nov 2022 (actual)
- Primary completion
- 21 Feb 2023 (actual)
- Study completion
- 21 Feb 2023 (actual)
- First posted
- 17 Aug 2022 (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.
The purpose of this Phase 1 study is to evaluate the pharmacokinetics (PK) and safety of a single dose of CSTI-500 10 mg in subjects with Prader-Willi syndrome (PWS) between 13 and 50 years of age with a genetically confirmed diagnosis of PWS.
Detailed description
This is an open-label, single center, Phase 1 study to evaluate the PK and safety of a 10 mg single oral dose of CSTI-500, a triple monoamine reuptake inhibitor (TRI), in patients with genetically confirmed PWS. The study will consist of a Screening Period of up to 1-3 days prior to the Baseline Visit (Visit 2). In addition to the Screening Visit (Visit 1), eligible subjects will attend five in-clinic site visits for PK blood draws and safety assessments over a 6-day period. At Visit 2 all subjects will receive one single oral dose of CSTI-500 10 mg. Approximately 14 patients aged 13 to 50 years who meet all eligibility criteria will receive one single dose of CSTI-500.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CSTI-500 10mg | Experimental | Drug: CSTI-500All eligible subjects will be administered a single oral dose of CSTI-500 10 mg at Visit 2 |
Who may qualify
- Ages
- 13 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Male and female subjects (13-50 years of age at screening) with a documented medical record history of PWS confirmed by genetic testing.
- Subject must have a reliable caregiver/parent to bring the subject to the site for the visits, remain with the subject during visit times when allowed to be with the subject and respond to any questions during the visits.
- Female subjects must not be pregnant or lactating and be willing to use double barrier birth control method throughout the study.
- A normal supine systolic blood pressure must be ≤140 mmHg and ≥100 mmHg; diastolic blood pressure must be ≤80 mmHg and ≥60 mmHg at Screening. Pulse rate must be ≥50 bpm and ≤100 bpm and pulse rate increase on standing must be within acceptable range.
- All concomitant medications including blood pressure medications and type 2 diabetic medications must be stable for ≥3 months prior to screening (≤10% change). Supplements and vitamins are not considered concomitant medications for eligibility purposes.
Key exclusion criteria (12 listed)
- Participation in any clinical study with an investigational drug/device within 3 months prior to screening or during the study.
- Recent use (within 3 months) of weight loss agents including prescription, herbal medications, and weight loss supplements.
- Major surgery within 6 months of screening or planned during the study or history of bariatric surgery.
- Any malignancy in the 2 years prior to screening (excluding basal cell carcinoma or squamous cell carcinoma of the skin or cervical carcinoma in situ that have been successfully treated).
- Current liver, pulmonary, cardiac, or GI disease that would be expected to adversely affect study participation. Stable disease, e.g., asthma or controlled hypertension is not excluded. Liver disease or liver injury as indicated by abnormal liver function tests, ALT, AST, alkaline phosphatase, or serum bilirubin (≥3X ULN for any of these tests).
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 |
|---|---|---|
| CSTI-500Also: AMR-001181Single 10 mg capsule | Drug | CSTI-500 10mg |
Primary outcome
Maximum Observed Plasma Concentration (Cmax)
Time frame: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Maximum observed plasma concentration following drug administration determined directly from the concentration-time profile.
AUC0-72
Time frame: Pre-dose to 1, 2, 4, 8, 12, 24, 48, and 72 hours post-dose
Area under the plasma-drug concentration-time curve from pre-dose (time 0) to 72 hours after drug administration
AUC0-inf
Time frame: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Area under the plasma-drug concentration-time curve from pre-dose (time 0) extrapolated to infinite time
CSTI-500 plasma concentration
Time frame: 1 hour post-dose
CSTI-500 plasma concentration
Time frame: 2 hour post-dose
CSTI-500 plasma concentration
Time frame: 4 hour post-dose
CSTI-500 plasma concentration
Time frame: 8 hour post-dose
CSTI-500 plasma concentration
Time frame: 12 hour post-dose
CSTI-500 plasma concentration
Time frame: 24 hour post-dose
CSTI-500 plasma concentration
Time frame: 48 hour post-dose
CSTI-500 plasma concentration
Time frame: 72 hour post-dose
CSTI-500 plasma concentration
Time frame: 144 hour post-dose
8 secondary outcomes
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: From pre-dose to 15 days post-dose
Incidence of clinically significant findings in physical examinations
Time frame: Screening to 12, 24, 48, 72, and 144 hours post-dose
Incidence of clinically significant findings in vital signs
Time frame: Screening and pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Incidence of clinically significant findings in laboratory values
Time frame: Screening to 24, 48, 72, and 144 hours post-dose
Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs)
Time frame: Screening and pre-dose to 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Comparison of CSTI-500 Cmax values obtained from venous blood draws with values obtained from finger prick samples in PWS subjects
Time frame: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
Comparison of CSTI-500 AUC0-72 values obtained from venous blood draws with values obtained from finger prick samples in PWS subjects
Time frame: Pre-dose to 1, 2, 4, 8, 12, 24, 48, and 72 hours post-dose
Comparison of CSTI-500 AUC0-inf values obtained from venous blood draws with values obtained from finger prick samples in PWS subjects
Time frame: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
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 9 Aug 2023.
- 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.
Italo Biaggioni, MD principal investigator
Vanderbilt Autonomic Dysfunction Center, Vanderbilt University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CSTI-500 in people with Prader-Willi Syndrome, run by ConSynance Therapeutics at 1 site 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 5 inclusion and 12 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 9 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 1 site 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?
Maximum Observed Plasma Concentration (Cmax) (time frame: Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose). The study lists 12 primary outcomes in all. 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 9 Aug 2023.