A Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome

August 8, 2023 updated by: ConSynance Therapeutics

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
10

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

13 years to 50 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: CSTI-500 10mg
All eligible subjects will be administered a single oral dose of CSTI-500 10 mg at Visit 2
Drug: CSTI-500
Single 10 mg capsule
Other Names:
  • AMR-001181

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
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
Pre-dose to 1, 2, 4, 8, 12, 24, 48, and 72 hours post-dose
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
Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose
CSTI-500 plasma concentration
Time Frame: 1 hour post-dose
1 hour post-dose
CSTI-500 plasma concentration
Time Frame: 2 hour post-dose
2 hour post-dose
CSTI-500 plasma concentration
Time Frame: 4 hour post-dose
4 hour post-dose
CSTI-500 plasma concentration
Time Frame: 8 hour post-dose
8 hour post-dose
CSTI-500 plasma concentration
Time Frame: 12 hour post-dose
12 hour post-dose
CSTI-500 plasma concentration
Time Frame: 24 hour post-dose
24 hour post-dose
CSTI-500 plasma concentration
Time Frame: 48 hour post-dose
48 hour post-dose
CSTI-500 plasma concentration
Time Frame: 72 hour post-dose
72 hour post-dose
CSTI-500 plasma concentration
Time Frame: 144 hour post-dose
144 hour post-dose

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Incidence of treatment-emergent adverse events (TEAEs)
Time Frame: From pre-dose to 15 days post-dose
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.
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
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
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.
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
Laboratory evaluations include hematology, thyroid function, and chemistry blood and urine laboratory tests.
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
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
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
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
Pre-dose to 1, 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
ConSynance Therapeutics

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Italo Biaggioni, MD, Vanderbilt Autonomic Dysfunction Center, Vanderbilt University Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 14, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 21, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 21, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
August 4, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 16, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 17, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 9, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 8, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CSTI-500-003

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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