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

NCT05504395 | Completed Phase 1 FDA Drug

• 1 other identifier: CSTI-500-003
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Prader-Willi Syndrome

Keywords

PWS; Phase 1; Single Dose; Non randomized; PK; Safety

Outcome Measures

Primary Outcomes (12)

• Maximum Observed Plasma Concentration (Cmax)

  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

  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

  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

  1 hour post-dose

• CSTI-500 plasma concentration

  2 hour post-dose

• CSTI-500 plasma concentration

  4 hour post-dose

• CSTI-500 plasma concentration

  8 hour post-dose

• CSTI-500 plasma concentration

  12 hour post-dose

• CSTI-500 plasma concentration

  24 hour post-dose

• CSTI-500 plasma concentration

  48 hour post-dose

• CSTI-500 plasma concentration

  72 hour post-dose

• CSTI-500 plasma concentration

  144 hour post-dose

Secondary Outcomes (8)

• Incidence of treatment-emergent adverse events (TEAEs)

  From pre-dose to 15 days post-dose

• Incidence of clinically significant findings in physical examinations

  Screening to 12, 24, 48, 72, and 144 hours post-dose

• Incidence of clinically significant findings in vital signs

  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

  Screening to 24, 48, 72, and 144 hours post-dose

• Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs)

  Screening and pre-dose to 2, 4, 8, 12, 24, 48, 72, and 144 hours post-dose

Study Arms (1)

CSTI-500 10mg

EXPERIMENTAL

All eligible subjects will be administered a single oral dose of CSTI-500 10 mg at Visit 2

Drug: CSTI-500

Interventions

CSTI-500 DRUG

Single 10 mg capsule

Also known as: AMR-001181

CSTI-500 10mg

Eligibility Criteria

• Age: 13 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• ConSynance Therapeutics: lead

Study Sites (1)

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Study Officials

• Italo Biaggioni, MD

  Vanderbilt Autonomic Dysfunction Center, Vanderbilt University Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 4, 2022
• First Posted: August 17, 2022
• Study Start: November 14, 2022
• Primary Completion: February 21, 2023
• Study Completion: February 21, 2023
• Last Updated: August 9, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)