Prader-Willi Syndrome Clinical Trial
Official title:
A Randomized, Double-blind, Placebo-controlled, Multi-center, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome
Verified date | May 2024 |
Source | Gedeon Richter Plc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
RGH-706 is a novel, potent, and orally active MCHR1 antagonist drug candidate discovered and being developed by Gedeon Richter Plc. for weight management. This will be the first Phase 2, proof-of-concept study using RGH-706 and is the third study in the clinical development program for RGH-706. The aim of this study is to evaluate the efficacy, safety, and tolerability of RGH-706 in patients with Prader-Willi Syndrome (PWS).
Status | Completed |
Enrollment | 39 |
Est. completion date | April 10, 2024 |
Est. primary completion date | April 10, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 17 Years and older |
Eligibility | Age Limits: - In United States (USA), minimum age will be 17 years old. - In European Union (EU) countries, minimum age will be 18 years old. Inclusion Criteria: - Male or female patients aged =17 years in USA at screening or aged =18 years in EU at screening - Genetically confirmed diagnosis of PWS - HQ-CT total score =14 at screening - Body weight =40 kg/88 lbs and =200 kg/450 lbs - Stable body weight - Negative pregnancy test for females of childbearing potential and nonlactating at screening. - Patients must be able to provide or have a parent or guardian who is able to provide written informed consent and/or assent (as applicable) - Patients must have at least 1 consistent and reliable primary caregiver Exclusion Criteria: - Severe psychiatric disorders (eg, schizophrenia, bipolar disorder, or major depressive disorder), recent (within 6 months) - Risk of suicide according to the investigator's judgment - Uncontrollable diabetes mellitus or diabetes mellitus requiring insulin administration - Poorly controlled hypothyroidism or hyperthyroidism - Chronic or acute liver disease - History of bariatric surgery procedure - Uncontrolled obstructive sleep apnea. - History of malignancy within 5 years of screening - Systolic blood pressure (BP) =160 mmHg and/or diastolic BP =100 mmHg, pulse rate =100/min at screening. - Use of weight-lowering pharmacotherapy within 6 months prior to screening. - Known QT prolongation - Clinically relevant laboratory abnormalities - Any other condition that, in the investigator's opinion, might indicate that the patient is unsuitable for the study |
Country | Name | City | State |
---|---|---|---|
Czechia | General University Hospital | Prague | |
France | Centre Hospitalier Universitaire d'Angers | Angers | |
France | Centre Hospitalier Lyon-Sud | Pierre-Bénite | |
France | Hôpital Larrey | Toulouse | |
Italy | Azienda Ospedaliero-Universitaria Careggi | Firenze | |
Italy | Istituto Giannina Gaslini | Genova | |
Italy | Azienda Ospedaliera Universitaria "Federico II" | Napoli | |
Italy | Fondazione Policlinico Universitario Agostino Gemelli | Roma | |
Italy | IRCCS Ospedale Pediatrico Bambino Gesù | Roma | |
Italy | Oasi Maria SS | Troina | |
Spain | Hospital General Universitario Dr. Balmis | Alicante | |
Spain | Hospital General Universitario Gregorio Maranon-Instituto Provincial de Psiquiatria y Salud Mental | Madrid | |
Spain | Hospital Universitario 12 de Octubre | Madrid | |
Spain | Hospital Universitario Virgen de la Victoria | Malaga | |
Spain | Hospital Regional Universitario de Málaga - Hospital General | Málaga | |
Spain | Parc Taulí Sabadell Hospital Universitari | Sabadell | |
Spain | Hospital Universitario Virgen del Rocío | Sevilla | |
United States | Maimonides Medical Center | Brooklyn | New York |
United States | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois |
United States | University Hospitals Cleveland Medical Center | Cleveland | Ohio |
United States | NYU Langone Hospital-Long Island | Mineola | New York |
United States | Morgan Stanley Children's Hospital of NewYork-Presbyterian | New York | New York |
United States | Rady Children's Hospital-San Diego | San Diego | California |
Lead Sponsor | Collaborator |
---|---|
Gedeon Richter Plc. |
United States, Czechia, France, Italy, Spain,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Change from baseline in the 9-item Hyperphagia Questionnaire for Clinical Trials (HQ-CT) | The HQ-CT is a questionnaire designed to measure symptoms of food-related preoccupations, problems, and behaviors completed by the caregiver. The scale provides a composite value from 9 questions, each rated on a scale of 0 to 4 units (possible total score range: 0 to 36).
Higher scores represent increased hyperphagia. |
Baseline to Day 42 | |
Other | Change from baseline in the 9-item Hyperphagia Questionnaire for Clinical Trials (HQ-CT) | The HQ-CT is a questionnaire designed to measure symptoms of food-related preoccupations, problems, and behaviors completed by the caregiver. It consists of 9 items, with a 2-week recall period. The scale provides a composite value from 9 questions, each rated on a scale of 0 to 4 units (possible total score range: 0 to 36). Higher scores represent increased hyperphagia. | Baseline to Days 28, 56, 98 and 133 | |
Other | Change from baseline in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) domain scores (drive and severity, self-directed behavior) | The HQ-CT is a questionnaire designed to measure symptoms of food-related preoccupations, problems, and behaviors completed by the caregiver. The scale provides a composite value from 9 questions, each rated on a scale of 0 to 4 units (possible total score range: 0 to 36). Higher scores represent increased hyperphagia. | Baseline to Days 28, 42, 56, 98 and 133 | |
Other | Absolute change from baseline in body weight | Screening, Days 1, 14, 28, 42, 56, 98 and 133 | ||
Other | Percentage change from baseline in body weight | Screening, Days 1, 14, 28, 42, 56, 98 and 133 | ||
Other | Change from baseline in waist circumference | Screening, Days 1, 14, 28, 42, 56, 98 and 133 | ||
Other | Change from baseline in body mass index (BMI) | Screening, Days 1, 14, 28, 42, 56, 98 and 133 | ||
Other | Change from baseline in metabolic biomarkers measured from serum | Baseline to Day 42 | ||
Other | Change from baseline in Clinical Global Impression-Severity (CGI-S) | The CGI-S rates overall symptom severity on a 4-point scale ranging from 1 (normal) to 7 (severely symptomatic), as assessed by the investigator. | Baseline to Days 28, 42 and 56 | |
Other | Clinical Global Impression-Improvement (CGI-I) | The CGI-I is a single statement designed to assess the investigator's overall perception of change in the patient's condition across the course of the clinical trial. The CGI-I uses a 7-point response scale ranging from 1 (very much improved) to 7 (very much worse). | Days 28 and 42 | |
Other | Change from baseline in Caregiver Global Impression-Severity (CaGI-S) | The CaGI-S rates severity of the patient's food-related behavior assessed by the caregiver following a 4-point scale ranging from 0 (none) to 3 (severe). | Baseline to Days 2 and 42 | |
Other | Caregiver Global Impression-Change (CaGI-C) | The CaGI-C is a single item designed to assess the primary caregiver's overall perception of change in the patient's hyperphagia symptoms. Responses are rated using a 7-point scale ranging from 1 (much better) to 7 (much worse). | Days 28, 42, 56, 98 and 133 | |
Other | Change from baseline in Zarit Burden Interview-22 (ZBI-22) | The ZBI-22 is a self-reported questionnaire in which primary caregivers rate the level of burden currently experienced while taking care of the patient rated on a 5-point scale ranging from 0 (never) to 4 (nearly always). | Baseline to Day 42 | |
Other | Safety - Incidence of treatment-emergent adverse events (TEAEs) | Screening thru study end; Up to 24 weeks | ||
Other | Safety - Incidence of clinically significant findings in laboratory values | Clinical laboratory evaluations (hematology, clinical chemistry, coagulation and lipids, thyroid function test, and urinalysis) | Screening thru study end; Up to 24 weeks | |
Other | Safety - Incidence of clinically significant findings in vital signs | Vital signs measurements (body temperature, pulse rate, respiration rate, blood pressure [BP]) | Screening thru study end; Up to 24 weeks | |
Other | Safety - Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs) | Screening thru study end; Up to 24 weeks | ||
Other | Safety - Incidence of clinically significant findings in Columbia-Suicide Severity Rating Scale (C-SSRS) | The C-SSRS is a clinician-rated instrument that captures the occurrence, severity, and frequency of suicidal ideation and/or behavior during the assessment period. The scale includes suggested questions to solicit the type of information needed to determine if suicidal ideation and/or behavior occurred. | Screening thru study end; Up to 24 weeks | |
Other | Safety - Incidence of clinically significant findings in laboratory values physical examinations | Screening thru study end; Up to 24 weeks | ||
Other | PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 maximum observed plasma concentration (Cmax) | Days 14, 42, 43, 44 and 46 | ||
Other | PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 time of the maximum observed plasma concentration (Tmax) | Days 14, 42, 43, 44 and 46 | ||
Other | PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 area under the plasma concentration-time curve to the end of the dosing period (AUC0-24) | Days 14, 42, 43, 44 and 46 | ||
Other | PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 minimum observed plasma concentration (Cmin) | Days 14, 42, 43, 44 and 46 | ||
Other | PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 accumulation ratio (Rac) | Days 14, 42, 43, 44 and 46 | ||
Primary | There are no Primary Outcome Measures | There are no Primary Outcome Measures |
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