Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome
- Registration Number
- NCT05322096
- Lead Sponsor
- Gedeon Richter Plc.
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 39
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description RGH-706 RGH-706 Dose A once daily for 6 weeks Placebo Placebo Placebo once daily for 6 weeks
- Primary Outcome Measures
Name Time Method There are no Primary Outcome Measures There are no Primary Outcome Measures
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (23)
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
Maimonides Medical Center
🇺🇸Brooklyn, New York, United States
Rady Children's Hospital-San Diego
🇺🇸San Diego, California, 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
Centre Hospitalier Universitaire d'Angers
🇫🇷Angers, France
Centre Hospitalier Lyon-Sud
🇫🇷Pierre-Bénite, France
General University Hospital
🇨🇿Prague, Czechia
Hôpital Larrey
🇫🇷Toulouse, France
Istituto Giannina Gaslini
🇮🇹Genova, Italy
Azienda Ospedaliero-Universitaria Careggi
🇮🇹Firenze, Italy
Azienda Ospedaliera Universitaria "Federico II"
🇮🇹Napoli, Italy
IRCCS Ospedale Pediatrico Bambino Gesù
🇮🇹Roma, Italy
Fondazione Policlinico Universitario Agostino Gemelli
🇮🇹Roma, Italy
Oasi Maria SS
🇮🇹Troina, Italy
Hospital General Universitario Dr. Balmis
🇪🇸Alicante, Spain
Hospital General Universitario Gregorio Maranon-Instituto Provincial de Psiquiatria y Salud Mental
🇪🇸Madrid, Spain
Hospital Universitario Virgen de la Victoria
🇪🇸Malaga, Spain
Hospital Regional Universitario de Málaga - Hospital General
🇪🇸Málaga, Spain
Hospital Universitario Virgen del Rocío
🇪🇸Sevilla, Spain
Parc Taulí Sabadell Hospital Universitari
🇪🇸Sabadell, Spain
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain