Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome

A Randomized, Double-blind, Placebo-controlled, Multi-center, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome

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).

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