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

Phase 2

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: RGH-706; Drug: Placebo

• 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

Study Timeline

• Study First Submitted: 2022-03-25
• Study First Submitted QC: 2022-04-04
• Study First Posted: 2022-04-11
• Study Start: 2022-09-22
• Primary Completion: 2024-04-10
• Study Completion: 2024-04-10
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-30
• Last Update Posted: 2024-06-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 39

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

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

Trial Locations

Locations (23)

Rady Children's Hospital-San Diego; 🇺🇸 San Diego, California, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

Maimonides Medical Center; 🇺🇸 Brooklyn, New York, 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

University Hospitals Cleveland Medical Center; 🇺🇸 Cleveland, Ohio, United States

General University Hospital; 🇨🇿 Prague, Czechia

Centre Hospitalier Universitaire d'Angers; 🇫🇷 Angers, France

Centre Hospitalier Lyon-Sud; 🇫🇷 Pierre-Bénite, France

Hôpital Larrey; 🇫🇷 Toulouse, France

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