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

NCT05322096 | Completed Phase 2 FDA Drug

• 2 other identifiers: RGH-706-0032021-004262-35
• Study Type: interventional
• Target: 39
• Locations: 5 countries
• Sites: 23

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).

Conditions

Prader-Willi Syndrome

Keywords

Prader-Willi Syndrome; Obesity; Hyperphagia control

Outcome Measures

Primary Outcomes (1)

• There are no Primary Outcome Measures

  There are no Primary Outcome Measures

Other Outcomes (24)

• Change from baseline in the 9-item Hyperphagia Questionnaire for Clinical Trials (HQ-CT)

  Baseline to Day 42

• Change from baseline in the 9-item Hyperphagia Questionnaire for Clinical Trials (HQ-CT)

  Baseline to Days 28, 56, 98 and 133

• Change from baseline in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) domain scores (drive and severity, self-directed behavior)

  Baseline to Days 28, 42, 56, 98 and 133

Study Arms (2)

RGH-706

EXPERIMENTAL

Dose A once daily for 6 weeks

Drug: RGH-706

Placebo

PLACEBO COMPARATOR

Placebo once daily for 6 weeks

Drug: Placebo

Interventions

RGH-706 DRUG

Capsules Oral administration

RGH-706

Placebo DRUG

Capsules Oral administration

Placebo

Eligibility Criteria

• Age: 17 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Gedeon Richter Plc.: lead

Study Sites (23)

Rady Children's Hospital-San Diego

San Diego, California, 92123, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Maimonides Medical Center

Brooklyn, New York, 11219, United States

Location

NYU Langone Hospital-Long Island

Mineola, New York, 11501, United States

Location

Morgan Stanley Children's Hospital of NewYork-Presbyterian

New York, New York, 10032, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

General University Hospital

Prague, 12808, Czechia

Location

Centre Hospitalier Universitaire d'Angers

Angers, 49933, France

Location

Centre Hospitalier Lyon-Sud

Pierre-Bénite, 69495, France

Location

Hôpital Larrey

Toulouse, 31059, France

Location

Azienda Ospedaliero-Universitaria Careggi

Florence, 50139, Italy

Location

Istituto Giannina Gaslini

Genova, 16147, Italy

Location

Azienda Ospedaliera Universitaria "Federico II"

Napoli, 80131, Italy

Location

Fondazione Policlinico Universitario Agostino Gemelli

Roma, 168, Italy

Location

IRCCS Ospedale Pediatrico Bambino Gesù

Roma, 50, Italy

Location

Oasi Maria SS

Troina, 94018, Italy

Location

Hospital General Universitario Dr. Balmis

Alicante, 03010, Spain

Location

Hospital General Universitario Gregorio Maranon-Instituto Provincial de Psiquiatria y Salud Mental

Madrid, 28009, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Regional Universitario de Málaga - Hospital General

Málaga, 29010, Spain

Location

Hospital Universitario Virgen de la Victoria

Málaga, 29010, Spain

Location

Parc Taulí Sabadell Hospital Universitari

Sabadell, 08208, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, 41013, Spain

Location

MeSH Terms

Conditions

Prader-Willi Syndrome; Obesity

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; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 25, 2022
• First Posted: April 11, 2022
• Study Start: September 22, 2022
• Primary Completion: April 10, 2024
• Study Completion: April 10, 2024
• Last Updated: June 3, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (1); FR (3); ES (7); US (6); IT (6)