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

NCT ID: NCT05322096

Last Updated: 2024-06-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

39 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-09-22

Study Completion Date

2024-04-10

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Prader-Willi Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

RGH-706

Dose A once daily for 6 weeks

Group Type EXPERIMENTAL

RGH-706

Intervention Type DRUG

Capsules

Oral administration

Placebo

Placebo once daily for 6 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Capsules

Oral administration

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

RGH-706

Capsules

Oral administration

Intervention Type DRUG

Placebo

Capsules

Oral administration

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Gedeon Richter Plc.

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Rady Children's Hospital-San Diego

San Diego, California, United States

Site Status

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

Chicago, Illinois, United States

Site Status

Maimonides Medical Center

Brooklyn, New York, United States

Site Status

NYU Langone Hospital-Long Island

Mineola, New York, United States

Site Status

Morgan Stanley Children's Hospital of NewYork-Presbyterian

New York, New York, United States

Site Status

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status

General University Hospital

Prague, , Czechia

Site Status

Centre Hospitalier Universitaire d'Angers

Angers, , France

Site Status

Centre Hospitalier Lyon-Sud

Pierre-Bénite, , France

Site Status

Hôpital Larrey

Toulouse, , France

Site Status

Azienda Ospedaliero-Universitaria Careggi

Florence, , Italy

Site Status

Istituto Giannina Gaslini

Genova, , Italy

Site Status

Azienda Ospedaliera Universitaria "Federico II"

Napoli, , Italy

Site Status

Fondazione Policlinico Universitario Agostino Gemelli

Roma, , Italy

Site Status

IRCCS Ospedale Pediatrico Bambino Gesù

Roma, , Italy

Site Status

Oasi Maria SS

Troina, , Italy

Site Status

Hospital General Universitario Dr. Balmis

Alicante, , Spain

Site Status

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

Madrid, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital Regional Universitario de Málaga - Hospital General

Málaga, , Spain

Site Status

Hospital Universitario Virgen de la Victoria

Málaga, , Spain

Site Status

Parc Taulí Sabadell Hospital Universitari

Sabadell, , Spain

Site Status

Hospital Universitario Virgen del Rocío

Seville, , Spain

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Czechia France Italy Spain

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2021-004262-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

RGH-706-003

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.