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Completed

NCT Number: NCT05322096

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).

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Body Weight Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight Pathological Conditions, Signs and Symptoms Signs and Symptoms

Age range

17 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

General University Hospital, Prague, Czechia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

RGH-706

Drug

Capsules

Oral administration

Placebo

Drug

Capsules

Oral administration

Primary outcomes

  1. There are no Primary Outcome Measures

    Time frame: There are no Primary Outcome Measures

Other outcomes

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

    Time frame: Baseline to Day 42

    The HQ-CT is a questionnaire designed to measure symptoms of food-related preoccupations, problems, and behaviors completed by the caregiver. The scale provides a composite value from 9 questions, each rated on a scale of 0 to 4 units (possible total score range: 0 to 36).

    Higher scores represent increased hyperphagia.

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

    Time frame: Baseline to Days 28, 56, 98 and 133

    The HQ-CT is a questionnaire designed to measure symptoms of food-related preoccupations, problems, and behaviors completed by the caregiver. It consists of 9 items, with a 2-week recall period. The scale provides a composite value from 9 questions, each rated on a scale of 0 to 4 units (possible total score range: 0 to 36). Higher scores represent increased hyperphagia.

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

    Time frame: Baseline to Days 28, 42, 56, 98 and 133

    The HQ-CT is a questionnaire designed to measure symptoms of food-related preoccupations, problems, and behaviors completed by the caregiver. The scale provides a composite value from 9 questions, each rated on a scale of 0 to 4 units (possible total score range: 0 to 36). Higher scores represent increased hyperphagia.

  4. Absolute change from baseline in body weight

    Time frame: Screening, Days 1, 14, 28, 42, 56, 98 and 133

  5. Percentage change from baseline in body weight

    Time frame: Screening, Days 1, 14, 28, 42, 56, 98 and 133

  6. Change from baseline in waist circumference

    Time frame: Screening, Days 1, 14, 28, 42, 56, 98 and 133

  7. Change from baseline in body mass index (BMI)

    Time frame: Screening, Days 1, 14, 28, 42, 56, 98 and 133

  8. Change from baseline in metabolic biomarkers measured from serum

    Time frame: Baseline to Day 42

  9. Change from baseline in Clinical Global Impression-Severity (CGI-S)

    Time frame: Baseline to Days 28, 42 and 56

    The CGI-S rates overall symptom severity on a 4-point scale ranging from 1 (normal) to 7 (severely symptomatic), as assessed by the investigator.

  10. Clinical Global Impression-Improvement (CGI-I)

    Time frame: Days 28 and 42

    The CGI-I is a single statement designed to assess the investigator's overall perception of change in the patient's condition across the course of the clinical trial. The CGI-I uses a 7-point response scale ranging from 1 (very much improved) to 7 (very much worse).

  11. Change from baseline in Caregiver Global Impression-Severity (CaGI-S)

    Time frame: Baseline to Days 2 and 42

    The CaGI-S rates severity of the patient's food-related behavior assessed by the caregiver following a 4-point scale ranging from 0 (none) to 3 (severe).

  12. Caregiver Global Impression-Change (CaGI-C)

    Time frame: Days 28, 42, 56, 98 and 133

    The CaGI-C is a single item designed to assess the primary caregiver's overall perception of change in the patient's hyperphagia symptoms. Responses are rated using a 7-point scale ranging from 1 (much better) to 7 (much worse).

  13. Change from baseline in Zarit Burden Interview-22 (ZBI-22)

    Time frame: Baseline to Day 42

    The ZBI-22 is a self-reported questionnaire in which primary caregivers rate the level of burden currently experienced while taking care of the patient rated on a 5-point scale ranging from 0 (never) to 4 (nearly always).

  14. Safety - Incidence of treatment-emergent adverse events (TEAEs)

    Time frame: Screening thru study end; Up to 24 weeks

  15. Safety - Incidence of clinically significant findings in laboratory values

    Time frame: Screening thru study end; Up to 24 weeks

    Clinical laboratory evaluations (hematology, clinical chemistry, coagulation and lipids, thyroid function test, and urinalysis)

  16. Safety - Incidence of clinically significant findings in vital signs

    Time frame: Screening thru study end; Up to 24 weeks

    Vital signs measurements (body temperature, pulse rate, respiration rate, blood pressure [BP])

  17. Safety - Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs)

    Time frame: Screening thru study end; Up to 24 weeks

  18. Safety - Incidence of clinically significant findings in Columbia-Suicide Severity Rating Scale (C-SSRS)

    Time frame: Screening thru study end; Up to 24 weeks

    The C-SSRS is a clinician-rated instrument that captures the occurrence, severity, and frequency of suicidal ideation and/or behavior during the assessment period. The scale includes suggested questions to solicit the type of information needed to determine if suicidal ideation and/or behavior occurred.

  19. Safety - Incidence of clinically significant findings in laboratory values physical examinations

    Time frame: Screening thru study end; Up to 24 weeks

  20. PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 maximum observed plasma concentration (Cmax)

    Time frame: Days 14, 42, 43, 44 and 46

  21. PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 time of the maximum observed plasma concentration (Tmax)

    Time frame: Days 14, 42, 43, 44 and 46

  22. PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 area under the plasma concentration-time curve to the end of the dosing period (AUC0-24)

    Time frame: Days 14, 42, 43, 44 and 46

  23. PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 minimum observed plasma concentration (Cmin)

    Time frame: Days 14, 42, 43, 44 and 46

  24. PK Cohort - Individual plasma concentrations of RGH-706 and its metabolite desisopropyl RGH-706 accumulation ratio (Rac)

    Time frame: Days 14, 42, 43, 44 and 46

Sponsors and collaborators

Lead sponsor

Gedeon Richter Plc.

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2024
Study completion
2024
First posted
Apr 11, 2022
Registry last updated
Jun 3, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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