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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05322096
Recruitment Status : Not yet recruiting
First Posted : April 11, 2022
Last Update Posted : August 18, 2022
Sponsor:
Information provided by (Responsible Party):
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).


Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: RGH-706 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 176 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled, Multi-center, 2-part, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome
Estimated Study Start Date : August 2022
Estimated Primary Completion Date : April 2024
Estimated Study Completion Date : April 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: RGH-706

Part A: Dose A once daily for 4 weeks

Part B: Dose B, Dose C or Dose D once daily (depending on the randomized arm) for 13 weeks

Drug: RGH-706

Capsules

Oral administration


Placebo Comparator: Placebo

Part A: Placebo once daily for 4 weeks

Part B: Placebo once daily for 13 weeks

Drug: Placebo

Capsules

Oral administration





Primary Outcome Measures :
  1. Part A: Change from baseline in the 9-item Hyperphagia Questionnaire for Clinical Trials (HQ-CT) [ Time Frame: Part A: 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. Part B: Change from baseline in the 9-item Hyperphagia Questionnaire for Clinical Trials (HQ-CT) [ Time Frame: Part B: Baseline to Day 105 ]
    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.


Secondary Outcome Measures :
  1. Part A and Part B: Change from baseline in the 9-item Hyperphagia Questionnaire for Clinical Trials (HQ-CT) [ Time Frame: Part A: Baseline to Days 28, 56, 98 and 133; Part B: Baseline to Days 42, 70 and 119 ]
    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.

  2. Part A and Part B: Change from baseline in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) domain scores (drive and severity, self-directed behavior) [ Time Frame: Part A: Baseline to Days 28, 42, 56, 98 and 133; Part B: Baseline to Days 42, 70, 105 and 119 ]
    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.

  3. Part A and Part B: Absolute change from baseline in body weight [ Time Frame: Part A: Screening, Days 1, 14, 28, 42, 56, 98 and 133; Part B: Screening, Days 1, 14, 28, 42, 70, 105 and 119 ]
  4. Part A and Part B: Percentage change from baseline in body weight [ Time Frame: Part A: Screening, Days 1, 14, 28, 42, 56, 98 and 133; Part B: Screening, Days 1, 14, 28, 42, 70, 105 and 119 ]
  5. Part A and Part B: Change from baseline in waist circumference [ Time Frame: Part A: Screening, Days 1, 14, 28, 42, 56, 98 and 133; Part B: Screening, Days 1, 14, 28, 42, 70, 105 and 119 ]
  6. Part A and Part B: Change from baseline in body mass index (BMI) [ Time Frame: Part A: Screening, Days 1, 14, 28, 42, 56, 98 and 133; Part B: Screening, Days 1, 14, 28, 42, 70, 105 and 119 ]
  7. Part A and Part B: Change from baseline in metabolic biomarkers measured from serum [ Time Frame: Part A: Baseline to Day 42; Part B: Baseline to Days 42, 70 and 105 ]
  8. Part A and Part B: Change from baseline in Clinical Global Impression-Severity (CGI-S) [ Time Frame: Part A: Baseline to Days 28, 42 and 56; Part B: Baseline to Days 42, 70, 105 and 119 ]
    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.

  9. Part A and Part B: Clinical Global Impression-Improvement (CGI-I) [ Time Frame: Part A: Days 28 and 42; Part B: Baseline to Days 42, 70 and 105 ]
    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).

  10. Part A and Part B: Change from baseline in Caregiver Global Impression-Severity (CaGI-S) [ Time Frame: Part A: Baseline to Days 2 and 42; Part B: Baseline to Days 42, 70 and 105 ]
    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).

  11. Part A and Part B: Caregiver Global Impression-Change (CaGI-C) [ Time Frame: Part A: Days 28, 42, 56, 98 and 133; Part B: Days 42, 70, 105 and 119 ]
    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).

  12. Part A and Part B: Change from baseline in Zarit Burden Interview-22 (ZBI-22) [ Time Frame: Part A: Baseline to Day 42; Part B: Baseline to Day 105 ]
    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).

  13. Part A and Part B: Safety - Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: Part A: Screening thru study end; Up to 24 weeks; Part B: Screening to end of study; Up to 17 weeks ]
  14. Part A and Part B: Safety - Incidence of clinically significant findings in laboratory values [ Time Frame: Part A: Screening thru study end; Up to 24 weeks; Part B: Screening to end of study; Up to 17 weeks ]
    Clinical laboratory evaluations (hematology, clinical chemistry, coagulation and lipids, thyroid function test, and urinalysis)

  15. Part A and Part B: Safety - Incidence of clinically significant findings in vital signs [ Time Frame: Part A: Screening thru study end; Up to 24 weeks; Part B: Screening to end of study; Up to 17 weeks ]
    Vital signs measurements (body temperature, pulse rate, respiration rate, blood pressure [BP])

  16. Part A and Part B: Safety - Incidence of clinically significant findings in 12-lead electrocardiograms (ECGs) [ Time Frame: Part A: Screening thru study end; Up to 24 weeks; Part B: Screening to end of study; Up to 17 weeks ]
  17. Part A and Part B: Safety - Incidence of clinically significant findings in Columbia-Suicide Severity Rating Scale (C-SSRS) [ Time Frame: Part A: Screening thru study end; Up to 24 weeks; Part B: Screening to end of study; Up to 17 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.

  18. Part A and Part B: Safety - Incidence of clinically significant findings in laboratory values physical examinations [ Time Frame: Part A: Screening thru study end; Up to 24 weeks; Part B: Screening to end of study; Up to 17 weeks ]
  19. Part B: Change from baseline in total body mass measured by Lunar dual-energy X-ray absorptiometry (iDXA) [ Time Frame: Part B: Baseline to Day 105 ]
  20. Part B: Change from baseline in lean body mass measured by Lunar dual-energy X-ray absorptiometry (iDXA) [ Time Frame: Part B: Baseline to Day 105 ]
  21. Part B: Change from baseline in total fat mass measured by Lunar dual-energy X-ray absorptiometry (iDXA) [ Time Frame: Part B: Baseline to Day 105 ]
  22. Part B: Change from baseline in visceral fat mass measured by Lunar dual-energy X-ray absorptiometry (iDXA) [ Time Frame: Part B: Baseline to Day 105 ]
  23. Part B: Change from baseline in subcutaneous fat mass measured by Lunar dual-energy X-ray absorptiometry (iDXA) [ Time Frame: Part B: Baseline to Day 105 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   17 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  • Severe 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05322096


Contacts
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Contact: Medical Information Scientific Service +36 1 505 7032 medinfo@richter.hu

Locations
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United States, California
Gedeon Richter Investigative Site
San Diego, California, United States, 92123
United States, Illinois
Gedeon Richter Investigative Site
Chicago, Illinois, United States, 60611
United States, New York
Gedeon Richter Investigative Site
Brooklyn, New York, United States, 11219
Gedeon Richter Investigative Site
Mineola, New York, United States, 11501
Gedeon Richter Investigative Site
New York, New York, United States, 10032
United States, Ohio
Gedeon Richter Investigative Site
Cleveland, Ohio, United States, 44106
United States, Utah
Gedeon Richter Investigative Site
Salt Lake City, Utah, United States, 84112
Czechia
Gedeon Richter Investigative Site
Prague, Czechia, 12808
Gedeon Richter Investigative Site
Prague, Czechia, 15000
France
Gedeon Richter Investigative Site
Angers, France, 49933
Gedeon Richter Investigative Site
Lille, France, 59000
Gedeon Richter Investigative Site
Pierre-Bénite, France, 69495
Gedeon Richter Investigative Site
Toulouse, France, 31059
Italy
Gedeon Richter Investigative Site
Firenze, Italy, 50139
Gedeon Richter Investigative Site
Genova, Italy, 16147
Gedeon Richter Investigative Site
Napoli, Italy, 80131
Gedeon Richter Investigative Site - 0406
Roma, Italy, 168
Gedeon Richter Investigative Site - 0403
Roma, Italy, 50
Gedeon Richter Investigative Site
Troina, Italy, 94018
Spain
Gedeon Richter Investigative Site - 304
Madrid, Spain, 28009
Gedeon Richter Investigative Site - 303
Madrid, Spain, 28041
Gedeon Richter Investigative Site - 305
Malaga, Spain, 29010
Gedeon Richter Investigative Site - 306
Málaga, Spain, 29010
Gedeon Richter Investigative Site
Sabadell, Spain, 08208
Gedeon Richter Investigative Site
Sevilla, Spain, 41013
Sponsors and Collaborators
Gedeon Richter Plc.
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Responsible Party: Gedeon Richter Plc.
ClinicalTrials.gov Identifier: NCT05322096    
Other Study ID Numbers: RGH-706-003
2021-004262-35 ( EudraCT Number )
First Posted: April 11, 2022    Key Record Dates
Last Update Posted: August 18, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Gedeon Richter Plc.:
Prader-Willi Syndrome
Obesity
Hyperphagia control
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders