Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome
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| ClinicalTrials.gov Identifier: NCT05322096 |
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Recruitment Status :
Active, not recruiting
First Posted : April 11, 2022
Last Update Posted : November 24, 2023
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Sponsor:
Gedeon Richter Plc.
Information provided by (Responsible Party):
Gedeon Richter Plc.
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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 |
| 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 |
| Actual Study Start Date : | September 22, 2022 |
| Estimated Primary Completion Date : | April 2024 |
| Estimated Study Completion Date : | April 2024 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Prader-Willi syndrome
MedlinePlus related topics:
Prader-Willi Syndrome
| Arm | Intervention/treatment |
|---|---|
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Experimental: RGH-706
Part A: Dose A once daily for 6 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 |
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Placebo Comparator: Placebo
Part A: Placebo once daily for 6 weeks Part B: Placebo once daily for 13 weeks |
Drug: Placebo
Capsules Oral administration |
Primary Outcome Measures :
- 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.
- 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 :
- 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.
- 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.
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- 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.
- 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).
- 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).
- 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).
- 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).
- 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 ]
- 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)
- 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])
- 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 ]
- 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.
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- 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 ]
- 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
- 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
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
Locations
Show 26 study locations
Sponsors and Collaborators
Gedeon Richter Plc.
| 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: | November 24, 2023 |
| Last Verified: | November 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Gedeon Richter Plc.:
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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 Imprinting Disorders Obesity Overweight Overnutrition Nutrition Disorders |