Prader-Willi SyndromePhase 2NCT05322096
Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome
A Phase 2 study testing RGH-706 in people with Prader-Willi Syndrome, run by Gedeon Richter Plc. at 23 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 17 years and older
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- RGH-706
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 23 sitesin 5 countries
- Study dates
- 22 Sep 2022to 10 Apr 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 17 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 13Show fewer
- 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
Trial locations and contacts
23 sites in 5 countries.
23 sites in 5 countries
Spain7 sites
Hospital General Universitario Dr. Balmis
Alicante, 03010
Hospital General Universitario Gregorio Maranon-Instituto Provincial de Psiquiatria y Salud Mental
Madrid, 28009
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Regional Universitario de Málaga - Hospital General
Málaga, 29010
Hospital Universitario Virgen de la Victoria
Málaga, 29010
Parc Taulí Sabadell Hospital Universitari
Sabadell, 08208
Hospital Universitario Virgen del Rocío
Seville, 41013
United States6 sites
Maimonides Medical Center
Brooklyn, New York, 11219
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
NYU Langone Hospital-Long Island
Mineola, New York, 11501
Morgan Stanley Children's Hospital of NewYork-Presbyterian
New York, New York, 10032
Rady Children's Hospital-San Diego
San Diego, California, 92123
Italy6 sites
Azienda Ospedaliero-Universitaria Careggi
Florence, 50139
Istituto Giannina Gaslini
Genova, 16147
Azienda Ospedaliera Universitaria "Federico II"
Naples, 80131
Fondazione Policlinico Universitario Agostino Gemelli
Roma, 168
IRCCS Ospedale Pediatrico Bambino Gesù
Roma, 50
Oasi Maria SS
Troina, 94018
France3 sites
Centre Hospitalier Universitaire d'Angers
Angers, 49933
Centre Hospitalier Lyon-Sud
Pierre-Bénite, 69495
Hôpital Larrey
Toulouse, 31059
About this study
Official 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
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
- Being tested
- RGH-706, Placebo
- Keywords
- Prader-Willi Syndrome, Obesity, Hyperphagia control
- Medical subject headings
- Prader-Willi Syndrome, Obesity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RGH-706Drug
Capsules
Oral administration
PlaceboDrug
Capsules
Oral administration
| Study group | Role | What they receive |
|---|---|---|
| RGH-706 | Experimental | Dose A once daily for 6 weeks Receives: Drug: RGH-706 |
| Placebo | Placebo comparator | Placebo once daily for 6 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
There are no Primary Outcome Measures
Measured: There are no Primary Outcome Measures
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2022
- Study start22 Sep 2022
- Primary completion10 Apr 2024
- Study completion10 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gedeon Richter Plc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05322096
- Sponsor's protocol ID
- RGH-706-003
- Other IDs
- 2021-004262-35
- Status last verified
- May 2024
- First submitted
- 25 Mar 2022
- Last updated
- 3 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05322096
- Acronym / sponsor ID
- RGH-706-003
- Conditions
- Prader-Willi Syndrome
- Keywords
- Prader-Willi Syndrome, Obesity, Hyperphagia control
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Gedeon Richter Plc.Industry (pharma, biotech, devices)
- Study start
- 22 Sep 2022 (actual)
- Primary completion
- 10 Apr 2024 (actual)
- Study completion
- 10 Apr 2024 (actual)
- First posted
- 11 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RGH-706 | Experimental | Drug: RGH-706Dose A once daily for 6 weeks |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo once daily for 6 weeks |
Who may qualify
- Ages
- 17 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RGH-706Capsules Oral administration | Drug | RGH-706 |
| PlaceboCapsules Oral administration | Drug | Placebo |
Primary outcome
There are no Primary Outcome Measures
Time frame: There are no Primary Outcome Measures
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RGH-706 in people with Prader-Willi Syndrome, run by Gedeon Richter Plc. at 23 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in Spain, United States, Italy, France, Czechia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
There are no Primary Outcome Measures (time frame: There are no Primary Outcome Measures). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2024.