Skip to content

Trial · Interventional

RecruitingPhase 4Registry data

Real-World Effects of MC4R Agonist Therapy in BBS and Severe Genetic Obesity

Short name REAL-MC4

NCTNCT07674290 (opens ClinicalTrials.gov in a new tab)Other idUME-BBS-RWD-001
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Recruiting
Phase
Phase 4
Enrollment
200 participants (estimated)
Sponsor
Tom Hühne · Other
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

Germany

Start
1 Jan 2023
Primary completion
31 Dec 2030 (estimated)
Completion
31 Dec 2030 (estimated)
First posted
29 Jun 2026
Last update posted
29 Jun 2026
Conditions
Bardet Biedl Syndrome (BBS) · Bardet Biedl Syndrome · Bardet-Biedl Syndrome (BBS) · Alstrom Syndrome

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2024202620282030
  1. Ph 4

    REAL-MC4

    NCT07674290

    1 Jan 2023 to est. 31 Dec 2030

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

Bardet-Biedl syndrome (BBS) and other rare disorders associated with impairment of the melanocortin-4 receptor (MC4R) pathway are characterized by severe early-onset obesity, hyperphagia, and substantial morbidity. Setmelanotide, an MC4R agonist, is approved in Europe for selected genetic obesity disorders and reimbursed in Germany for eligible patients. This study aims to evaluate the effectiveness, safety, treatment persistence, metabolic outcomes, and patient-reported outcomes of Setmelanotide under real-world conditions. The registry is designed to allow future inclusion of additional MC4R agonists as they become approved and clinically available. The study will primarily be conducted at University Hospital Essen and will collect longitudinal routine clinical data from pediatric and adult patients receiving MC4R agonist therapy according to approved indications.

Interventions

  • SetmelanotideDrug

Arms1

  • Experimental

    MC4R Therapy

    Patients receiving approved MC4 receptor agonists according to licensed indications and routine clinical practice.

Primary outcomes3

Outcomes the sponsor listed as primary.

  1. Percent change in BMI z-score

    Time frame · Baseline to 12/24/36/48/60/72 months

    Registry description

    Relative change in BMI z-Score after initiation of MC4 receptor agonist therapy

  2. Impact on lipid profile

    Time frame · Baseline to 12/24/36/48/60/72 months

    Registry description

    Changes in lipid profile measured by cholesterol blood levels

  3. Change in Hepatic Fat Attenuation

    Time frame · Baseline to 12/24/36/48/60/72 months

    Registry description

    Hepatic Fat Attenuation will be measured by ultrasound Attenuation imaging across different time points

Secondary outcomes(5)

  1. Life quality

    Time frame · Baseline to 12/24/36/48/60/72 months

    Registry description

    Patient-reported quality of life using e.g. the "Impact of weight on Quality of life"-questionnaire (IWQOL). The assessment is based on a scale from 0 to 100, with 100 representing the best possible weight-related quality of life.

  2. Safety and Tolerability

    Time frame · Baseline to 12/24/36/48/60/72 months

    Registry description

    Incidence of adverse events, serious adverse events and treatment discontinuations and reasons for it

  3. Cognitive changes

    Time frame · Baseline to 12/24/36/48/60/72 months

    Registry description

    Neurocognitive impairment is common in Bardet-Biedl Syndrome. Wechsler Intelligence Scale for Children - Fourth Edition (WISC-IV) for children and Wechsler Adult Intelligence Scale (WAIS) for adults are performed to investigate cognition before and after intervention.

  4. Functional brain connectivity

    Time frame · Baseline to 12/24/36/48/60/72 months

    Registry description

    Newly diagnosed patients undergo non-invasive functional magnetic resonance imaging (fMRI) both prior to treatment initiation and three months afterward. The scanning protocol will include structural T1-weighted MRI sequences (8 minutes), resting-state fMRI (4 runs of 5.5 minutes each; 22 minutes total), and task-based fMRI to assess responses to high- and low-fat food cues (2 runs of 5.5 minutes each; 11 minutes total). The imaging component will enable the investigation of treatment-related changes in functional brain connectivity associated with setmelanotide.

  5. Changes on hypothalamic-pituitary-gonadal axis

    Time frame · Baseline to 12/24/36/48/60/72 months

    Registry description

    Hypothalamic-pituitary-gonadal axis is investigated by longitudinal measurements of testosterone and estradiol levels in blood.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies6

Studies that cite this trial.

Studies with results

4 studies on this page

Indexing hint: Observational study · 2026

8 results

Impact of the Melanocortin-4 Receptor Agonist Setmelanotide on MASLD and Kidney Function in Bardet-Biedl Syndrome.

Hühne et al. 2026The Journal of clinical endocrinology and metabolismcited by 2

PMID 40903014DOI 10.1210/clinem/dgaf483PUB-40903014

Full text at the publisher8 results
Publisher pageSee results (8)

Indexing hint: Clinical trial · 2023

6 results

Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results.

Forsythe et al. 2023Orphanet journal of rare diseasescited by 34

PMID 36647077PMC9841937DOI 10.1186/s13023-022-02602-4PUB-36647077

CC BY6 results
Download PDFSee results (6)

Indexing hint: Review · 2023

Targeting the central melanocortin system for the treatment of metabolic disorders.

Sweeney et al. 2023Nature reviews. Endocrinologycited by 52

PMID 37365323DOI 10.1038/s41574-023-00855-yPUB-37365323

Full text at the publisher
Publisher page

Indexing hint: In vitro study · 2019

5 results

Setmelanotide, a Novel, Selective Melanocortin Receptor-4 Agonist Exerts Anti-inflammatory Actions in Astrocytes and Promotes an Anti-inflammatory Macrophage Phenotype.

Kamermans et al. 2019Frontiers in immunologycited by 30

PMID 31636637PMC6788433DOI 10.3389/fimmu.2019.02312PUB-31636637

CC BY5 results
Download PDFSee results (5)

Indexing hint: Clinical trial · 2017

7 results

Evaluation of a melanocortin-4 receptor (MC4R) agonist (Setmelanotide) in MC4R deficiency.

Collet et al. 2017Molecular metabolismcited by 237

PMID 29031731PMC5641599DOI 10.1016/j.molmet.2017.06.015PUB-29031731

CC BY7 results
Download PDFSee results (7)

More studies

1 study on this page

Indexing hint: Randomized controlled trial · 2022

Corrected

Efficacy and safety of setmelanotide, a melanocortin-4 receptor agonist, in patients with Bardet-Biedl syndrome and Alström syndrome: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial with an open-label period.

Haqq et al. 2022The lancet. Diabetes & endocrinologycited by 149

PMID 36356613PMC9847480DOI 10.1016/s2213-8587(22)00277-7PUB-36356613

Rights unknown
Source linkRead the notice

Eligibility

Who could join, as posted.

Sex
All
Age
Not stated
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * clinical phenotype corresponding to Bardet-Biedl Syndrome * genetic testing with notable finding Exclusion Criteria: * patients younger than the age approved for treatment with setmelanotide

PepBank is a research reference. Talk to a doctor before you use any peptide.