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Completed

NCT Number: NCT06046443

A Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity

The goal of this study is to determine how well LB54640 works and how safe it is in patients with Hypothalamic Obesity (HO). The study will evaluate the effect of LB54640 on safety, weight reduction, hunger, and quality of life in patients 12 years of age and older with HO. Patients will take an oral daily dose of either LB54640 (low, middle, or high dose) or placebo through Week 14. Eligible patients who consent to continue in the study after Week 14 will take an oral daily dose of LB54640 through Week 56.

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Key information

Conditions

Hypothalamic Obesity Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Sex Development Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Gonadal Disorders Gonadal Dysgenesis Hypogonadism Male Urogenital Diseases Sexual Infantilism Urogenital Abnormalities Urogenital Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Birmingham Women's and Children's NHS Foundation Trust, Birmingham, United Kingdom

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Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Documented evidence of acquired hypothalamic obesity (HO)
  • Age 12 years and older
  • Weight gain associated with the hypothalamic injury and a BMI of ≥30 kg/m2 for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients <18 years of age
  • Male and female participants agree to follow study contraception requirements and use a highly effective form of contraception throughout the study and for 90 days after the study

Key Exclusion Criteria:

  • Weight loss >2% in the previous 3 months for patients ≥18 years of age or >2% reduction in BMI for patients <18 years of age
  • History of major surgical procedure within 30 days
  • HbA1c >10.9%
  • Fasting glucose level >270 mg/dL
  • Previous use of MC4R agonists
  • Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET).
  • Diagnosis of severe psychiatric disorders; any suicidal ideation, attempt or behavior
  • History or close family history of skin cancer or melanoma
  • Current clinically significant pulmonary, cardiac, or oncologic disease considered severe enough to interfere with the study and/or confound the results

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

LB54640

Drug

Oral daily administration

Placebo

Drug

Placebo matched to LB54640

Primary outcomes

  1. Mean percentage change in BMI

    Time frame: From Baseline to Week 14

Secondary outcomes

  1. Frequency and severity of adverse events (AEs)

    Time frame: From Baseline to Week 52

  2. Mean change in BMI

    Time frame: Baseline to Weeks 6, 10, 14

  3. Proportion of patients who achieve ≥5% reduction in body weight

    Time frame: Baseline to Week 14

  4. Mean change in the weekly average of the daily most hunger score

    Time frame: Baseline to Week 14

  5. Mean change and mean percentage change from baseline in body weight

    Time frame: From Baseline to Week 52

  6. Mean change and mean percentage change from baseline in waist circumference.

    Time frame: From Baseline to Week 52

  7. Mean change and mean percentage change from baseline in body composition assessed by dual energy x-ray absorptiometry

    Time frame: From Baseline to Week 52

    Fat mass and lean mass will be measured through dual energy x-ray absorptiometry

Sponsors and collaborators

Lead sponsor

Rhythm Pharmaceuticals, Inc.

Industry

Registry information

Official study title

A Randomized, Placebo-controlled, Double-Blind Phase 2 Study to Assess Efficacy and Safety of LB54640 in Patients With Hypothalamic Obesity, With an Open-Label Extension

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Sep 21, 2023
Registry last updated
Feb 12, 2026

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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