A Study to Assess the Efficacy, Safety, and Pharmacokinetics of Debio 4326 in Pediatric Participants With Central Precocious Puberty (LIBELULA™ Clinical Trial)

NCT06129539 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: Debio 4326-301
• Study Type: interventional
• Target: 56
• Locations: 5 countries
• Sites: 17

Brief Summary

The primary objective of this study is to evaluate the efficacy of Debio 4326 in suppressing serum luteinizing hormone (LH) to prepubertal levels 52 weeks after the first Debio 4326 injection in pediatric participants with central precocious puberty (CPP).

Conditions

Central Precocious Puberty

Outcome Measures

Primary Outcomes (1)

• Part A: Percentage of Participants With Suppression of Gonadotropin-Releasing Hormone Agonist Stimulated Serum Luteinizing Hormone (LH) to Less Than or Equal to (≤)5 International Units per Liter (IU/L)

  Week 52 in Part A

Secondary Outcomes (22)

• Parts A and B: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs) and Serious TEAEs

  Up to 104 weeks

• Parts A and B: Number of Participants with Clinically Significant Abnormalities in Vital Signs

  Up to 104 weeks

• Parts A and B: Change From Baseline in Body Weight

  Up to 104 weeks

• Parts A and B: Change From Baseline in Body Mass Index

  Up to 104 weeks

• Parts A and B: Number of Participants With Erythema, Swelling, and Induration at the Injection Site Immediately and 2 Hours After Each Debio 4326 Injection as per Investigator's Assessment

  Up to 2 hours post-dose on Day 1 in both Parts A and B

Study Arms (1)

Debio 4326

EXPERIMENTAL

Participants will receive the first injection of Debio 4326, on Day 1 in Part A followed by a second injection 52 weeks later in Part B of the study.

Drug: Debio 4326

Interventions

Debio 4326 DRUG

Administered as an intramuscular (IM) injection

Debio 4326

Eligibility Criteria

• Age: 5 Years - 8 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosis of central precocious puberty.
• Onset of development of sex characteristics (i.e., breast development in girls or testicular enlargement in boys according to the Tanner method) before the age of 8 years in girls and 9 years in boys.
• Initially, only participants aged (a) 5 to 8 years inclusive (i.e., \<9 years) are eligible. The Sponsor will determine based on the recommendation of the DMC following the interim analysis whether participants aged (b) 2 to 4 years inclusive (i.e., \<5 years) and/or (c) 9 to 10 years inclusive (i.e., \<11 years) may be recruited.
• Participant to receive at least 1 year of gonadotropin-releasing hormone agonist (GnRHa) therapy from study treatment start.
• (a) Pre-treated participants: Start of initial GnRHa therapy no later than 18 months after onset of the first signs of CPP.
• (b) Treatment-naive participants: Start of Debio 4326 treatment no later than 18 months after onset of the first signs of CPP.
• (a) Pre-treated participants: Difference between bone age (Greulich and Pyle method) and chronological age of ≥1 year based on historical values at the initiation of the GnRHa therapy.
• (b) Treatment-naive participants: Difference between bone age (Greulich and Pyle method) and chronological age of ≥1 year.
• (a) Pre-treated participants: Pubertal-type LH response (LH ≥6 IU/L) following a GnRH/GnRHa stimulation test, or random non-stimulated serum LH \>0.5 IU/L (if considered local standard of care), based on historical values prior to the initiation of GnRHa therapy.
• (b) Treatment-naive participants: Pubertal-type LH response (≥6 IU/L) 30 minutes following a GnRHa \[leuprolide acetate 20 micrograms per kilogram (μg/kg) subcutaneous injection (SC)\] stimulation test before treatment initiation.
• (a) Pre-treated participants: Clinical evidence of puberty, defined as Tanner Staging ≥2 for breast development for girls and testicular volume ≥4 milliliter (mL) (cubic centimeter \[cc\]) for boys, prior to the initiation of GnRHa therapy.
• (b) Treatment-naive participants: Clinical evidence of puberty, defined as Tanner Staging ≥2 for breast development for girls and testicular volume ≥4 mL (cc) for boys.

You may not qualify if:

• Gonadotropin-independent (peripheral) precocious puberty: gonadotropin-independent gonadal or adrenal sex steroid secretion.
• (a) Pre-treated participants: Non-progressing, isolated premature thelarche prior to the initial GnRHa therapy.
• (b) Treatment-naive participants: Non-progressing, isolated premature thelarche.
• Presence of an unstable intracranial tumor or an intracranial tumor potentially requiring neurosurgery or cerebral irradiation. Participants with hamartomas not requiring surgery are eligible.
• Any other condition or chronic illness possibly interfering with growth (e.g., renal failure, diabetes, moderate to severe scoliosis, previously treated intracranial tumor).
• Other than GnRHa therapy in pre-treated participants, any ongoing treatment with a potential effect on serum levels of gonadotropins or sex steroids, or possibly interfering with growth, opioids, central nervous system \[CNS\] stimulants).
• Prior or current therapy with medroxyprogesterone acetate, growth hormone, or Insulin-like growth factor-1 (IGF-1).
• Diagnosis of short stature, i.e., more than 2.25 standard deviations (SD) below the mean height-for-age.
• Known history of seizures, epilepsy, and/or central nervous system disorders that may have been associated with seizures or convulsions.
• Prior (within 2 months of study treatment start) or current use of medications that have been associated with seizures or convulsions.
• Use of anticoagulants (heparin or coumarin derivatives).

Sponsors & Collaborators

• Debiopharm International SA: lead

Study Sites (17)

Rady Children's Hospital - San Diego

San Diego, California, 92123, United States

Location

University of California San Francisco-Benioff Children's Hospital

San Francisco, California, 94143, United States

Location

Prisma Health Pediatric Endocrinology

Columbia, South Carolina, 29203, United States

Location

Instituto de Investigaciones Metabolicas (IDIM)

Buenos Aires, C1012AAR, Argentina

Location

Centro Medico Dra Laura Maffei Investigacion Clinica Aplicada

Buenos Aires, C1425AGC, Argentina

Location

Centro de Investigaciones Medicas Mar del Plata

Mar del Plata, B7600FYK, Argentina

Location

Clinica Mayo de Urgencias Medicas Cruz Blanca S.R.L

San Miguel de Tucumán, T4000, Argentina

Location

Hospital Da Criança de Brasília Jose Alencar

Brasília, 70684-831, Brazil

Location

Hospital Universitario Walter Cantidio

Fortaleza, 60430-270, Brazil

Location

Clínica de Endocrinologia e Metabologia Ltda

Lago Sul, 71625175, Brazil

Location

Nucleo de Pesquisa Clínica do Rio Grande do Sul-NPCRS

Porto Alegre, 90430-001, Brazil

Location

CPQuali Pesquisa Clinica

São Paulo, 01228000, Brazil

Location

Irmandade Santa Casa de São Paulo

São Paulo, REG1 01222-020, Brazil

Location

Integral Pesquisa e Ensino

Votuporanga, 15501-405, Brazil

Location

ENDOMET

Antofagasta, 1271987, Chile

Location

Hospital Clinico San Borja Arriaran (HCSBA)

Santiago, 8360160, Chile

Location

Christus Latam Hub Center of Excellence and Innovation S C

Monterrey, 64060, Mexico

Location

MeSH Terms

Conditions

Puberty, Precocious

Condition Hierarchy (Ancestors)

Gonadal Disorders; Endocrine System Diseases

Study Officials

• Study Director

  Debiopharm International SA

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 8, 2023
• First Posted: November 13, 2023
• Study Start: July 31, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): February 1, 2028
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

ME (1); AR (4); BR (7); CL (2); US (3)