ACTIVE_NOT_RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

LIBELULA™: An Open-label, Single-arm, Multi-center, Phase 3 Study on the Efficacy, Safety, and Pharmacokinetics of Debio 4326, a Triptorelin 12-month Formulation, in Pediatric Participants With Central Precocious Puberty

Quick Facts

Study Start:2024-07-31

Study Completion:2028-02

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06129539

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:5 Years to 8 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
1. Diagnosis of central precocious puberty. 2. 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. 3. 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. 4. Participant to receive at least 1 year of gonadotropin-releasing hormone agonist (GnRHa) therapy from study treatment start. 5. (a) Pre-treated participants: Start of initial GnRHa therapy no later than 18 months after onset of the first signs of CPP. 6. (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. 7. (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. 8. (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.	1. Gonadotropin-independent (peripheral) precocious puberty: gonadotropin-independent gonadal or adrenal sex steroid secretion. 2. (a) Pre-treated participants: Non-progressing, isolated premature thelarche prior to the initial GnRHa therapy. 3. Presence of an unstable intracranial tumor or an intracranial tumor potentially requiring neurosurgery or cerebral irradiation. Participants with hamartomas not requiring surgery are eligible. 4. Any other condition or chronic illness possibly interfering with growth (e.g., renal failure, diabetes, moderate to severe scoliosis, previously treated intracranial tumor). 5. 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). 6. Prior or current therapy with medroxyprogesterone acetate, growth hormone, or Insulin-like growth factor-1 (IGF-1). 7. Diagnosis of short stature, i.e., more than 2.25 standard deviations (SD) below the mean height-for-age. 8. Known history of seizures, epilepsy, and/or central nervous system disorders that may have been associated with seizures or convulsions. 9. Prior (within 2 months of study treatment start) or current use of medications that have been associated with seizures or convulsions. 10. Use of anticoagulants (heparin or coumarin derivatives).

Inclusion Criteria

Exclusion Criteria

1. Diagnosis of central precocious puberty.
2. 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.
3. 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.
4. Participant to receive at least 1 year of gonadotropin-releasing hormone agonist (GnRHa) therapy from study treatment start.
5. (a) Pre-treated participants: Start of initial GnRHa therapy no later than 18 months after onset of the first signs of CPP.
6. (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.
7. (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.
8. (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.

1. Gonadotropin-independent (peripheral) precocious puberty: gonadotropin-independent gonadal or adrenal sex steroid secretion.
2. (a) Pre-treated participants: Non-progressing, isolated premature thelarche prior to the initial GnRHa therapy.
3. Presence of an unstable intracranial tumor or an intracranial tumor potentially requiring neurosurgery or cerebral irradiation. Participants with hamartomas not requiring surgery are eligible.
4. Any other condition or chronic illness possibly interfering with growth (e.g., renal failure, diabetes, moderate to severe scoliosis, previously treated intracranial tumor).
5. 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).
6. Prior or current therapy with medroxyprogesterone acetate, growth hormone, or Insulin-like growth factor-1 (IGF-1).
7. Diagnosis of short stature, i.e., more than 2.25 standard deviations (SD) below the mean height-for-age.
8. Known history of seizures, epilepsy, and/or central nervous system disorders that may have been associated with seizures or convulsions.
9. Prior (within 2 months of study treatment start) or current use of medications that have been associated with seizures or convulsions.
10. Use of anticoagulants (heparin or coumarin derivatives).

Contacts and Locations

Principal Investigator

Study Director

STUDY_DIRECTOR

Debiopharm International SA

Study Locations (Sites)

TMC HealthCare

Tucson, Arizona, 85712

United States

Rady Children's Hospital - San Diego

San Diego, California, 92123

United States

University of California San Francisco-Benioff Children's Hospital

San Francisco, California, 94143

United States

Wolfson's Children's Hospital

Jacksonville, Florida, 32207

United States

Nemours Children's Health

Pensacola, Florida, 32514

United States

Atlanta Diabetes Associates

Atlanta, Georgia, 30318

United States

Ann and Robert H.Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611

United States

Indiana University/Riley Hospital for Children

Indianapolis, Indiana, 46202

United States

University of Michigan

Ann Arbor, Michigan, 48109

United States

Washington University

St Louis, Missouri, 63110

United States

Icahn School of Medicine at Mount Sinai

New York, New York, 10029

United States

Children's Hospital at Montefiore

New York, New York, 10467

United States

Akron Children's Hospital

Akron, Ohio, 44224

United States

Investigational Drug Service, The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104

United States

Prisma Health Pediatric Endocrinology

Columbia, South Carolina, 29203

United States

Research Institute of Dallas

Dallas, Texas, 75231

United States

University of Texas Southwestern Medical Center

Dallas, Texas, 75235

United States

Texas Children's Hospital

Houston, Texas, 77030

United States

Virginia Commonwealth University Health System

Richmond, Virginia, 23298

United States

Collaborators and Investigators

Sponsor: Debiopharm International SA

Study Director, STUDY_DIRECTOR, Debiopharm International SA

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-07-31

Study Completion Date2028-02

Study Record Updates

Study Start Date2024-07-31

Study Completion Date2028-02

Terms related to this study

Additional Relevant MeSH Terms

Central Precocious Puberty