Efficacy, Safety, and PK of Triptorelin 6-month Formulation in Patients With Central Precocious Puberty

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Debiopharm International SA
ClinicalTrials.gov Identifier:
NCT01467882
First received: November 7, 2011
Last updated: January 15, 2014
Last verified: January 2014
  Purpose

The study will investigate the efficacy, safety and pharmacokinetics of triptorelin 22.5 mg 6-month formulation in 44 patients suffering from central precocious puberty. The null hypothesis of the study is that the proportion of patients achieving LH suppression to prepubertal levels at Month 6 is 80%.


Condition Intervention Phase
Central Precocious Puberty
Drug: Triptorelin 22.5 mg
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Non-comparative, Multicenter Study on the Efficacy, Safety, and Pharmacokinetics of Triptorelin Pamoate (Embonate) 22.5 mg 6-month Formulation in Patients Suffering From Central (Gonadotropin-dependent) Precocious Puberty

Resource links provided by NLM:


Further study details as provided by Debiopharm International SA:

Primary Outcome Measures:
  • Percentage of children with LH suppression to prepubertal levels at Month 6. [ Time Frame: Month 6 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Percentage of children with LH, FSH, estradiol/testosterone suppression to prepubertal levels and triptorelin serum levels at Months 1, 2 ,3 ,6 ,9 and 12. Incidence of adverse events, changes in growth and sexual maturation. [ Time Frame: Each visit throughout the study ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 44
Study Start Date: April 2012
Estimated Study Completion Date: November 2014
Estimated Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Triptorelin 6 months
Single arm study: 2 injections of 6-month formulation of Triptorelin
Drug: Triptorelin 22.5 mg
2 IM injections at Day 1 and Day 169
Other Name: Trelstar 22.5 mg

Detailed Description:

Two injections of the triptorelin 6-month formulation will be administered by the intramuscular route at an interval of 24 weeks. The total study duration per patients will be 12 months (48 weeks).

  Eligibility

Ages Eligible for Study:   2 Years to 9 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria:

  1. Onset of development of sex characteristics before 8 and 9 years in girls and boys, respectively (breast development in girls or testicular enlargement in boys according to the Tanner method), and candidate to receive at least 12 months of GnRH agonist therapy after study entry.
  2. Aged 2-8 years inclusive (i.e. < 9 years) for girls and 2-9 years inclusive (i.e. < 10 years) for boys at initiation of triptorelin treatment.
  3. Initiation of triptorelin treatment at the latest 18 months after onset of the first signs of precocious puberty.
  4. Difference (Δ) bone age (Greulich and Pyle method) - chronological age ≥ 1 year.
  5. Pubertal-type LH response 30 minutes following a GnRH agonist stimulation test before treatment initiation (leuprolide acetate 20 μg/kg SC) ≥ 6 IU/L.
  6. Clinical evidence of puberty, defined as Tanner Staging ≥ 2 for breast development for girls and testicular volume ≥ 4 mL (cc) for boys.
  7. Informed consent signed by one parent or both parents (as per local requirements), by the liable parent or by the legal guardian (when applicable); assent signed by the child if ≥ 7 years.

Non-inclusion criteria:

  1. Gonadotropin-independent (peripheral) precocious puberty: extra pituitary secretion of gonadotropins or gonadotropin-independent gonadal or adrenal sex steroid secretion.
  2. Non-progressing isolated premature thelarche.
  3. Presence of an unstable intracranial tumour or an intracranial tumour requiring neurosurgery or cerebral irradiation. Patients with hamartomas not requiring surgery are eligible.
  4. Evidence of renal (creatinine > 2 x ULN) or hepatic impairment (bilirubin or ASAT > 3 x ULN).
  5. Any other condition or chronic illness or treatment possibly interfering with growth or other study endpoints (e.g. chronic steroid use [except mild topical steroids], renal failure, diabetes, moderate to severe scoliosis, previously treated intracranial tumour).
  6. Prior or current therapy with a GnRH agonist, medroxyprogesterone acetate, growth hormone or insulin-like growth factor-1 (IGF 1).
  7. Major medical or psychiatric illness that could interfere with study visits.
  8. Diagnosis of short stature, i.e. > 2.25 SD below the mean height for age.
  9. Positive pregnancy test.
  10. Known hypersensibility to any of the test materials or related compounds.
  11. Use of anticoagulants (heparin and coumarin derivatives).
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01467882

Locations
United States, Arizona
Pediatric Endocrinology of Phoenix
Phoenix, Arizona, United States, 85053
United States, California
Children's National Medical Center
San Diego, California, United States
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States
United States, Florida
Arnold Palmer Pediatric Endocrinology Practice
Orlando, Florida, United States
Nancy Wright MD P.A.
Tallahasse, Florida, United States, 32308
United States, Missouri
Washington University
St. Louis, Missouri, United States
United States, New Jersey
Hackensack university medical center
Hackensack, New Jersey, United States
United States, New York
Women's & Children's Hospital of Buffalo
Buffalo, New York, United States
United States, Ohio
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
United States, Oklahoma
Lynn health Science Institute
Oklahoma City, Oklahoma, United States, 73112
United States, Washington
Swedish Pediatric Specialist
Seattle, Washington, United States
Chile
IDIMI
Santiago, Chile
Mexico
Hospital Universitario de Monterrey
Monterrey, Mexico
Sponsors and Collaborators
Debiopharm International SA
Investigators
Principal Investigator: Tala Dajani, MD Pediatric Endocrinology of Phoenix, Arizona
Principal Investigator: Barry Reiner, MD Barry J. Reiner, MD, LLC, Baltimore, Maryland
Principal Investigator: Galal Salem, MD SRCR, Inc, Bell Gardens, California
Principal Investigator: Heidi Shea, MD Endocrine Associates of Dallas, Plano, Texas
Principal Investigator: Mark Rappaport, MD Pediatric Endocrine Associates, Atlanta, Georgia
Principal Investigator: Opada Alzohaili, MD Alzohaili Medical Consultants, Dearborn, Michigan
Principal Investigator: Quentin Van Meter, MD Van Meter Pediatric Endocrinology, Peachtree City, Georgia
Principal Investigator: David Domek, MD Lynn health Science Institute, Oklahoma City
Principal Investigator: Kathleen Bethin, MD Women's & Children's Hospital of Buffalo, New York
Principal Investigator: Paul Kaplowitz, MD Children's National Medical Center, Washington
Principal Investigator: Karen Klein, MD Children's National Medical Center, San Diego, California
Principal Investigator: Diane Merritt, MD Washington University, St. Louis, Missouri
Principal Investigator: Susan Rose, MD Cincinnati Children's Hospital, Ohio
Principal Investigator: Gad Kletter, MD Swedish Pediatric Specialist, Seattle, Washington
Principal Investigator: Javier Aisenberg, MD Hackensack university medical center, New Jersey
Principal Investigator: Dennis Brenner, MD St. Barnabas Medical Center, Livingston, New Jersey
Principal Investigator: Douglas Rogers, MD Cleveland Clinic, Ohio
Principal Investigator: Lawrence Silverman, MD Goryeb Children's Hospital, Morristown, New Jersey
Principal Investigator: Peter Lee, MD Penn State Hershey Children's Hospital, Pennsylvania
Principal Investigator: Ricardo Gomez, MD Children's Hospital, New Orleans, Louisiana
Principal Investigator: Fernando Cassorla, MD IDIMI, Santiago, Chile
Principal Investigator: Joshua Yang, MD Arnold Palmer Pediatric Endocrinology Practice, Orlando, Florida
Principal Investigator: Erica Eugster, MD James Whitcomb Riley Hospital for Children, Indianapolis, Indiana
Principal Investigator: Oscar Flores, MD Hospital Universitario de Monterrey, Mexico
Principal Investigator: Nancy Wright, MD Nancy Wright MD P.A., Tallahasse, Florida
  More Information

Additional Information:
Publications:
Responsible Party: Debiopharm International SA
ClinicalTrials.gov Identifier: NCT01467882     History of Changes
Other Study ID Numbers: Debio 8206-CPP-301
Study First Received: November 7, 2011
Last Updated: January 15, 2014
Health Authority: United States: Food and Drug Administration
Chile: Instituto de Salud Publica de Chile
Mexico: Secretaria de Salud

Additional relevant MeSH terms:
Puberty, Precocious
Gonadal Disorders
Endocrine System Diseases
Triptorelin
Luteolytic Agents
Contraceptive Agents, Female
Contraceptive Agents
Reproductive Control Agents
Physiological Effects of Drugs
Pharmacologic Actions
Therapeutic Uses
Antineoplastic Agents, Hormonal
Antineoplastic Agents

ClinicalTrials.gov processed this record on April 17, 2014