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

NCT ID: NCT01467882

Last Updated: 2017-07-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 44 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-04-30
• Study Completion Date: 2014-07-31

Brief Summary

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 total study duration per patient will be 12 months (48 weeks).

Conditions

Central Precocious Puberty

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Triptorelin

Triptorelin 22.5 mg, intramuscular (IM), at Day 1 and Day 169

Group Type: EXPERIMENTAL

Triptorelin

Intervention Type: DRUG

Powder and solution for solution for injection

Interventions

Triptorelin

Powder and solution for solution for injection

Intervention Type: DRUG

Other Intervention Names

Trelstar

Eligibility 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.

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).

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 9 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Debiopharm International SA

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Tala Dajani, MD

Role: PRINCIPAL_INVESTIGATOR

Pediatric Endocrinology of Phoenix, Arizona

Barry Reiner, MD

Role: PRINCIPAL_INVESTIGATOR

Barry J. Reiner, MD, LLC, Baltimore, Maryland

Galal Salem, MD

Role: PRINCIPAL_INVESTIGATOR

SRCR, Inc, Bell Gardens, California

Heidi Shea, MD

Role: PRINCIPAL_INVESTIGATOR

Endocrine Associates of Dallas, Plano, Texas

Mark Rappaport, MD

Role: PRINCIPAL_INVESTIGATOR

Pediatric Endocrine Associates, Atlanta, Georgia

Opada Alzohaili, MD

Role: PRINCIPAL_INVESTIGATOR

Alzohaili Medical Consultants, Dearborn, Michigan

Quentin Van Meter, MD

Role: PRINCIPAL_INVESTIGATOR

Van Meter Pediatric Endocrinology, Peachtree City, Georgia

David Domek, MD

Role: PRINCIPAL_INVESTIGATOR

Lynn health Science Institute, Oklahoma City

Kathleen Bethin, MD

Role: PRINCIPAL_INVESTIGATOR

Women's & Children's Hospital of Buffalo, New York

Paul Kaplowitz, MD

Role: PRINCIPAL_INVESTIGATOR

Children's National Medical Center, Washington

Karen Klein, MD

Role: PRINCIPAL_INVESTIGATOR

Children's National Medical Center, San Diego, California

Diane Merritt, MD

Role: PRINCIPAL_INVESTIGATOR

Washington University, St. Louis, Missouri

Susan Rose, MD

Role: PRINCIPAL_INVESTIGATOR

Cincinnati Children's Hospital, Ohio

Gad Kletter, MD

Role: PRINCIPAL_INVESTIGATOR

Swedish Pediatric Specialist, Seattle, Washington

Javier Aisenberg, MD

Role: PRINCIPAL_INVESTIGATOR

Hackensack university medical center, New Jersey

Dennis Brenner, MD

Role: PRINCIPAL_INVESTIGATOR

St. Barnabas Medical Center, Livingston, New Jersey

Douglas Rogers, MD

Role: PRINCIPAL_INVESTIGATOR

Cleveland Clinic, Ohio

Lawrence Silverman, MD

Role: PRINCIPAL_INVESTIGATOR

Goryeb Children's Hospital, Morristown, New Jersey

Peter Lee, MD

Role: PRINCIPAL_INVESTIGATOR

Penn State Hershey Children's Hospital, Pennsylvania

Ricardo Gomez, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital, New Orleans, Louisiana

Fernando Cassorla, MD

Role: PRINCIPAL_INVESTIGATOR

IDIMI, Santiago, Chile

Joshua Yang, MD

Role: PRINCIPAL_INVESTIGATOR

Arnold Palmer Pediatric Endocrinology Practice, Orlando, Florida

Erica Eugster, MD

Role: PRINCIPAL_INVESTIGATOR

James Whitcomb Riley Hospital for Children, Indianapolis, Indiana

Oscar Flores, MD

Role: PRINCIPAL_INVESTIGATOR

Hospital Universitario de Monterrey, Mexico

Nancy Wright, MD

Role: PRINCIPAL_INVESTIGATOR

Nancy Wright MD P.A., Tallahasse, Florida

Locations

Pediatric Endocrinology of Phoenix

Phoenix, Arizona, United States

Children's National Medical Center

San Diego, California, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Arnold Palmer Pediatric Endocrinology Practice

Orlando, Florida, United States

Nancy Wright MD P.A.

Tallahassee, Florida, United States

Washington University

St Louis, Missouri, United States

Hackensack university medical center

Hackensack, New Jersey, United States

Women's & Children's Hospital of Buffalo

Buffalo, New York, United States

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Lynn health Science Institute

Oklahoma City, Oklahoma, United States

Swedish Pediatric Specialist

Seattle, Washington, United States

IDIMI

Santiago, , Chile

Hospital Universitario de Monterrey

Monterrey, , Mexico

Countries

United States; Chile; Mexico

References

Carel JC, Eugster EA, Rogol A, Ghizzoni L, Palmert MR; ESPE-LWPES GnRH Analogs Consensus Conference Group; Antoniazzi F, Berenbaum S, Bourguignon JP, Chrousos GP, Coste J, Deal S, de Vries L, Foster C, Heger S, Holland J, Jahnukainen K, Juul A, Kaplowitz P, Lahlou N, Lee MM, Lee P, Merke DP, Neely EK, Oostdijk W, Phillip M, Rosenfield RL, Shulman D, Styne D, Tauber M, Wit JM. Consensus statement on the use of gonadotropin-releasing hormone analogs in children. Pediatrics. 2009 Apr;123(4):e752-62. doi: 10.1542/peds.2008-1783. Epub 2009 Mar 30.

Reference Type: BACKGROUND

PMID: 19332438 (View on PubMed)

Martinez-Aguayo A, Hernandez MI, Beas F, Iniguez G, Avila A, Sovino H, Bravo E, Cassorla F. Treatment of central precocious puberty with triptorelin 11.25 mg depot formulation. J Pediatr Endocrinol Metab. 2006 Aug;19(8):963-70. doi: 10.1515/jpem.2006.19.8.963.

Reference Type: BACKGROUND

PMID: 16995580 (View on PubMed)

Houk CP, Kunselman AR, Lee PA. The diagnostic value of a brief GnRH analogue stimulation test in girls with central precocious puberty: a single 30-minute post-stimulation LH sample is adequate. J Pediatr Endocrinol Metab. 2008 Dec;21(12):1113-8. doi: 10.1515/jpem.2008.21.12.1113.

Reference Type: BACKGROUND

PMID: 19189683 (View on PubMed)

Lahlou N, Carel JC, Chaussain JL, Roger M. Pharmacokinetics and pharmacodynamics of GnRH agonists: clinical implications in pediatrics. J Pediatr Endocrinol Metab. 2000 Jul;13 Suppl 1:723-37. doi: 10.1515/jpem.2000.13.s1.723.

Reference Type: BACKGROUND

PMID: 10969915 (View on PubMed)

Related Links

http://www.debiopharm.com

Study sponsor

Other Identifiers

Debio 8206-CPP-301

Identifier Type: -

Identifier Source: org_study_id