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A Study to Evaluate Leuprolide Acetate 45 mg 6-Month Formulation in Children With Central Precocious Puberty (CPP)

Phase 3
Completed
Conditions
Central Precocious Puberty (CPP)
Interventions
Registration Number
NCT03695237
Lead Sponsor
AbbVie
Brief Summary

The primary objective of the study is to assess the safety and efficacy of a leuprolide acetate (LA) 45 mg 6-month depot formulation for the treatment of CPP in children who are either naïve to treatment with a gonadotropin-releasing hormone agonist (GnRHa) or who have been previously treated with a GnRHa.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
45
Inclusion Criteria
  • Children with CPP who are naïve to treatment with a gonadotropin-releasing hormone agonist (GnRHa) (females 2 - 8 years of age, or males 2 - 9 years of age) or who have been on standard GnRHa therapy for at least 6 months (females 2 - 10 years of age, or males 2 - 11 years of age).
  • No history of clinically significant medical conditions or any other reason that the investigator determines would make the participant an unsuitable candidate to receive study drug.
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Exclusion Criteria

Not provided

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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Leuprolide Acetate (LA)Leuprolide Acetate (LA)Participants received leuprolide acetate (LA) 45 mg every 6 months administered as an intramuscular injection for up to 144 weeks.
Primary Outcome Measures
NameTimeMethod
Percentage of Participants With Suppression of Peak Gonadotropin-releasing Hormone Agonist (GnRHa)-Stimulated Luteinizing Hormone (LH) to Less Than 4 mlU/mL at Week 24Week 24 (prior to the Week 24 dose); samples for LH measurement were taken 30 and 60 minutes after the stimulation test injection.

Suppression of GnRHa-stimulated luteinizing hormone (LH) was measured using a peak GnRHa stimulation test, performed using subcutaneous injection with an aqueous formulation of leuprolide acetate at 20 µg/kg.

Peak stimulated LH was calculated by taking the maximum LH concentrations measured from blood samples taken at 30 or 60 min following the GnRHa stimulation test.

Suppression of GnRHa-stimulated luteinizing hormone is defined as peak stimulated LH less than 4 mIU/mL.

Secondary Outcome Measures
NameTimeMethod
Part 2: Percentage of Male Participants With Maintenance of Suppression of Basal Testosterone to < 30 ng/dLWeeks 72, 96, 120, and 144

Male participants with maintenance of suppression of testosterone to \< 30 ng/dL are assessed

Part 1: Percentage of Female Participants With Suppression of Basal Estradiol to < 20 pg/mL at Weeks 12, 20, 24, 44, and 48Weeks 12, 20, 24, 44, and 48

Estradiol concentrations were measured from blood samples taken at each study visit prior to stimulation testing (and prior to study drug administration at Weeks 24 and 48).

Part 1: Percentage of Male Participants With Suppression of Basal Testosterone to < 30 ng/dL at Weeks 12, 20, 24, 44, and 48Weeks 12, 20, 24, 44, and 48

Testosterone concentrations were measured from blood samples taken at each study visit prior to stimulation testing (and prior to study drug administration for Weeks 24 and 48).

Parts 1 and 2: Change From Baseline in Incremental Growth RatePart 1: Baseline and Weeks 4, 12, 20, 24, 44, and 48; Part 2: Weeks 72, 96, 120, and 144

Growth rate (height in centimeter/year) was calculated both prior to treatment in the study and during the study. For Baseline calculation a historical measurement of height at least 6 months prior to Screening and the Screening value was used.

Parts 1 and 2: Ratio of Change From Baseline in Bone Age to Change From Baseline in Chronological AgePart 1: Baseline and Weeks 24 and 48; Part 2: Weeks 72, 96, 120, and 144

Bone age was assessed from radiographs of the hand and wrist by a central imaging vendor using the BoneXpert automated system.

A ratio less than 1 indicates less advancement of bone age compared to chronological age.

Part 2: Percentage of Participants With Maintenance of Suppression of GnRHa-stimulated LH (< 4 mlU/mL)Weeks 72, 96, 120, and 144

Suppression of GnRHa-stimulated luteinizing hormone (LH) was measured using a peak GnRHa stimulation test, performed using subcutaneous injection with an aqueous formulation of leuprolide acetate at 20 µg/kg.

Peak stimulated LH was calculated by taking the maximum LH concentrations measured from blood samples taken at 30 or 60 min following the GnRHa stimulation test.

Suppression of GnRHa-stimulated luteinizing hormone is defined as peak stimulated LH less than 4 mIU/mL.

Part 1: Percentage of Participants With Suppression of Peak GnRHa-stimulated LH to Less Than 4 mlU/mL at Weeks 12, 20, 44, and 48Weeks 12, 20, 44, and 48 (prior to Week 48 dose); samples for LH measurement were taken 30 and 60 minutes after the stimulation test injection.

Suppression of GnRHa-stimulated luteinizing hormone (LH) was measured using a peak GnRHa stimulation test, performed using subcutaneous injection with an aqueous formulation of leuprolide acetate at 20 µg/kg.

Peak stimulated LH was calculated by taking the maximum LH concentrations measured from blood samples taken at 30 or 60 min following the GnRHa stimulation test.

Suppression of GnRHa-stimulated luteinizing hormone is defined as peak stimulated LH less than 4 mIU/mL.

Parts 1 and 2: Percentage of Participants With Suppression of the Physical Signs of PubertyPart 1: Weeks 24 and 48; Part 2: Weeks 72, 96, 120, and 144

Breast development in females and testicular volume or genital development in males was assessed using modified Tanner staging, on a scale from Stage 1 (prepubertal) to Stage 5 (adult characteristics).

Females: Suppression is defined as regression or no progression of breast development according to modified Tanner staging.

Males: Suppression is defined as regression or no progression in testicular volume and genital staging according to modified Tanner staging.

Part 2: Percentage of Female Participants With Maintenance of Suppression of Basal Estradiol to < 20 pg/mLWeeks 72, 96, 120, and 144

Estradiol concentrations were measured from blood samples taken at each study visit prior to stimulation testing.

Trial Locations

Locations (18)

Rady Children's Hospital San Diego /ID# 202491

🇺🇸

San Diego, California, United States

Van Meter Pediatric Endocrinology /ID# 201688

🇺🇸

Atlanta, Georgia, United States

Rocky Mountain Diabetes and Osteoporosis Center /ID# 209878

🇺🇸

Idaho Falls, Idaho, United States

Indiana University /ID# 200526

🇺🇸

Indianapolis, Indiana, United States

Pediatric Endocrine Associates /ID# 202396

🇺🇸

Boston, Massachusetts, United States

Children's Hospital Colorado /ID# 201645

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Aurora, Colorado, United States

University of Minnesota /ID# 200508

🇺🇸

Minneapolis, Minnesota, United States

Pediatric Endocrinology Associates /ID# 200629

🇺🇸

Long Beach, California, United States

Pediatric Endocrine Associates /ID# 201089

🇺🇸

Greenwood Village, Colorado, United States

Nemours Children's Health System /ID# 201331

🇺🇸

Jacksonville, Florida, United States

University of Oklahoma /ID# 200659

🇺🇸

Tulsa, Oklahoma, United States

Penn State Hershey Medical Ctr /ID# 200287

🇺🇸

Hershey, Pennsylvania, United States

Children's Hospital of Philadelphia - Main /ID# 203846

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Philadelphia, Pennsylvania, United States

Pediatric Endocrine Research Associates /ID# 200131

🇵🇷

San Juan, Puerto Rico

Cook Children's Med. Center /ID# 212937

🇺🇸

Fort Worth, Texas, United States

Multicare Institute for Research and Innovation /ID# 202188

🇺🇸

Tacoma, Washington, United States

Arnold Palmer Hospital /ID# 201624

🇺🇸

Orlando, Florida, United States

Children's Mercy Hospital/ID# 200221

🇺🇸

Kansas City, Missouri, United States

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