A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency

NCT ID: NCT03305016

Last Updated: 2022-01-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 146 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-11-13
• Study Completion Date: 2019-03-19

Brief Summary

A 26 week trial of TransCon hGH, a long-acting growth hormone product, administered once-a-week. Approximately 150 children (males and females) with growth hormone deficiency (GHD) will be included. All study participants will receive TransCon hGH. This is a global trial that will be conducted in, but not limited to, the United States, Canada, Australia, and New Zealand.

Conditions

Growth Hormone Deficiency, Pediatric; Endocrine System Diseases; Hormone Deficiency; Pituitary Diseases

Study Design

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

Study Groups

TransCon hGH

Once weekly subcutaneous injection of TransCon hGH

Group Type: EXPERIMENTAL

TransCon hGH

Intervention Type: DRUG

Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week

Interventions

TransCon hGH

Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy.

2. 6 months to 17 years old, inclusive, at Visit 1

1. If 3 to 17 years old, are taking daily hGH at a dose of ≥ 0.20 mg hGH/kg/week for at least 13 weeks but no more than 130 weeks prior to Visit 1

2. If ≥ 6 months but < 3 years old, are either hGH treatment-naïve or are taking daily hGH at a dose of ≥ 0.20mg hGH/kg/week for no more than 130 weeks prior to Visit 1

3. Tanner stage < 5 at Visit 1

4. Open epiphyses (bone age ≤14.0 years for females or ≤16.0 years for males)

5. Written, signed, informed consent of the parent or legal guardian of the subject and written assent of the subject as required by the IRB/HREC/IEC

Exclusion Criteria

1. Weight of < 5.5 kg or > 80 kg at Visit 1

2. Females of child-bearing potential

3. History of malignant disease

4. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth (eg, chronic diseases or conditions such as renal insufficiency, spinal cord irradiation, hypothyroidism, active celiac disease, malnutrition or psychosocial dwarfism)

5. Poorly-controlled diabetes mellitus (HbA1c >8.0%) or diabetic complications

6. Known neutralizing antibodies against hGH

7. Major medical conditions, unless approved by Medical Monitor

8. Pregnancy

9. Presence of contraindications to hGH treatment

10. Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)

11. Participation in any other trial of an investigational agent within 30 days prior to Visit 1

12. Prior exposure to investigational hGH

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ascendis Pharma Endocrinology Division A/S

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Aimee D Shu, MD

Role: STUDY_DIRECTOR

Ascendis Pharma, Inc.

David B Karpf, MD

Role: STUDY_DIRECTOR

Ascendis Pharma, Inc.

Locations

University of Alabama

Birmingham, Alabama, United States

Neufeld Medical Group Inc.

Los Angeles, California, United States

Center of Excellence in Diabetes and Endocrinology

Sacramento, California, United States

Rocky Mountain Pediatric Endocrinology

Centennial, Colorado, United States

Nemours Children's Health System

Jacksonville, Florida, United States

Orlando Health Inc.

Orlando, Florida, United States

Tallahassee Memorial Hospital

Tallahassee, Florida, United States

University of Iowa

Iowa City, Iowa, United States

Children's Minnesota

Saint Paul, Minnesota, United States

University of Mississippi Medical Center

Jackson, Mississippi, United States

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States

NYU Winthrop Hospital

Mineola, New York, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Children's Diabetes and Endocrine Center

Portland, Oregon, United States

Oregon Health & Science University

Portland, Oregon, United States

Children's Medical Center

Dallas, Texas, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

University of Virginia Children's Hospital

Charlottesville, Virginia, United States

Children's Hospital of The King's Daughters

Norfolk, Virginia, United States

Monash Children's Hospital

Clayton, Victoria, Australia

Stollery Children's Hospital

Edmonton, Alberta, Canada

The Liggins Institute, The University of Auckland

Grafton, Auckland, New Zealand

Countries

United States; Australia; Canada; New Zealand

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

U1111-1199-8218

Identifier Type: OTHER

Identifier Source: secondary_id

TransCon hGH CT-302

Identifier Type: -

Identifier Source: org_study_id