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

NCT03305016 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: TransCon hGH CT-302U1111-1199-8218
• Study Type: interventional
• Target: 146
• Locations: 4 countries
• Sites: 24

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

Keywords

Human Growth Hormone; hGH; GHD; rhGH; Pediatric Growth Hormone Deficiency; Long Acting Growth Hormone; Somatropin; Prodrug; Growth Failure; Growth Hormone Replacement Therapy; Sustained Release Growth Hormone; Growth Hormone Deficiency; TransCon GH

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability]

  Safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment

  26 weeks

Secondary Outcomes (4)

• Annualized Height Velocity (AHV) at 26 Weeks of Weekly Lonapegsomatropin Treatment

  26 weeks

• Number of Subjects With IGF-1 Standard Deviation Score (SDS) in the Range of 0.0 to +2.0 at 26 Weeks of Weekly Lonapegsomatropin Treatment

  26 weeks

• Change in Height Standard Deviation Scores (SDS) at 26 Weeks of Weekly Lonapegsomatropin Treatment

  Baseline and 26 weeks

• Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation

  26 weeks

Study Arms (1)

TransCon hGH

EXPERIMENTAL

Once weekly subcutaneous injection of TransCon hGH

Drug: TransCon hGH

Interventions

TransCon hGH DRUG

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

TransCon hGH

Eligibility Criteria

• Age: 6 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Investigator-determined GHD diagnosis prior to the historical initiation of daily hGH therapy.
• months to 17 years old, inclusive, at Visit 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
• 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
• Tanner stage \< 5 at Visit 1
• Open epiphyses (bone age ≤14.0 years for females or ≤16.0 years for males)
• 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

You may not qualify if:

• Weight of \< 5.5 kg or \> 80 kg at Visit 1
• Females of child-bearing potential
• History of malignant disease
• 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)
• Poorly-controlled diabetes mellitus (HbA1c \>8.0%) or diabetic complications
• Known neutralizing antibodies against hGH
• Major medical conditions, unless approved by Medical Monitor
• Pregnancy
• Presence of contraindications to hGH treatment
• Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
• Participation in any other trial of an investigational agent within 30 days prior to Visit 1
• Prior exposure to investigational hGH

Sponsors & Collaborators

• Ascendis Pharma Endocrinology Division A/S: lead

Study Sites (24)

University of Alabama

Birmingham, Alabama, 35233, United States

Location

Neufeld Medical Group Inc.

Los Angeles, California, 90048, United States

Location

Center of Excellence in Diabetes and Endocrinology

Sacramento, California, 95821, United States

Location

Rocky Mountain Pediatric Endocrinology

Centennial, Colorado, 80112, United States

Location

Nemours Children's Health System

Jacksonville, Florida, 32207, United States

Location

Orlando Health Inc.

Orlando, Florida, 32806, United States

Location

Tallahassee Memorial Hospital

Tallahassee, Florida, 32308, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Children's Minnesota

Saint Paul, Minnesota, 55102, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

NYU Winthrop Hospital

Mineola, New York, 11501, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Children's Diabetes and Endocrine Center

Portland, Oregon, 97227, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Children's Medical Center

Dallas, Texas, 75235, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

University of Virginia Children's Hospital

Charlottesville, Virginia, 22903, United States

Location

Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States

Location

Monash Children's Hospital

Clayton, Victoria, 3168, Australia

Location

Stollery Children's Hospital

Edmonton, Alberta, T6G 2B7, Canada

Location

The Liggins Institute, The University of Auckland

Grafton, Auckland, 1023, New Zealand

Location

MeSH Terms

Conditions

Dwarfism, Pituitary; Endocrine System Diseases; Pituitary Diseases; Failure to Thrive

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Aimee D Shu, MD
• Organization: Ascendis Pharma, Inc.

ADS@ascendispharma.com

+1 650 352 8389

Study Officials

• Aimee D Shu, MD

  Ascendis Pharma, Inc.

  STUDY DIRECTOR

• David B Karpf, MD

  Ascendis Pharma, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: All study participants will receive TransCon hGH

Study Record Dates

• First Submitted: October 4, 2017
• First Posted: October 9, 2017
• Study Start: November 13, 2017
• Primary Completion: March 19, 2019
• Study Completion: March 19, 2019
• Last Updated: January 4, 2022
• Results First Posted: January 4, 2022

Record last verified: 2021-12

Locations

NZ (1); CA (1); US (21); AU (1)