Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency

NCT04326374 | Unknown Phase 3 DMC

• 2 other identifiers: CT301-CNCTR20200399
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 1

Brief Summary

This study is conducted in China only. The purpose is to demonstrate the efficacy and safety of once weekly dosing of TransCon hGH, a long-acting growth hormone product, compare to once-daily dosing of human growth hormone (hGH) after 52 weeks of treatment in prepubertal children with growth hormone deficiency (GHD).

Conditions

Growth Hormone Deficiency; Endocrine System Diseases; Hormones; Pituitary Diseases; Pituitary Disease, Anterior

Keywords

Growth Hormone Deficiency; TransCon hGH; human growth hormone; hGH

Outcome Measures

Primary Outcomes (1)

• Annualized height velocity(AHV) at Week 52

  Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups

  52 Weeks

Secondary Outcomes (6)

• AHV and the change from baseline over 52 weeks at each visit

  52 Weeks

• Height SDS and the change from baseline over 52 weeks

  52 weeks

• Serum IGF-1 and IGFBP-3 levels, and IGF-1 SDS, IGFBP-3 SDS, and the change from baseline over 52 weeks at each visit

  52 weeks

• The percentage of participants achieving IGF-1 SDS 0 to +2.0

  52 weeks

• Incidence of Treatment-Emergent Adverse Events

  52 weeks

Study Arms (2)

TransCon hGH

EXPERIMENTAL

TransCon hGH will be self-administered or injected by parents once weekly. The dose will be adjusted based on subject's weight. The treatment will continue 52 weeks.

Drug: TransCon hGH

Daily hGH

ACTIVE COMPARATOR

Daily hGH will be self-administered or injected by parents once daily. The dose will be adjusted based on subject's weight. The treatment will continue 52 weeks.

Drug: daily hGH

Interventions

TransCon hGH DRUG

Once weekly subcutaneous injection

Also known as: ACP-011

TransCon hGH

daily hGH DRUG

Once daily subcutaneous injection

Also known as: somatropin (rDNA orgin) for injection

Daily hGH

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Prepubertal children with GHD in Tanner stage 1, aged of 3 years and below 17 years;
• Impaired HT defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS≤-2.0) according to the Chinese 2005 standard；
• Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay. Bone age (BA) at least 6 months less than the chronological age；
• Baseline IGF-1 level of at least 1.0 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1.0)；
• Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is 8 years old or above).

You may not qualify if:

• Children with a body weight below 12 kg；
• Prior exposure to recombinant hGH or IGF-1 therapy；
• Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast dye recommended) at Screening
• Children born SGA (birth weight ≤10th percentile for gestational age according to the Chinese reference)；
• Children with psychosocial dwarfism；
• Children with idiopathic short stature；
• Other causes of short stature such as coeliac disease, hypothyroidism, or rickets；
• History or presence of malignant disease; any evidence of present tumor growth;
• Subjects with diabetes mellitus；
• Closed epiphyses；
• Major medical conditions and/or presence of contraindication to hGH treatment；
• Participation in any other trial of an investigational agent within 3 months prior to Screening.

Sponsors & Collaborators

• Visen Pharmaceuticals (Shanghai) Co., Ltd.: lead
• Ascendis Pharma A/S: collaborator

Study Sites (1)

Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,

Wuhan, Hubei, 430030, China

RECRUITING

MeSH Terms

Conditions

Dwarfism, Pituitary; Endocrine System Diseases; Pituitary Diseases

Interventions

lonapegsomatropin; Human Growth Hormone; WW Domain-Containing Oxidoreductase

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

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins; Short Chain Dehydrogenase-Reductases; NAD (+) and NADP (+) Dependent Alcohol Oxidoreductases; Alcohol Oxidoreductases; Oxidoreductases; Enzymes; Enzymes and Coenzymes; Tumor Suppressor Proteins; Neoplasm Proteins; Proteins

Study Officials

• Xiaoping Luo, MD

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lisa Chen, MD

CONTACT

+86-02152999605; lisa.chen@visenpharma.com

xiangling Wang, MD

CONTACT

xiangling.wang@visenpharma.com

Study Design

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

Model Details: Eligible subjects will be randomized to 1 of the 2 treatment groups: either weekly TransCon hGH or daily hGH, in a 2:1 ratio

Study Record Dates

• First Submitted: March 26, 2020
• First Posted: March 30, 2020
• Study Start: December 30, 2019
• Primary Completion: April 1, 2022
• Study Completion: April 1, 2022
• Last Updated: March 30, 2020

Record last verified: 2020-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)