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

VISEN Pharmaceuticals

Status and phase: Unknown; Phase 3

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

Treatments: Drug: daily hGH; Drug: TransCon hGH

Study type: Interventional

Funder types: Industry

Identifiers

NCT04326374

CT301-CN

CTR20200399 (Other Identifier)

Details and patient eligibility

About

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).

Full description

Not Provided

Enrollment: 150 estimated patients

Sex: All

Ages: 3 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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).

Exclusion criteria

• 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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150 participants in 2 patient groups

TransCon hGH

Experimental group

Description:

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.

Treatment:

Drug: TransCon hGH

Daily hGH

Active Comparator group

Description:

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.

Treatment:

Drug: daily hGH