A Study Extension Period of PEG-somatropin (Pegylated-somatropin) in the Treatment of Children With Idiopathic Short Stature

GeneScience Pharmaceuticals (GenSci)

Status and phase: Active, not recruiting; Phase 2

Conditions: Dwarfism

Treatments: Drug: PEG-somatropin

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03255694

GenSci 033 CT-Extension Period

Details and patient eligibility

About

After the first stage (52 weeks) of Phase II clinical trial, Pegylated recombinant human growth hormone (PEG-rhGH) injection of appropriate dose in compliance with ISS clinical treatment strategy is used to treat children with ISS (Idiopathic Short Stature). The long-term efficacy and safety of the investigational product are evaluated, which can provide more scientific and reliable medication guidance information for clinical diagnosis and treatment.

Enrollment: 360 estimated patients

Sex: All

Ages: 4 to 9 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• All subjects who have completed the first stage (52 weeks) of Phase II clinical trial (including negative controls) with completed follow-up records may be enrolled in the extension period study.
• Before the extension period study, the investigator shall fully inform the subjects and their guardians of all the information about the extension period study, including detailed follow-up procedure, treatment plan, laboratory examination items during follow-ups and possible benefits and risks. The extension period study shall only be initiated after the subjects and their guardians are well informed, and agree to cooperate and complete the treatment, follow-ups and examinations during the study, and sign the written informed consent.

Exclusion criteria

• Subjects who have taken the following medications within 2 months before entering the extension period study:

  1. Aromatase inhibitors (which include but are not limited to Lelrozol and Anastrozole), with continuous medication ≥1 month;
  2. Gonadotropin releasing hormone analogues (which include but are not limited to Triptorelin, Leuprorelin and Goserelin),, with continuous medication ≥1 month;
  3. Sex steroids (which include but are not limited to any type of estrogen, progestin and androgen) , with continuous medication ≥1 month;
  4. Protein anabolic drugs (which include but are not limited to Oxandrolone, Danazol and Strombafort), with continuous medication ≥1 month;
  5. Glucocorticoids via oral/intravenous administration for more than 1 month..

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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360 participants in 1 patient group

PEG-somatropin

Experimental group

Description:

After the first stage (52 weeks) of Phase II clinical trial, the initial medication dose of this extension period is 0.2 mg/kg weight/week of PEG-rhGH for the high dose group, low dose group and negative control group, and it is adjusted in accordance with yearly height velocity (HV) and IGF-1 SDS of each visit. The maximum dose shall not exceed 0.4 mg/kg weight/week.

Treatment:

Drug: PEG-somatropin