A Safety, Tolerability and Efficacy Study of TransCon hGH in Children With Growth Hormone Deficiency
Condition: Growth Hormone Deficiency, Pediatric; Endocrine System Diseases; Hormone Deficiency; Pituitary Diseases
MeSH terms: Dwarfism, Pituitary; Pituitary Diseases; Endocrine System Diseases
Intervention: TransCon hGH
Intervention description: Once weekly subcutaneous injection at a starting dose of 0.24 mg/kg/week
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Intervention model description: All study participants will receive TransCon hGH
Masking: None (Open Label)
Enrollment: 146.0
Sex: All
Minimum age: 6 Months
Maximum age: 17 Years
Healthy volunteers: No
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

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.
    

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