Clinical trial: Investigating Efficacy and Safety of Once-weekly NNC0195-0092 (Somapacitan) Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency (NCT02616562)

Eligibility criteria:
Inclusion Criteria:

        Cohort I:

          -  Boys: Tanner stage 1 for pubic hair and testis volume below 4 ml , age at least 2
             years and 26 weeks and below or equal to 10.0 years at screening

          -  Girls: Tanner stage 1 for breast development (no palpable glandular breast tissue) and
             pubic hair, age at least 2 years and 26 weeks and below or equal to 9.0 years at
             screening

          -  Confirmed diagnosis of GHD (growth hormone deficiency) within 12 months prior to
             screening as determined by two different GH (growth hormone) stimulation tests,
             defined as a peak GH level of below or equal to 7.0 ng/ml. For children with three or
             more pituitary hormone deficiencies only one GH stimulation test is needed

          -  No prior exposure to GH therapy and/or IGF-I (insulin-like growth factor I) treatment

          -  Height of at least 2.0 standard deviations below the mean height for chronological age
             (CA) and gender according to the standards of Centers for Disease Control and
             Prevention 2-20 years: Girls/Boys stature-for-age and weight-for-age percentiles CDC
             at screening

          -  Annualized height velocity (HV) below the 25th percentile for CA (chronological age)
             and gender or below -0.7 SD (standard deviation) score for CA and sex, according to
             the standards of Prader calculated over a time span of minimum 6 months and maximum 18
             months

        Cohort II:

          -  Below 2 years and 26 weeks and a minimum weight of 5 kg at screening.

          -  Confirmed diagnosis of GHD, the GHD diagnosis must be confirmed by investigator
             according to local practice.

          -  For GH treatment naïve subjects, no prior exposure to GH therapy and/or IGF-I
             treatment.

          -  For GH treatment naïve subjects, IGF-1 SDS below -1.0 at screening, compared to age
             and sex normalized range according to central laboratory measurements.

        Cohort III:

        Age:

          -  Girls: Above 9 years and below or equal to 17 years at screening.

          -  Boys: Above 10 years and below or equal to 17 years at screening.

          -  Confirmed diagnosis of GHD

               1. for GH treatment naïve subjects, confirmed diagnosis within 12 months prior to
                  screening as determined by two different GH stimulation tests, defined as a peak
                  GH level of equal to or below 7.0 ng/ml. For children with three or more
                  pituitary hormone deficiencies only one GH stimulation test is needed. FOR JAPAN
                  ONLY: Confirmed diagnosis of GHD within 12 months prior to screening as
                  determined by one GH stimulation tests for patients with intracranial organic
                  disease or symptomatic hypoglycaemia and two different GH stimulation test for
                  other patients, defined as a peak GH level of equal to or below 6 ng/ml by assay
                  using recombinant GH standard.

               2. for non-GH treatment naïve subjects, confirmed GHD diagnosis by investigator
                  according to local practice

          -  For GH treatment naïve subjects, no prior exposure to GH therapy and/or IGF-I
             treatment.

          -  Open epiphyses; defined as bone age below 14 years for females and bone age below 16
             years for males.

        Exclusion Criteria:

          -  Any clinically significant abnormality likely to affect growth or the ability to
             evaluate

          -  growth with standing/length measurements: Chromosomal aneuploidy and significant gene
             mutations causing medical "syndromes" with short stature, including but not limited to
             Turner syndrome, Laron syndrome, Noonan syndrome, or absence of GH receptors.
             Congenital abnormalities (causing skeletal abnormalities), including but not limited
             to Russell-Silver Syndrome, skeletal dysplasias. Significant spinal abnormalities
             including but not limited to scoliosis, kyphosis and spina bifida variants

          -  Children born small for gestational age (SGA - birth weight and/or birth length
             below-2 SD for gestational age)

          -  Concomitant administration of other treatments that may have an effect on growth (not
             applicable to non-GH treatment naïve subjects in cohort II and III), including but not
             limited to methylphenidate for treatment of attention deficit hyperactivity disorder
             (ADHD)

          -  Prior history or presence of malignancy and/or intracranial tumour
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.

Subject must be at most 17 Years

Patient description (medical language): A 9-year-old girl is brought to the office for evaluation of short stature and overweight body habitus. The patient's mother and father are 170 cm and 181 cm tall, respectively.  On physical examination, the patient's height is in the 5th percentile of her age.  Other findings include low-set ears, a high arched palate, a webbed neck, and cubitus valgus.  Chromosomal analysis reveals a 45, XO karyotype.

Patient description (patient's own words): I took my 9-year-old daughter to the doctor because my mother-in-law kept saying she seemed small and overweight. My husband and I are 170 cm and 181 cm tall, respectively. The physical health highlighted that she is in the 5th percentile of her age. The doctor said that she has low-set ears, a high-arched palate, a webbed neck, and cubitus valgus. She also did a chromosomal analysis, which revealed a 45, XO karyotype.

Does the patient match the trial criteria? 0