A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (REAL 8)

J
John Fuqua, MD

Primary Investigator

Enrolling By Invitation
2-10 years
All
Phase 3
399 participants needed
2 Locations

Brief description of study

The study compares two medicines for treatment of children born small and who stay small, or with Turner Syndrome, Noonan Syndrome, or idiopathic short stature. The purpose of the studyhow well treatment with somapacitan works compared to treatment with Norditropin®.w medicine, and Norditropin® is a medicine doctors can already prescribeuntries. The study will last for about 3 years. The participants will either getweek for 3 years or Norditropin® once a day for 1 year followed byweek for 2 years. Which treatment the participants get is decided byhance.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: SGA, Turner Syndrome, Noonan Syndrome, ISS, Riley
  • Age: Between 2 Years - 10 Years
  • Gender: All

Inclusion criteria:
  1. Informed consent of parent or legally acceptable representative of participant andhild assent, as age appropriate must be obtained before any study-related activities.udy-related activities are any procedures that are carried out as part of the study,uding activities to determine suitability for the study.
  2. No prior exposure to growth promoting therapy, including but not limited to growthhormone, IGF-I and ghrelin analogues.
    Applicable to children with SGA:
  3. Born small for gestational age (birth length below -2 SDS OR birth weight below -2 SDSR both) (according to national standards).
  4. Prepubertal children:
    1. Boys:
      • Age above or equal to 2 years and 26 weeks and below 11.0 years atg.
      • Testis volume below 4 mL
    2. Girls:
      • Age above or equal to 2 years and 26 weeks and below 10.0 years atg.
      • Tanner stage 1 for breast development: No palpable glandular breast tissue)
  5. Impaired height defined as at least 2.5 standard deviations below the mean height for
    chronological age and sex at screening according to the standards of Centers forDisease Control and Prevention.
  6. Impaired height velocity defined as annualized height velocity below the 50thhronological age and sex according to the standards of Praderulated over a time span of minimum 6 months and maximum 18 months prior tog.
  7. Body Mass Index below the 95th percentile according to Centers for Disease Control andPrevention, Body Mass Index-for-age growth charts.
    Applicable to girls with TS:
  8. Confirmed diagnosis of TS by 30-cell (or more) lymphocyte chromosomal analysis.*
  9. Prepubertal girls:
    • Age above or equal to 2 years and 26 weeks and below 10.0 years at screening.
    • Tanner stage 1 for breast development: No palpable glandular breast tissue)
  10. Impaired height defined as at least 2.0 standard deviations below the mean height for
    chronological age and sex at screening according to the standards of Centers forDisease Control and Prevention.
  11. Historical height measured 6-18 months prior to screening.
  12. Thyroid hormone replacement therapy should be adequate and stable for at least 90 daysdomization, if applicable.
    Applicable to children with NS:
  13. Clinical diagnosis of NS according to van der Burgt score list
  14. Prepubertal children:
    1. Boys:
      • Age above or equal to 2 years and 26 weeks and below 11.0 years atg.
      • Testis volume below 4 mL
    2. Girls:
      • Age above or equal to 2 years and 26 weeks and below 10.0 years atg.
      • Tanner stage 1 for breast development: No palpable glandular breast tissue)
  15. Impaired height defined as at least 2.0 standard deviations below the mean height for
    chronological age and sex at screening according to the standards of Centers forDisease Control and Prevention.
  16. Historical height measured 6-18 months prior to screening.
  17. Thyroid hormone replacement therapy should be adequate and stable for at least 90 daysdomization, if applicable.
    Applicable to children with ISS:
  18. Prepubertal children:
    1. Boys:
      • Age above or equal to 2 years and 26 weeks and below 11.0 years atg.
      • Testis volume below 4 mL
    2. Girls:
      • Age above or equal to 2 years and 26 weeks and below 10.0 years atg.
      • Tanner stage 1 for breast development: No palpable glandular breast tissue)
  19. Bone age:
    1. Boys:
      • Bone age below or equal to 12 years.
      • Bone age not delayed or advanced more than 2 years compared to chronologicalge.
    2. Girls:
      • Bone age below or equal to 11 years.
      • Bone age not delayed or advanced more than 2 years compared to chronologicalge.
  20. Impaired height defined as at least 2.5 standard deviations below the mean height for
    chronological age and sex at screening according to the standards of Centers forDisease Control and Prevention.
  21. Historical height measured 6-18 months prior to screening.
  22. One normal GH secretion (GH peak above 7 ng/mL) during GH stimulation test performedwithin 18 months prior to screening or if such a test is not available for childrenwith ISS, a test should be performed as part of the screening assessments and theult must be available prior to randomization.
    • If a 30-cell count is not available for patients with TS, a test should be done,d results must be available prior to randomization.
Exclusion criteria:
  1. Known or suspected hypersensitivity to study intervention(s) or related products.
  2. Previous randomization into same sub-study in this study.
  3. Receipt of any investigational medicinal product within 3 months before screening orher clinical study at the time of randomization.
  4. Children with suspected or confirmed growth hormone deficiency according to local.
  5. laboratory of
    1. fasting plasma glucose above or equal to 126 mg/dL (7.0 mmol/L) or
    2. HbA1c above or equal to 6.5%.
  6. Current inflammatory diseases requiring systemic corticosteroid treatment for longer
    than 2 consecutive weeks within the last 3 months prior to screening.
  7. Children requiring inhaled glucocorticoid therapy at a dose greater than 400 µg/day ofhaled budesonide or equivalent (i.e., 250 µg/day for fluticasone propionate) forger than 4 consecutive weeks within the last 12 months prior to screening.
  8. Concomitant administration of other treatments that may have an effect on growth,.g., but not limited to methylphenidate for treatment of attention deficithyperactivity disorder (ADHD).
  9. Diagnosis of attention deficit hyperactivity disorder (ADHD).
  10. History or known presence of malignancy including intracranial tumours.
  11. History or known presence of active Hepatitis B or Hepatitis C (exceptions to thisusion criterion is the presence of antibodies due to vaccination against HepatitisB).
  12. Any disorder, which in the investigator's opinion, might jeopardize participant'sy or compliance with the protocol.
  13. The participant or the parent/legally acceptable representative is likely to beudy conduct, as judged by the investigator.
  14. Current treatment with sex hormones or aromatase inhibitors.
  15. Any known or suspected clinically significant abnormality likely to affect growth orhe ability to evaluate growth with standing height measurements, such as, but notd to:
    1. Known family history of skeletal dysplasia.
    2. Significant spinal abnormalities including but not limited to scoliosis, kyphosisd spina bifida variants.
    3. Any other disorder/condition that can cause short stature such as, but notd to, psychosocial deprivation, nutritional disorders, chronic systemicd chronic renal disease.
Applicable to children with SGA:
  1. TS (including mosaicism).
  2. NS.
  3. Hormonal deficiencies.
  4. Children who are small due to malnutrition defined as -2 standard deviations accordingdards. 0¬-5 years: weight for height on World Health Organization MulticentreGrowth Reference Study 2006. Above 5 years: World Health Organization 2007 Body Massdex.
  5. Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes'with short stature, including but not limited to Laron syndrome, Prader-Williyndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal SHOX gene analysis orbsence of GH receptors.
Applicable to children with TS:
  1. NS.
  2. Mosaicism below 10%.
  3. TS with Y-chromosome mosaicism where gonadectomy has not been performed.
  4. NYHA class II or above or requiring medication for any heart condition.
  5. Coeliac disease where participant is not stable on gluten free diet for the previous12 months prior to screening.
Applicable to children with NS:
  1. TS (including mosaicism).
  2. Noonan-related disorders: Noonan syndrome with multiple lentigines (formerly calledLEOPARD' syndrome), Noonan syndrome with loose anagen hair, cardiofaciocutaneousyndrome (CFC), Costello syndrome, neurofibromatosis type 1 (NF1) and Legius syndrome.ular genetic panel testing results must be available prior to randomisation toude these.
  3. Coeliac disease where participant is not stable on gluten free diet for the previous12 months prior to screening.
Applicable to children with ISS:
  1. TS (including mosaicism).
  2. NS.
  3. Hormonal deficiencies.
  4. Born small for gestational age (defined as birth length below -2 SDS OR birth weightbelow -2 SDS OR both) (according to national standards).
  5. Known chromosomal aneuploidy or significant gene mutations causing medical 'syndromes'with short stature, including but not limited to Laron syndrome, Prader-Williyndrome, Russell-Silver Syndrome, skeletal dysplasias, abnormal SHOX gene analysis orbsence of GH receptors.

Updated on 13 Sep 2024. Study ID: NN8640-4467, PENDO-NOVONORDISK-REAL-8, 15746
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