Efficacy & safety study of weekly MOD-4023 in GHD - CP-4-006 - V1.0

  • Research type

    Research Study

  • Full title

    Study title: A phase 3, open-label, randomized, multicenter, 12 months , efficacy and safety study of weekly MOD-4023 compared to daily Genotropin - therapy in pre-pubertal children with growth hormone deficiency

  • IRAS ID

    209462

  • Contact name

    Director of Clinical Affairs

  • Contact email

    CP4006_OBL@opko.com

  • Sponsor organisation

    KCR CRO GmbH

  • Eudract number

    2016-003874-42

  • Clinicaltrials.gov Identifier

    79,745, IND number

  • Duration of Study in the UK

    2 years, 3 months, 0 days

  • Research summary

    This is a research study comparing the effects of a long acting weekly growth hormone (MOD4023) compared with Genotropin, a licensed daily growth hormone. The study will be conducted over a 12 month period.
    After a screening period lasting up to 8 weeks, patients meeting the eligibility criteria, will be randomly in a 1:1 ratio to MOD-4023 (investigational treatment, 0.66 mg/kg/week) or growth hormone Genotropin (reference therapy, 0.034 mg/kg/daily which is equivalent to 0.24 mg/kg/week divided
    equally to 7 injections over a week) for 12 months. To compare the efficacy and safety of weekly MOD- 4023 to daily growth hormone, Genotropin. A daily growth hormone (genotropin) will be used to compare to MOD 4023.
    MOD-4023, the investigational medication, that is similar to the natural growth hormone from the body, but is slightly changed so that its action in the body lasts longer allowing for weekly (instead of daily) subcutaneous (under the skin) injections. This is convenient for children since it would decrease the
    number of injections needed.
    MOD-4023 has already been tested in other clinical studies and to date has been well tolerated in adult healthy volunteers, and adult and children patients suffering from growth hormone deficiency.
    Girls between 3 years of age and under the age of 11 (i.e. not yet the age of 10 years and 364 days) and boys 3 years of age and under the age of 12 (i.e. not yet the age of 11 years and 364 days) with growth hormone deficiency are suitable to take part in this study. All participants need to have a screening visit
    to make sure they are eligible for this study.
    Approximately 150-180 sites in 30-40 countries worldwide will participate in this study.
    An independent and external Data and Safety Monitoring Board (DSMB) will review the key safety data approximately every 6 months or on ad hoc basis.

  • REC name

    Yorkshire & The Humber - Leeds West Research Ethics Committee

  • REC reference

    17/YH/0040

  • Date of REC Opinion

    5 Apr 2017

  • REC opinion

    Further Information Favourable Opinion