CompletedPhase 3

Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

United States224 participantsStarted 2016-12

Plain-language summary

This is an open-label, randomized, multicenter, efficacy and safety study of weekly MOD-4023 compared to daily Genotropin therapy in pre-pubertal children with growth hormone deficiency.

Who can participate

Age range3 Years – 11 Years

SexALL

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Inclusion criteria

✓. Pre-pubertal children aged ≥3 years, and not yet 11 years for girls or not yet 12 years for boys with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency.
✓. Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of ≤10 ng/mL.
✓. Bone age (BA) is not older than chronological age and should be less than 10 for girls and less than 11 for boys.
✓. Without prior exposure to any r-hGH therapy (naïve patients).
✓. Impaired height and height velocity defined as:
✓. Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
✓. Normal calculated GFR per updated bedside Schwartz formula for pediatric patients.
✓. Children with multiple hormonal deficiencies must be on stable replacement therapies (no change in dose) for other hypothalamo-pituitary-organ axes for at least 3 months prior ICF signing.

Exclusion criteria

✕. Children with prior history of leukemia, lymphoma, sarcoma or any other forms of cancer.
✕. History of radiation therapy or chemotherapy.
✕. Malnourished children defined as BMI \< -2 SDS for age and sex.
✕. Children with psychosocial dwarfism.
✕. Children born small for gestational age (SGA - birth weight and/or birth length \<-2 SDS for gestational age).
✕. Presence of anti-hGH antibodies at screening.
✕. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc.
✕. T2 and T1 diabetic patients, who in the opinion of the investigator are not receiving standard of care treatment or are non-compliant with their prescribed treatment or who are in poor metabolic control.

What they're measuring

Annual Height Velocity in Main Study Phase

Timeframe: 52 weeks

Annual Height Velocity in OLE Phase

Timeframe: Up to 60 months

Trial details

NCT IDNCT02968004

SponsorOPKO Health, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2019-08

Results submitted2021-05-10

View on ClinicalTrials.gov More Pediatric Growth Hormone Deficiency trials