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The problem
Intervention: Daily growth hormone therapy (Norditropin®, somatropin)
Who it was tested with: 70 Japanese children with Noonan syndrome; 35 new patients and 35 existing patients from a prior trial.
The same group is measured before the intervention and again afterwards, with a baseline and an endline and sometimes a midline. Change is visible, but other causes cannot be ruled out.
"This post-marketing, prospective, non-interventional study (ClinicalTrials.gov NCT03435627) evaluated the long‑term safety and effectiveness of daily GH therapy in this patient population." The study measured height improvement before and after treatment in the same participants without a comparison group, making it a pre-post design that demonstrates the intervention correlates with improved height outcomes.
Non-interventional design without control group makes it difficult to attribute outcomes definitively to the intervention. Single-arm design and modest sample size limit causal inference.
Across countries and designs
2 studies · 1 country
