Phase 3 Trial for Pediatric Growth Hormone Deficiency in China Completed Enrollment On Time Despite COVID-19 Setback

VISEN Pharmaceuticals has recently announced that enrollment has been completed for their Phase 3 clinical trial examining the safety and efficacy of TransCon hGH compared to daily rhGH in patients with pediatric growth hormone deficiency (PGHD). This study is being conducted in China. Even though it had been delayed due to COVID-19 for 4 months, the team was still able to complete enrollment by their initial target date!

PGHD

PGHD is a rare disease that occurs when the pituitary gland doesn’t produce as much growth hormone as it should. Patients may face slow growth, short stature, and metabolic abnormalities, cognitive challenges, and psychosocial challenges.

The current treatment for this condition is a daily injection of growth hormone. This helps to improve growth as well as metabolic abnormalities.

Unfortunately, this therapy is associated with low adherence due to the burden it places on patients. This is why more exploratory treatment options are being investigated.

The Study

This study is focused on patients who are pre-puberty in China. It is multicenter, open-label, and randomized. The study will last for 52 weeks.

Unfortunately, many children currently take a long time to receive a diagnosis, causing a delay in treatment which can lead to poorer outcomes over the course of their lives.

A previous trial called heiGHT demonstrated that TransCon hGH was superior to daily use of somatropin after 52 weeks. Additionally, the two treatments had similar safety results.

A global study lasting 2 years also showed positive results of TransCon. It led to continuous growth in patients and safety over the 2 year period.

The TransCon technology allows somatropin to be released predictably over time while maintaining its normal mode of action. When this trial is complete, it will serve as critical evidence of the BLA VISEN will submit for the therapy in China.

Looking Forward

Researchers are hopeful about the potential of this therapy.

It has already been studied in a Phase 3  study in the U.S. as well as Europe. The BLA for the US was submitted in June of last year. An MAA was also submitted in September of last year in the European Union.

You can read more about this therapy and Phase 3 trial here.

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