NCT05105685

Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS

Effectiveness of Recombinant Human Growth Hormone Therapy for Children With Phelan-McDermid Syndrome: An Open-label, Cross-over, Preliminary Study

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Affiliated Hospital of Jiangnan University · Academic / Other
• Sex: All
• Age: 1 Year – 5 Years
• Healthy volunteers: Accepted

Summary

In summary, this piot study with 6 participants shown that recombinant human growth hormone (rhGH) has a positive effect on the treatment with PMS. In addition, This study indicated that rhGH can improve PMS symptoms via increase the level of serum insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein 3 (IGFBP-3). RhGH may be low cost, more accessible, alternative treatment for PMS.

Detailed description

Compared with the placebo, rhGH treatment significantly decreased the total scores and subscale scores of GDS (P \<0.05), while the total scores and subscale scores of SC-ABC significantly decreased (P \< 0.05) following three-months rhGH treatment. The similar results were also observed in comparison with baseline. Compared with the baseline, the level of serum IGF-1 and IGFBP-3 increased significantly (P \< 0.05) following three-months rhGH treatment, while the placebo group had no significant impact on serum IGF-1 and IGFBP-3 (P \> 0.05). One participant developed skin allergy the day after the first rhGH treatment, which were resolved later.

Conditions

• Phelan-McDermid Syndrome
• Growth Hormone Treatment

Interventions

Type	Name	Description
DRUG	recombinant human growth hormone	RhGH treatment was started at 0.1 IU/kg once daily
DRUG	Saline	Saline was started at 0.1 IU/kg once daily as the placebo

Timeline

• Start date: 2020-06-14
• Primary completion: 2021-05-12
• Completion: 2021-06-08
• First posted: 2021-11-03
• Last updated: 2022-10-10

Locations

1 site across 1 country: China