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rhGH Therapy for Children With Phelan-McDermid Syndrome
NCT05105685Phase 2
Key facts
- Status
- Completed
- Start
- Jun 2020
- Primary completion
- May 2021
- Countries
- 1
- Sponsor
- Affiliated Hospital of Jiangnan University
- Modality
- Hormone, rProtein (Recombinant Protein), Biologic
- Drugs
- somatropin
- Conditions
- Phelan-McDermid syndrome (PMS)
01
Overview
Phase 2, randomised, open-label trial of somatropin in Phelan-McDermid syndrome (PMS) (2 arms), sponsored by Affiliated Hospital of Jiangnan University. Primary endpoints: Level of serum insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein 3 (IGFBP-3) at 3 months, Gesell Development Scale at 3 months and 2 more. Started Jun 2020; status: completed.
02
Design
2 arms- Study design
- RandomizedOpen-label
Placebo -> rhGH
Began with placebo (Saline) for 3 months, then crossed over to rhGH for 3 months.
rhGH -> Placebo
Began with rhGH for 3 months, then crossed over to placebo (Saline) for 3 months.
03
Who it enrols
Patients aged 1 to 5 years diagnosed with Phelan-McDermid Syndrome, excluding those with active or suspected tumour, intracranial hypertension, chronic kidney disease, acute proliferative or severe nonproliferative diabetic retinopathy, allergy to rhGH or severe comorbidity.
04
Endpoints
Primary
Level of serum insulin-like growth factor-1 (IGF-1) and insulin-like growth factor binding protein 3 (IGFBP-3)
Gesell Development Scale
Simplified Chinese version of the Aberrant Behavior Checklist
Adverse events
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