Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS

Phase 1

Completed

• Conditions: Phelan-McDermid Syndrome; Growth Hormone Treatment
• Interventions: Drug: Saline; Drug: recombinant human growth hormone

• Registration Number: NCT05105685
• Lead Sponsor: Affiliated Hospital of Jiangnan University

Brief 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.

Study Timeline

• Study Start: 2020-06-14
• Primary Completion: 2021-05-12
• Study Completion: 2021-06-08
• Study First Submitted: 2021-10-25
• Study First Submitted QC: 2021-10-25
• Study First Posted: 2021-11-03
• Status Verified: 2022-10
• Last Update Submitted: 2022-10-07
• Last Update Posted: 2022-10-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

• Patients diagnosed with PMS aged from 1years to 5 years

Exclusion Criteria

• active or suspected tumour, intracranial hypertension, chronic kidney disease, acute proliferative or severe nonproliferative diabetic retinopathy, allergy to rhGH or severe comorbidity

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Group A	Saline	Subjects were randomly divided into two different groups. Group A began with placebo while group B with rhGH. Randomisation of the treatment order (i.e., starting with rhGH or placebo) was done through a permuted four-block designed by the physician, who was the only one not blinded for treatment allocation.
Group B	recombinant human growth hormone	Subjects were randomly divided into two different groups. Group A began with placebo while group B with rhGH. Randomisation of the treatment order (i.e., starting with rhGH or placebo) was done through a permuted four-block designed by the physician, who was the only one not blinded for treatment allocation.

Primary Outcome Measures

Name	Time	Method
Simplified Chinese version of the Aberrant Behavior Checklist (SC-ABC)	3 months	Different from its English version that created by Krug in 1980 , SC-ABC was based on the study of Krug in 2009, in which proved ABC can be used in 14 months children, then it was translated into Simplified Chinese by the researchers of Peking University Sixth Hospital. To date, the SC-ABC scale has been verified and widely used for more than 10 years to assess the changes of symptoms in Chinese children aged from 14 months to 14 years old with behavioral problems.; This checklist includes 57 items and five subscales: sensory behavior, social relating, body and object use, language and communication skills, and social and adaptive skills. Each item was scored from 0 to 3,with higher scores indicate more severe symptoms.
level of serum IGF-1 and IGFBP-3	3 months	All laboratory indices were completed in the morning while the participants fasted and were analysed by the laboratory of the investigators' hospital.
Chinese version of the Gesell Development Scale (GDS)	3 months	To measure neuropsychological development. The development quotient (DQ) in the GDS was used to quantify neurodevelopment, which can indicate the level of neurodevelopment and is interpreted as follows: DQ=86 as normal, DQ 76 to 85 as marginally delayed, DQ at 55-75 as slightly delayed, DQ at 40 to 54 as moderately delayed, and DQ⩽39 as severely delayed.
Adverse events	3 months	Adverse events (AEs) were measured during the trial during via monitoring visits or phone calls using an adapted semi-structured interview every two weeks

Trial Locations

Locations (1)

Affiliated Hospital of JiangNan University, Department of Pediatrics; 🇨🇳 Wuxi, Jiangsu, China