Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS

Growth Hormone Treatment Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05105685
• Other study ID #: 395779225
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: June 14, 2020
• Est. completion date: June 8, 2021

Study information

• Verified date: October 2022
• Source: Affiliated Hospital of Jiangnan University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: June 8, 2021
• Est. primary completion date: May 12, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 1 Year to 5 Years

Eligibility

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

Related Conditions & MeSH terms

• Chromosome Deletion
• Chromosome Disorders
• Growth Hormone Treatment
• Phelan-McDermid Syndrome
• Syndrome

Intervention

Drug:
• recombinant human growth hormone
RhGH treatment was started at 0.1 IU/kg once daily
• Saline
Saline was started at 0.1 IU/kg once daily as the placebo

Locations

Country	Name	City	State
China	Affiliated Hospital of JiangNan University, Department of Pediatrics	Wuxi	Jiangsu

Sponsors (1)

Lead Sponsor	Collaborator
Affiliated Hospital of Jiangnan University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	level of serum IGF-1 and IGFBP-3	All laboratory indices were completed in the morning while the participants fasted and were analysed by the laboratory of the investigators' hospital.	3 months
Primary	Chinese version of the Gesell Development Scale (GDS)	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.	3 months
Primary	Simplified Chinese version of the Aberrant Behavior Checklist (SC-ABC)	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.	3 months
Primary	Adverse events	Adverse events (AEs) were measured during the trial during via monitoring visits or phone calls using an adapted semi-structured interview every two weeks	3 months