Back to resultsEffectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS
Primary Purpose
Phelan-McDermid Syndrome, Growth Hormone Treatment
Status
Completed
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
recombinant human growth hormone
Saline
Sponsored by
About this trial
This is an interventional treatment trial for Phelan-McDermid Syndrome focused on measuring Phelan-McDermid Syndrome, Growth Hormone Treatment, cross-over, Placebo-controlled
Eligibility Criteria
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
Sites / Locations
- Affiliated Hospital of JiangNan University, Department of Pediatrics
Arms of the Study
Arm 1
Arm 2
Arm Type
Placebo Comparator
Experimental
Arm Label
Group A
Group B
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
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.
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.
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.
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
Secondary Outcome Measures
Full Information
NCT ID
NCT05105685
First Posted
October 25, 2021
Last Updated
October 7, 2022
Sponsor
Affiliated Hospital of Jiangnan University
1. Study Identification
Unique Protocol Identification Number
NCT05105685
Brief Title
Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS
Official Title
Effectiveness of Recombinant Human Growth Hormone Therapy for Children With Phelan-McDermid Syndrome: An Open-label, Cross-over, Preliminary Study
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Completed
Study Start Date
June 14, 2020 (Actual)
Primary Completion Date
May 12, 2021 (Actual)
Study Completion Date
June 8, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Affiliated Hospital of Jiangnan University
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phelan-McDermid Syndrome, Growth Hormone Treatment
Keywords
Phelan-McDermid Syndrome, Growth Hormone Treatment, cross-over, Placebo-controlled
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group A
Arm Type
Placebo Comparator
Arm Description
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.
Arm Title
Group B
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
recombinant human growth hormone
Other Intervention Name(s)
rhGH (Changchun Jinsai Pharmaceutical Co., Ltd, S20080011)
Intervention Description
RhGH treatment was started at 0.1 IU/kg once daily
Intervention Type
Drug
Intervention Name(s)
Saline
Intervention Description
Saline was started at 0.1 IU/kg once daily as the placebo
Primary Outcome Measure Information:
Title
level of serum IGF-1 and IGFBP-3
Description
All laboratory indices were completed in the morning while the participants fasted and were analysed by the laboratory of the investigators' hospital.
Time Frame
3 months
Title
Chinese version of the Gesell Development Scale (GDS)
Description
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.
Time Frame
3 months
Title
Simplified Chinese version of the Aberrant Behavior Checklist (SC-ABC)
Description
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.
Time Frame
3 months
Title
Adverse events
Description
Adverse events (AEs) were measured during the trial during via monitoring visits or phone calls using an adapted semi-structured interview every two weeks
Time Frame
3 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
YueYing Liu, Phd
Organizational Affiliation
Affiliated Hospital of Jiangnan University
Official's Role
Study Director
Facility Information:
Facility Name
Affiliated Hospital of JiangNan University, Department of Pediatrics
City
Wuxi
State/Province
Jiangsu
ZIP/Postal Code
226600
Country
China
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS
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