Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS

Sponsor
Affiliated Hospital of Jiangnan University (Other)
Overall Status
Completed
CT.gov ID
NCT05105685
Collaborator
(none)
6
1
2
11.8
0.5

Study Details

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.

Condition or Disease Intervention/Treatment Phase
Phase 1/Phase 2

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 Design

Study Type:
Interventional
Actual Enrollment :
6 participants
Allocation:
Randomized
Intervention Model:
Crossover Assignment
Masking:
Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose:
Treatment
Official Title:
Effectiveness of Recombinant Human Growth Hormone Therapy for Children With Phelan-McDermid Syndrome (PMS) : A Randomized, Cross-over, Placebo-controlled, Preliminary Study
Actual Study Start Date :
Jun 14, 2020
Actual Primary Completion Date :
May 12, 2021
Actual Study Completion Date :
Jun 8, 2021

Arms and Interventions

Arm Intervention/Treatment
Placebo Comparator: Group A

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.

Drug: Saline
Saline was started at 0.1 IU/kg once daily as the placebo

Experimental: Group B

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.

Drug: recombinant human growth hormone
RhGH treatment was started at 0.1 IU/kg once daily
Other Names:
  • rhGH (Changchun Jinsai Pharmaceutical Co., Ltd, S20080011)
  • Outcome Measures

    Primary Outcome Measures

    1. 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.

    2. 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.

    3. 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.

    4. 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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    1 Year to 5 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    Yes
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Affiliated Hospital of JiangNan University, Department of Pediatrics Wuxi Jiangsu China 226600

    Sponsors and Collaborators

    • Affiliated Hospital of Jiangnan University

    Investigators

    • Study Director: YueYing Liu, Phd, Affiliated Hospital of Jiangnan University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Affiliated Hospital of Jiangnan University
    ClinicalTrials.gov Identifier:
    NCT05105685
    Other Study ID Numbers:
    • 395779225
    First Posted:
    Nov 3, 2021
    Last Update Posted:
    Nov 10, 2021
    Last Verified:
    Oct 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Affiliated Hospital of Jiangnan University
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Nov 10, 2021