Phelan-McDermid SyndromePhase 1/2NCT05105685
Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS
A Phase 1/2 study testing recombinant human growth hormone and Saline in people with Phelan-McDermid Syndrome and Growth Hormone Treatment, run by Affiliated Hospital of Jiangnan University at 1 site in China.
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… Read more about this study
- Who is it for?
- Ages 1 year to 5 yearsHealthy volunteers can take part
- Conditions the study is for
- Phelan-McDermid Syndrome, Growth Hormone Treatment
- What is being tested?
- recombinant human growth hormone and Saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 14 Jun 2020to 8 Jun 2021
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 5 years
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Patients diagnosed with PMS aged from 1years to 5 years
Who cannot join
- active or suspected tumour, intracranial hypertension, chronic kidney disease, acute proliferative or severe nonproliferative diabetic retinopathy, allergy to rhGH or severe comorbidity
Trial locations and contacts
1 site in 1 country.
China1 site
Affiliated Hospital of JiangNan University, Department of Pediatrics
Wuxi, Jiangsu, 226600
About this study
Official title: Effectiveness of Recombinant Human Growth Hormone Therapy for Children With Phelan-McDermid Syndrome: An Open-label, Cross-over, Preliminary Study
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.
Read the 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.
- Conditions
- Phelan-McDermid Syndrome, Growth Hormone Treatment
- Being tested
- recombinant human growth hormone, Saline
- Keywords
- Phelan-McDermid Syndrome, Growth Hormone Treatment, cross-over, Placebo-controlled
- Medical subject headings
- Telomeric 22q13 Monosomy Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
recombinant human growth hormoneDrug
Also called rhGH (Changchun Jinsai Pharmaceutical Co., Ltd, S20080011)
RhGH treatment was started at 0.1 IU/kg once daily
SalineDrug
Saline was started at 0.1 IU/kg once daily as the placebo
| Study group | Role | What they receive |
|---|---|---|
| Group A | Placebo comparator | 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. Receives: Drug: Saline |
| Group B | Experimental | 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. Receives: Drug: recombinant human growth hormone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 3 months
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.
Measured: 3 months
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.
Measured: 3 months
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
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2021
- Study start14 Jun 2020
- Primary completion12 May 2021
- Study completion8 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Phelan-McDermid SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Affiliated Hospital of Jiangnan University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- YueYing Liu, Phd, Study director, Affiliated Hospital of Jiangnan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05105685
- Sponsor's protocol ID
- 395779225
- Status last verified
- Oct 2022
- First submitted
- 25 Oct 2021
- Last updated
- 10 Oct 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness of Recombinant Human Growth Hormone Therapy for Children With Phelan-McDermid Syndrome: An Open-label, Cross-over, Preliminary Study
- ClinicalTrials.gov ID
- NCT05105685
- Acronym / sponsor ID
- 395779225
- Conditions
- Phelan-McDermid Syndrome, Growth Hormone Treatment
- Keywords
- Phelan-McDermid Syndrome, Growth Hormone Treatment, cross-over, Placebo-controlled
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Affiliated Hospital of Jiangnan UniversityUniversity, hospital or other
- Study start
- 14 Jun 2020 (actual)
- Primary completion
- 12 May 2021 (actual)
- Study completion
- 8 Jun 2021 (actual)
- First posted
- 3 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A | Placebo comparator | Drug: SalineSubjects 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 | Experimental | Drug: recombinant human growth hormoneSubjects 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. |
Who may qualify
- Ages
- 1 year to 5 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Patients diagnosed with PMS aged from 1years to 5 years
Key exclusion criteria (1 listed)
- active or suspected tumour, intracranial hypertension, chronic kidney disease, acute proliferative or severe nonproliferative diabetic retinopathy, allergy to rhGH or severe comorbidity
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| recombinant human growth hormoneAlso: rhGH (Changchun Jinsai Pharmaceutical Co., Ltd, S20080011)RhGH treatment was started at 0.1 IU/kg once daily | Drug | Group B |
| SalineSaline was started at 0.1 IU/kg once daily as the placebo | Drug | Group A |
Primary outcome
level of serum IGF-1 and IGFBP-3
Time frame: 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)
Time frame: 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.
Simplified Chinese version of the Aberrant Behavior Checklist (SC-ABC)
Time frame: 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.
Adverse events
Time frame: 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
YueYing Liu, Phd study director
Affiliated Hospital of Jiangnan University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing recombinant human growth hormone and Saline in people with Phelan-McDermid Syndrome and Growth Hormone Treatment, run by Affiliated Hospital of Jiangnan University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
level of serum IGF-1 and IGFBP-3 (time frame: 3 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Oct 2022.