A Real World Study of the Effect of Early PEG-rhGH Therapy on Cognitive Development of SGA Infants
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age6-2
SponsorTongji Hospital
About this trial
Cognitive impairment is independently related to low birth weight, low birth length and small head circumference. SGA children who have not experienced height and / or head circumference catch-up have the worst cognitive function. The serum IGF-1 level of short SGA children is significantly lower than that of catch-up SGA children. This may be due to the defect of GH-IGF-1 axis, resulting in some hGH / IGF-1 deficiency.
GH treatment can induce catch-up growth of head circumference, especially for those with small birth head circumference, growth hormone can help to improve IQ, behavior and self cognition of children with SGA.
Two years after birth is the most critical period for children's physical, neurological, cognitive and emotional development. This study evaluated the effect of growth hormone treatment on the improvement of cognitive function and growth and development of symmetrical SGA children who did not show catch-up growth from 6 months to 2 years old.
This is an innovative study. The minimum age of previous similar studies is 19 months. The starting age of this study is 6 months, and the results are to improve the cognitive development of SGA infants. This is the first of its kind. Although the safety of growth hormone in SGA infants younger than 2 years old has not been reported, it is based on a number of studies on the application of growth hormone in infants, such as PWS and GHD, It can be expected that there will be no short-term and long-term adverse reactions.
The study was conducted in 17 hospitals led by Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of science and technology
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Provide informed consent signed and dated by the subject's legal guardian;
The subjects met the clinical diagnosis of small for gestational age infants.
The age ranged from 6 months to 2 years old (including 6 months and 2 years old);
Height and head circumference are lower than the reference value - 2sd (including-2sd), whose weight is lower than the 10th percentile of the reference value of normal children of the same age and sex;
Disqualifiers
Patients with abnormal liver and kidney function (ALT > 2 times the upper limit of normal value, Cr > the upper limit of normal value);
Severe familial dwarfism (father height < 155cm or mother height < 145cm);
Definite neurological defects and / or severe neurodevelopmental retardation (the total development quotient calculated according to Griffiths mental development scale is less than 70), definite syndrome affecting cognitive development; Severe perinatal complications (such as severe asphyxia, sepsis, necrotizing enterocolitis, respiratory distress syndrome with long-term sequelae);
Genetic metabolic diseases (such as congenital hypothyroidism, phenylketonuria, methylmalonic acidemia);
Trial design
Design model
Parallel
Treatments tested in this trial
PEG-rhGH
Drugthe subjects were given PEG-rhGH injection 0.2 mg / kg / week (initial dose), once a week, subcutaneously before going to bed for 104 weeks. Each follow-up, the researchers adjusted the dosage according to the IGF-1 results of the center and other individual conditions.
Treatment groups
138 Participants
are divided into 2 treatment groupsGroup A: GH treatment groupExperimental treatment 1 intervention
Group B: Control groupNo intervention 0 interventions
Trial outcomes
Primary outcomes
1
total development quotient (GQ)
The changes of total development quotient (GQ) of young SGA children were calculated according to Griffiths mental development scale before and after treatment.（The Griffiths Scales,The normal range is from the 16th percentile to the 84th percentile of children of the same age. 100 is the mean. The higher the score, the better）
Time frame
104 weeks2
head circumference SDS
Changes of head circumference SDS in young SGA children before and after treatment
Time frame
104 weeksSecondary outcomes
1
Motor domain development quotient
Changes of motor domain development quotient (AQ) calculated according to Griffiths mental development scale before and after treatment.（The Griffiths Scales,The normal range is from the 16th percentile to the 84th percentile of children of the same age. 100 is the mean. The higher the score, the better）
Time frame
104 weeks2
Personal and social domain development quotient
Changes of personal and social domain development quotient (BQ) calculated according to Griffiths mental development scale before and after treatment.（The Griffiths Scales,The normal range is from the 16th percentile to the 84th percentile of children of the same age. 100 is the mean. The higher the score, the better）
Time frame
104 weeks3
Hearing and language domain development quotient
Changes of hearing and language domain development quotient (CQ) calculated according to Griffiths mental development scale before and after treatment.（The Griffiths Scales,The normal range is from the 16th percentile to the 84th percentile of children of the same age. 100 is the mean. The higher the score, the better）
Time frame
104 weeks4
Hand eye coordination domain development quotient
Changes of hand eye coordination domain development quotient (DQ) calculated according to Griffiths mental development scale before and after treatment.（The Griffiths Scales,The normal range is from the 16th percentile to the 84th percentile of children of the same age. 100 is the mean. The higher the score, the better）
Time frame
104 weeksSponsors and contacts
Click on the lead sponsor to view all of their trials.
Tongji Hospital
Lead sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborator
Source ClinicalTrials.gov