Clinical trial NCT05353192: A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
Official title: A Multicenter, Open-label, Single Arm Phase IV Clinical Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Achondroplasia
Keywords: Achondroplasia
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Children's Hospital of Fudan University
name: Tongji Hospital
name: Children's Hospital of Nanjing Medical University
name: Jiangxi Province Children's Hospital
name: Chengdu Women's and Children's Central Hospital
name: Shandong Provincial Hospital
name: Shengjing Hospital
name: Shanghai Children's Hospital
name: Children's Hospital of The Capital Institute of Pediatrics
name: West China Second University Hospital
First posted: 2022-04-29
Enrollment: 38
Sex: ALL
Minimum age (years): 2
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia


Eligibility criteria: Inclusion Criteria:

* 2-10 years old;
* In Tanner I stage;
* ACH, documented and confirmed by genetic testing;
* Short stature;
* Ambulatory and able to stand or walk without assistance;
* Parent(s) or guardian(s) consent；
* Had never been treated with growth hormone

Exclusion criteria:

* Short stature condition other than ACH;
* Evidence of growth plate closure (proximal tibia, distal femur);
* Had a fracture of the long bones within 6 months prior to screening;
* Planned or expected bone-related surgery;
* Chronic diseases condition that affect bone metabolism and weight;
* Severe intracranial hypertension

Primary outcomes:
measure: Change From Baseline in Annualized Height Velocity (AHV) at Week 52
timeFrame: week 52
Secondary outcomes:
measure: Change From Baseline in AHV
timeFrame: week 4, week 13, week 26, week 39
measure: Change From Baseline in Height Standard Deviation Score (Ht SDS)
timeFrame: week 4, week 13, week 26, week 39, week 52
measure: Change From Baseline in BMI Standard Deviation Score (BMI SDS)
timeFrame: week 4, week 13, week 26, week 39, week 52
measure: Change From Baseline in Sitting Height/Leg Length ratio Standard Deviation Score (SH/LL SDS)
timeFrame: week 4, week 13, week 26, week 39, week 52
measure: Change From Baseline in IGF-1 Standard Deviation Score (IGF -1 SDS)
timeFrame: week 4, week 13, week 26, week 39, week 52
measure: Change From Baseline in IGF-1/IGFBP-3 ratio
timeFrame: week 4, week 13, week 26, week 39, week 52
measure: Change From Baseline in Bone Age/Chronological Age ratio (BA/CA)
timeFrame: week 52
Other outcomes:
measure: Change From Baseline in Metabolism-related Proteins
timeFrame: week 13, week 26, week 52
Locations:
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: Feihong Luo
role: PRINCIPAL_INVESTIGATOR
affiliation: Tongji Hospital
name: Xiaoping Luo
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital of Nanjing Medical University
name: Wei Gu
role: PRINCIPAL_INVESTIGATOR
affiliation: Jiangxi Province Children's Hospital
name: Yu Yang
role: PRINCIPAL_INVESTIGATOR
affiliation: Chengdu Women's and Children's Central Hospital
name: Xinran Cheng
role: PRINCIPAL_INVESTIGATOR
affiliation: Shandong Provincial Hospital
name: Guimei Li
role: PRINCIPAL_INVESTIGATOR
affiliation: Shengjing Hospital of China Medical Hospital
name: Ying Xin
role: PRINCIPAL_INVESTIGATOR
affiliation: Shanghai Children's Hospital
name: Pin Li
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital of The Capital Institute of Pediatrics
name: Xiaobo Chen
role: PRINCIPAL_INVESTIGATOR
affiliation: West China Second University Hospital
name: Jin Wu
role: PRINCIPAL_INVESTIGATOR
References: