A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

GeneScience Pharmaceuticals (GenSci)

Status and phase: Enrolling; Phase 4

Conditions: Achondroplasia

Treatments: Drug: Recombinant human growth hormone

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT05353192

GenSci001-01

Details and patient eligibility

About

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

Enrollment: 38 estimated patients

Sex: All

Ages: 2 to 10 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

38 participants in 1 patient group

Recombinant human growth hormone

Experimental group

Description:

Recombinant human growth hormone Injection (15IU/5mg/3ml/bottle)；0.05 mg/kg/d by subcutaneous injection for 52 weeks

Treatment:

Drug: Recombinant human growth hormone