Effectiveness of Recombinant Human Growth Hormone Therapy for Children With PMS

Affiliated Hospital of Jiangnan University

Status and phase: Completed; Phase 2; Phase 1

Conditions: Growth Hormone Treatment; Phelan-McDermid Syndrome

Treatments: Drug: recombinant human growth hormone; Drug: Saline

Study type: Interventional

Funder types: Other

Identifiers

NCT05105685

395779225

Details and patient eligibility

About

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.

Full 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.

Enrollment: 6 patients

Sex: All

Ages: 1 to 5 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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6 participants in 2 patient groups, including a placebo group

Group A

Placebo Comparator group

Description:

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.

Treatment:

Drug: Saline

Group B

Experimental group

Description:

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.

Treatment:

Drug: recombinant human growth hormone