Growth Hormone Treatment in Children With Hypophosphatemic Rickets

NCT02720770 | Completed Phase 1 DMC

• 1 other identifier: RBM 03-47
• Study Type: interventional
• Target: 19
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study evaluates the effect on height of a two year treatment with growth hormone in 19 children with X linked hypophosphatemic rickets.

Conditions

X Linked Hypophosphatemic Rickets

Keywords

growth hormone

Outcome Measures

Primary Outcomes (1)

• change from baseline in height SDS (standard deviation score)

  at year one and year two from baseline

Study Arms (1)

norditropine simplex

EXPERIMENTAL

Drug: norditropine simplex

Interventions

norditropine simplex DRUG

Also known as: growth hormone

norditropine simplex

Eligibility Criteria

• Age: 3 Years - 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• clinical, biochemical and genetic diagnosis of XLH
• height SDS \< 2
• at least two years of treatment with oral phosphate and calcitriol

You may not qualify if:

• uncontrolled rickets (ALP\>600 IU)
• growth hormone deficiency
• hyperparathyroidism, nephrocalcinosis, renal insufficiency
• associated disease
• previous treatment with growth hormone

Sponsors & Collaborators

• Bicetre Hospital: lead

Related Publications (1)

• Rothenbuhler A, Esterle L, Gueorguieva I, Salles JP, Mignot B, Colle M, Linglart A. Two-year recombinant human growth hormone (rhGH) treatment is more effective in pre-pubertal compared to pubertal short children with X-linked hypophosphatemic rickets (XLHR). Growth Horm IGF Res. 2017 Oct;36:11-15. doi: 10.1016/j.ghir.2017.08.001. Epub 2017 Aug 15.

  PMID: 28822957 DERIVED

MeSH Terms

Conditions

Familial Hypophosphatemic Rickets

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Rickets, Hypophosphatemic; Rickets; Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Hypophosphatemia, Familial; Renal Tubular Transport, Inborn Errors; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Calcium Metabolism Disorders; Hypophosphatemia; Phosphorus Metabolism Disorders; Vitamin D Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: March 15, 2016
• First Posted: March 28, 2016
• Study Start: January 1, 2006
• Primary Completion: January 1, 2009
• Study Completion: January 1, 2011
• Last Updated: March 28, 2016

Record last verified: 2016-03

Data Sharing

• IPD Sharing: Will not share