Effects of GH on Body Proportions and Final Height in X-Linked Hypophosphatemic Rickets

NCT00473187 | Unknown Phase 1

• 1 other identifier: UKJ-Haffner-XLHR-08-2004
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

X-linked hypophosphatemic rickets (XLH) is characterized by rickets, disproportionate short stature, impaired renal phosphate reabsorption and vitamin D metabolism. Despite oral phosphate and vitamin D treatment, most children with XLH demonstrate reduced adult height. The main objective of the study is to determine the beneficial effects of recombinant human growth hormone (rhGH) therapy on body proportions after 36 month in these patients. Secondary objective is to monitor side effects of the therapy.

Conditions

Hypophosphatemic Rickets; Growth Disorders; Somatropin Therapy

Keywords

X-linked dominant hypophosphatemic rickets; growth disorders; anthropometry; human growth hormone

Outcome Measures

Primary Outcomes (1)

• change of body proportion and final height

  within 5 years

Secondary Outcomes (1)

• side effects of therapy with rhGH

  within 5 years

Interventions

somatropin DRUG

Eligibility Criteria

• Age: 3 Years - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Tanner stages of puberty B1, G1
• body height \< 2,5 SDS
• growth velocity \< 75%
• confirmed diagnosis of XLHR
• conservative treatment for at least 1 year (phosphate, vitamin D)
• informed consent, written agreement

You may not qualify if:

• growth hormone deficiency
• hypothyreosis
• severe rickets
• severe physical deformities
• severe hyperparathyreoidism
• chronic renal failure
• complex syndrome involving failure to thrive
• chronic disease with failure to thrive
• impairment of glucose tolerance
• Tanner stages of puberty greater than B1, G1
• medical history of malignancy
• therapy with growth hormone, glucocorticoides, anabolica
• attending another clinical trial

Sponsors & Collaborators

• University of Rostock: lead

Study Sites (1)

University Childrens Hospital, Rostock

Rostock, Mecklenburg-Vorpommern, 18055, Germany

Location

MeSH Terms

Conditions

Rickets, Hypophosphatemic; Growth Disorders; Familial Hypophosphatemic Rickets

Interventions

Human Growth Hormone

Condition Hierarchy (Ancestors)

Rickets; Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Calcium Metabolism Disorders; Hypophosphatemia; Phosphorus Metabolism Disorders; Vitamin D Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders; Pathologic Processes; Pathological Conditions, Signs and Symptoms; 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

Intervention Hierarchy (Ancestors)

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

Study Officials

• Dirk Schnabel, MD

  University Childrens Hospital - Charite, Berlin

  PRINCIPAL INVESTIGATOR

• Hagen Staude

  University Childrens Hospital, Rostock

  PRINCIPAL INVESTIGATOR

• Dieter Haffner, MD

  University Childrens Hospital, Rostock

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: May 11, 2007
• First Posted: May 14, 2007
• Study Start: August 1, 2004
• Study Completion: August 1, 2009
• Last Updated: May 14, 2007

Record last verified: 2007-05

Locations

DE (1)