NCT00497484

Brief Summary

We have recently demonstrated resistance to GHRH leading to GH deficiency in patients with Pseudohypoparathyroidism type Ia (Mantovani et al., J Clin Endocrinol Metab, 2003. 88: 4070-4074). The purpose of this study is to evaluate the effect of at least 1-year GH replacement in these patients. In particular, we will focus our attention on growth velocity in children affected with this disease.

Trial Health

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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 5, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 6, 2007

Completed
Last Updated

July 6, 2007

Status Verified

June 1, 2007

First QC Date

July 5, 2007

Last Update Submit

July 5, 2007

Conditions

PseudohypoparathyroidismGrowth Hormone Deficiency, Dwarfism

Outcome Measures

Primary Outcomes (1)

  • growth velocity

    One year

Secondary Outcomes (1)

  • IGF-1 levels

    one month

Interventions

recombinant human somatotropinDRUG

Eligibility Criteria

Age1 Year - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Clinical and/or genetic diagnosis of Pseudohypoparathyroidism type Ia
  • Growth hormone deficiency

You may not qualify if:

  • Known malignancies

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Endocrine Unit, Dpt. of Medical Sciences, Fondazione Policlinico IRCCS

Milan, 20122, Italy

Location

Related Publications (3)

  • Mantovani G, Maghnie M, Weber G, De Menis E, Brunelli V, Cappa M, Loli P, Beck-Peccoz P, Spada A. Growth hormone-releasing hormone resistance in pseudohypoparathyroidism type ia: new evidence for imprinting of the Gs alpha gene. J Clin Endocrinol Metab. 2003 Sep;88(9):4070-4. doi: 10.1210/jc.2002-022028.

    PMID: 12970263BACKGROUND

  • Germain-Lee EL, Groman J, Crane JL, Jan de Beur SM, Levine MA. Growth hormone deficiency in pseudohypoparathyroidism type 1a: another manifestation of multihormone resistance. J Clin Endocrinol Metab. 2003 Sep;88(9):4059-69. doi: 10.1210/jc.2003-030028.

    PMID: 12970262BACKGROUND

  • Mantovani G, Ferrante E, Giavoli C, Linglart A, Cappa M, Cisternino M, Maghnie M, Ghizzoni L, de Sanctis L, Lania AG, Beck-Peccoz P, Spada A. Recombinant human GH replacement therapy in children with pseudohypoparathyroidism type Ia: first study on the effect on growth. J Clin Endocrinol Metab. 2010 Nov;95(11):5011-7. doi: 10.1210/jc.2010-1649. Epub 2010 Aug 18.

    PMID: 20719837DERIVED

MeSH Terms

Conditions

PseudohypoparathyroidismDwarfism, Pituitary

Condition Hierarchy (Ancestors)

Bone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesMetal Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesCalcium Metabolism DisordersDwarfismBone Diseases, DevelopmentalBone Diseases, EndocrineHypopituitarismPituitary DiseasesHypothalamic DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesEndocrine System Diseases

Study Officials

  • Paolo Beck-Peccoz, MD/PhD

    Endocrine Unit, Dpt. of Medical Sciences, Fondazione Policlinico IRCCS, Milan

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

July 5, 2007

First Posted

July 6, 2007

Last Updated

July 6, 2007

Record last verified: 2007-06

Locations

IT(1)