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Growth Hormone Therapy for Pseudohypoparathyroidism

N/A

Recruiting

Led By Emily L Germain-Lee, MD

Research Sponsored by Connecticut Children's Medical Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

For cognitive/behavioral studies: Ages 4 - 65 yrs

For cognitive/behavioral studies: Confirmed diagnosis of Pseudohypoparathyroidism type 1A and Pseudopseudohypoparathyroidism

Timeline

Screening 3 weeks

Treatment Varies

Follow Up until achieve final height (approximately 12-15 years)

Awards & highlights

Study Summary

This trial is observing the effects of growth hormone on patients with Albright hereditary osteodystrophy, specifically those with pseudohypoparathyroidism type 1a. This form of the condition typically leads to patients being short and obese. The trial is also observing the neurocognitive and psychosocial effects of the condition in order to better understand how to manage it.

Who is the study for?

This trial is for individuals aged 0.2 to 89 years with conditions like pseudohypoparathyroidism type 1A or pseudopseudohypoparathyroidism. Growth hormone treatment participants must be over 3, pre-pubertal, and meet criteria for idiopathic short stature or SGA if they are not growth hormone deficient.Check my eligibility

What is being tested?

The study observes the natural progression of Albright hereditary osteodystrophy and tests the effects of growth hormone in certain patients. It also assesses cognitive and psychosocial functioning to identify common impairments and management strategies.See study design

What are the potential side effects?

Since this trial focuses on observation and neurocognitive testing rather than medication, it does not list specific side effects related to drug interventions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 4 and 65 years old.

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I have been diagnosed with Pseudohypoparathyroidism type 1A or Pseudopseudohypoparathyroidism.

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I have been diagnosed with Pseudohypoparathyroidism type 1A or Pseudopseudohypoparathyroidism.

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I have been diagnosed with pseudohypoparathyroidism type 1A.

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I am over 3 years old and have not started puberty yet.

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I am diagnosed with idiopathic short stature or was born small for gestational age.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ until achieve final height (approximately 12-15 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~until achieve final height (approximately 12-15 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and until achieve final height (approximately 12-15 years) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Cognitive and behavioral function in Albright hereditary osteodystrophy

Lipids

Trial Design

1Treatment groups

Experimental Treatment

Group I: AHO:neurocognitive and pyschosocialExperimental Treatment1 Intervention

Neurocognitive and psychosocial testing

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.OTHER

88 Previous Clinical Trials

24,338 Total Patients Enrolled

UConn HealthOTHER

211 Previous Clinical Trials

60,021 Total Patients Enrolled

Johns Hopkins UniversityOTHER

2,258 Previous Clinical Trials

14,819,933 Total Patients Enrolled

Media Library

Albright Osteodystrophy Research Study Groups: AHO:neurocognitive and pyschosocial

Albright Osteodystrophy Clinical Trial 2023: Neurocognitive and psychosocial testing Highlights & Side Effects. Trial Name: NCT00209235 — N/A

Neurocognitive and psychosocial testing 2023 Treatment Timeline for Medical Study. Trial Name: NCT00209235 — N/A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there opportunities available for individuals to participate in this clinical experiment?

"According to clinicaltrials.gov, this research is presently recruiting volunteers for participation. The trial was initially announced on the 1st of January 2003 and it has been recently updated on October 3rd 2022."

Answered by AI

How many participants is the clinical trial currently accommodating?

"Confirmed, the information published on clinicaltrials.gov demonstrates that this trial is actively recruiting. This research programme was initially made available on January 1st 2003 with its most recent update occurring October 3rd 2022 and requires 600 patients at one location to participate."

Answered by AI

Are persons aged 25 or more admissible to this clinical test?

"Participants for this medical study must be between 2 Months and 89 Years of age. Additionally, those under the legal adult age are eligible to enroll in 6 additional trials while individuals over 65 can participate in 3 different trial arms."

Answered by AI

What criteria must an individual meet in order to be considered qualified for this trial?

"This trial is currently accepting 600 individuals diagnosed with albright hereditary osteodystrophy who are between 2 Months and 89 years of age. Notably, for patients that do not have a growth hormone deficiency (growth hormone sufficient), they must be over three years old at the time of GH initiation and pre-pubertal in order to meet FDA requirements for idiopathic short stature or SGA indication. The criteria for admission also includes confirmed diagnoses of Pseudohypoparathyroidism type 1A/Pseudopseudohypoparathyroidism and absence of any other conditions mentioned above."

Answered by AI

Recent research and studies

British Journal of DermatologyJournal

Multiple Miliary Osteoma Cutis Is a Distinct Disease Entity: Four Case Reports and Review of the Literature

Myllylä, R.M., K.M. Haapasaari, R. Palatsi, E.L. Germain-Lee, P.M. Hägg, J. Ignatius, and J. Tuukkanen. 2011. “Multiple Miliary Osteoma Cutis Is a Distinct Disease Entity: Four Case Reports and Review of the Literature”. British Journal of Dermatology. Wiley. doi:10.1111/j.1365-2133.2010.10121.x.

PLoS ONEJournal

Heterotopic Ossifications in a Mouse Model of Albright Hereditary Osteodystrophy

Huso, David L., Sarah Edie, Michael A. Levine, William Schwindinger, Yingli Wang, Harald Jüppner, and Emily L. Germain-Lee. 2011. “Heterotopic Ossifications in a Mouse Model of Albright Hereditary Osteodystrophy”. Edited by Frank Beier. Plos ONE. Public Library of Science (PLoS). doi:10.1371/journal.pone.0021755.

Nature Reviews EndocrinologyJournal

Diagnosis and Management of Pseudohypoparathyroidism and Related Disorders: First International Consensus Statement

Mantovani, Giovanna, Murat Bastepe, David Monk, Luisa de Sanctis, Susanne Thiele, Alessia Usardi, S. Faisal Ahmed, et al.. 2018. “Diagnosis and Management of Pseudohypoparathyroidism and Related Disorders: First International Consensus Statement”. Nature Reviews Endocrinology. Springer Science and Business Media LLC. doi:10.1038/s41574-018-0042-0.

The Journal of Clinical Endocrinology & MetabolismJournal