Albright Hereditary Osteodystrophy: Growth Hormone Trial and Cognitive/Behavioral Assessments

• Status: Recruiting
• Trial end date: 0000-00-00
• Target enrollment: 300
• Participant gender: Both

Summary

We, the researchers, have found that growth hormone deficiency is very common in patients with pseudohypoparathyroidism type 1a, which falls under the broader condition termed Albright hereditary osteodystrophy. Patients with pseudohypoparathyroidism type 1a typically are short and obese. Some of these patients are not short during childhood, but due to a combination of factors, they end up short as adults. We are evaluating the effect of growth hormone treatment in those patients with pseudohypoparathyroidism type 1a who are found to be growth hormone deficient. We hypothesize that growth hormone deficiency may contribute to the short stature and obesity found in this condition. We are also evaluating the effect of growth hormone on patients with pseudohypoparathyroidism type 1a who are not growth hormone deficient (i.e., growth hormone sufficient) in those who had been on study drug through R01 FD003409 or who meet the criteria of idiopathic short stature or SGA.

We are also evaluating neurocognitive and psychosocial functioning in participants with AHO in order to determine the specific impairments that are most common in the condition and to determine the best approach toward management.

Funding source -- Growth hormone study: FDA OOPD [R01 FD003409 (which has ended) and R01 FD002568 (which has ended)] Cognitive/behavior: NICHD R21 HD078864

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
• Collaborators: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); Johns Hopkins University
• Treatments: Hormones
• Last Updated: 2016-08-23

Criteria

Inclusion Criteria for GH study:

• Diagnosis of pseudohypoparathyroidism type 1a

• For the portion of the study in which growth hormone is used for participants who are not growth hormone deficient (ie., growth hormone sufficient), the patient must be over 3 years of age (ie., after 3rd birthday) AND also be pre-pubertal at the time of GH initiation. As of now, the growth hormone sufficient participants must meet the criteria of idiopathic short stature or SGA indication.

Exclusion Criteria:

• Absence of above diagnosis

Inclusion for cognitive/behavioral studies:

• Confirmed diagnosis of Pseudohypoparathyroidism type 1a and Pseudopseudohypoparathyroidism

• Ages 6 - 65 yrs

Exclusion:

• Absence of above