Growth Hormone Therapy for Pseudohypoparathyroidism

Overseen By Alexzandrea Buscarello, B.S.

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Connecticut Children's Medical Center

Must be taking: Growth hormone

Disqualifiers: No diagnosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 12-15 years

600 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how growth hormone therapy can benefit individuals with pseudohypoparathyroidism type 1A, a condition often associated with short stature and obesity. Researchers focus on individuals who either lack sufficient growth hormone or have shown a positive response to growth hormone in past studies. The trial also examines how the condition impacts thinking and social skills to develop better management strategies. Ideal candidates for this trial have a diagnosis of pseudohypoparathyroidism type 1A, confirmed by genetic testing. Participants may experience challenges in height growth and everyday social interactions. As an unphased trial, this study provides a unique opportunity to enhance understanding and improve management strategies for this condition.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that growth hormone therapy is safe for patients with pseudohypoparathyroidism type 1A?

Research has shown that growth hormone therapy is usually safe for people. Studies have found that while some patients might experience side effects, these are generally mild and manageable. Common issues include joint pain, headaches, or mild swelling, but not everyone experiences these.

The FDA has already approved growth hormone for other uses, such as treating growth hormone deficiency and certain height issues. This approval indicates it is safe when used correctly. However, a doctor should monitor any treatment to ensure it remains safe and effective.

Why are researchers excited about this trial?

Unlike traditional treatments for pseudohypoparathyroidism, which often focus on managing calcium and phosphate levels, this new approach through neurocognitive and psychosocial testing aims to address the broader cognitive and social challenges that patients face. Researchers are excited about this because it goes beyond the physical symptoms and targets the quality of life improvements, potentially helping patients cope better with everyday activities and social interactions. This holistic focus on mental health and well-being is a significant shift from the typical biochemical management of the condition.

What evidence suggests that growth hormone therapy could be effective for pseudohypoparathyroidism?

Research has shown that growth hormone treatment can benefit individuals with pseudohypoparathyroidism type 1A, a condition associated with Albright hereditary osteodystrophy. In previous studies, patients with growth hormone deficiency responded well to this treatment. These patients often experience short stature and obesity, and growth hormone helps address these issues. Even those with normal growth hormone levels but who responded well in the past may experience similar benefits. Overall, evidence suggests that growth hormone therapy can improve growth and possibly other related symptoms for individuals with this condition.

Note: This trial will include neurocognitive and psychosocial testing as part of the study.

Who Is on the Research Team?

Emily L Germain-Lee, MD

Principal Investigator

Connecticut Children's Medical Ctr. and Univ. of Connecticut School of Medicine

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I am between 4 and 65 years old.

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

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

Exclusion Criteria

I do not have pseudohypoparathyroidism type 1A.

I do not have a diagnosis of Pseudohypoparathyroidism type 1A or Pseudopseudohypoparathyroidism.

I do not have a confirmed diagnosis of Pseudohypoparathyroidism type 1A or Pseudopseudohypoparathyroidism.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive growth hormone treatment to assess effects on height, weight, and metabolic health

12-15 years

Neurocognitive and Psychosocial Assessment

Participants undergo neurocognitive and psychosocial testing to identify specific impairments

1-2 days

1-2 visits (in-person)

Follow-up

Participants are monitored for long-term effects of growth hormone treatment and neurocognitive assessments

Ongoing

What Are the Treatments Tested in This Trial?

Interventions

• Neurocognitive and psychosocial testing

Trial Overview 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.

How Is the Trial Designed?

Group I: AHO:neurocognitive and pyschosocial Experimental Treatment 1 Intervention

Neurocognitive and psychosocial testing

Who Is Running the Clinical Trial?

Connecticut Children's Medical Center

Lead Sponsor

Trials

76

Recruited

30,000+

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

Collaborator

Trials

93

Recruited

25,200+

UConn Health

Collaborator

Trials

218

Recruited

59,100+

Johns Hopkins University

Collaborator

Trials

2,366

Recruited

15,160,000+

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborator

Trials

2,103

Recruited

2,760,000+

Published Research Related to This Trial

In a study of 40 men with acquired growth hormone deficiency (GHD), 18 months of growth hormone replacement therapy did not lead to significant improvements in cognitive function or quality of life, despite initial concerns about memory performance.

The research indicates that acquired GHD in adult men does not significantly alter cognitive abilities, and low-dose GH therapy does not provide the expected benefits in cognitive function or overall well-being.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of physiological growth hormone (GH) therapy on cognition and quality of life in patients with adult-onset GH deficiency. Baum, HB., Katznelson, L., Sherman, JC., et al.[2007]

Growth hormone deficiency (GHD) is linked to reduced cognitive performance, affecting memory, processing speed, and attention in adults, as indicated by various neuropsychological assessments.

GH replacement therapy may reverse some cognitive deficits associated with GHD, although more reliable intervention studies are needed to confirm these effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Neurocognitive function in adults with growth hormone deficiency. van Dam, PS.[2006]

In a 10-year follow-up study involving 2,348 patients with adult growth hormone deficiency (AGHD), GH replacement therapy (GHRT) showed similar clinical outcomes in both older (≥60 years) and middle-aged (35-<60 years) patients, with effective increases in IGF-I levels for both groups.

There was no significant increase in the risk of nonserious or serious adverse reactions related to GHRT in older patients compared to middle-aged patients, although serious adverse events were more frequent in the older group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term Effectiveness and Safety of GH Replacement Therapy in Adults ≥60 Years: Data From NordiNet® IOS and ANSWER. Biller, BMK., Höybye, C., Ferran, JM., et al.[2023]

Patients with pituitary insufficiency receiving conventional hormone replacement therapy may experience negative impacts on mood, behavior, and cognitive functioning due to factors like pituitary surgery and inadequate hormone replacement regimens.

Growth hormone deficiency can significantly affect psychosocial functioning and quality of life, but growth hormone therapy may improve these outcomes, suggesting a direct effect of growth hormone on brain function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Quality of life and cognitive function in patients with pituitary insufficiency. Burman, P., Deijen, JB.[2018]

Over the past 15 years, patients receiving growth hormone replacement therapy (GHR) have shown less severe growth hormone deficiency, with IGF-I standard deviation scores improving from -2.1 to -1.0 across three time intervals, indicating a shift in treatment approaches.

Patients are now starting on lower doses of growth hormone, with mean doses decreasing from 0.30 mg/day to 0.21 mg/day, which may affect treatment outcomes and highlights the need for careful consideration of changing patient characteristics in future studies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Variation of the baseline characteristics and treatment parameters over time: an analysis of 15 years of growth hormone replacement in adults in the German KIMS database. Kreitschmann-Andermahr, I., Siegel, S., Francis, F., et al.[2021]

A systematic review of 94 studies indicates that both short-term and long-term growth hormone (GH) replacement therapy in adults with GH deficiency is generally safe, showing no increased risk of diabetes mellitus or cancer recurrence.

Despite the positive findings, the review highlights limitations such as the small number of subjects and insufficient long-term data, which may affect the reliability of the conclusions drawn.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Reviewing the safety of GH replacement therapy in adults. Stochholm, K., Johannsson, G.[2015]

A 6-month double-blind, randomized placebo-controlled trial involving 18 adults with growth hormone deficiency showed that long-term treatment with recombinant human growth hormone (rhGH) significantly improved attentional performance compared to placebo.

While rhGH treatment enhanced attention after 3 and 6 months, it did not lead to improvements in verbal memory or non-verbal intelligence, indicating that its cognitive benefits may be specific to attention rather than broader cognitive functions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effect of growth hormone substitution on cognitive performance in adult patients with hypopituitarism. Oertel, H., Schneider, HJ., Stalla, GK., et al.[2013]

Growth hormone replacement therapy (GHRT) significantly improved cognitive functions and quality of life (QoL) in patients with severe traumatic brain injury (TBI) and growth hormone deficiency (GHD), based on a study of 23 treated patients compared to 27 untreated patients over one year.

The most notable cognitive improvements were seen in memory recall and specific QoL domains, particularly in patients who had lower performance levels before treatment, indicating that GHRT may be especially beneficial for those with more severe disabilities.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Growth hormone replacement therapy in patients with traumatic brain injury. Moreau, OK., Cortet-Rudelli, C., Yollin, E., et al.[2013]

Growth hormone (GH) replacement therapy over 2 years significantly improves memory function in adults with childhood-onset growth hormone deficiency (CO-GHD), particularly with supraphysiological doses that accelerate recovery.

Despite improvements in memory, GH treatment does not affect psychological well-being or perceptual-motor skills, indicating that its cognitive benefits are specific to memory enhancement rather than overall psychological health.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cognitive changes during growth hormone replacement in adult men. Deijen, JB., de Boer, H., van der Veen, EA.[2022]

In a study of 19 adult patients with traumatic brain injury (TBI), those with growth hormone deficiency (GHD) showed significant cognitive improvements after 3 months of growth hormone treatment combined with cognitive rehabilitation, outperforming control patients who received a placebo.

The results suggest that exogenous growth hormone administration plays a crucial role in enhancing cognitive recovery in GHD patients, as indicated by greater improvements in various cognitive measures compared to controls.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of growth hormone (GH) replacement and cognitive rehabilitation in patients with cognitive disorders after traumatic brain injury. Reimunde, P., Quintana, A., Castañón, B., et al.[2014]

Growth hormone (GH) deficiency (GHD) is linked to cognitive impairments, particularly in memory and attention, as shown by comparisons with matched controls.

Treatment with GH has been associated with improvements in cognitive function in individuals with GHD, indicating that GH therapy may help alleviate some cognitive deficits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cognitive function in growth hormone deficiency and growth hormone replacement. Maruff, P., Falleti, M.[2006]

Citations