CLINICAL TRIALS PROFILE FOR NUTROPIN AQ PEN

All Clinical Trials for Nutropin Aq Pen

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00079742 ↗	A Study to Evaluate Nutropin AQ for the Treatment of Growth Restriction in Children With Cystic Fibrosis	Completed	Genentech, Inc.	Phase 2	2003-09-01	This is a Phase II, multicenter, randomized, controlled, open-label trial of the safety and efficacy of Nutropin AQ administered subcutaneously (SC) daily in prepubertal children with CF and growth restriction.
NCT00102258 ↗	Role of Nutrition and Hormones in Boys With Disordered Growth	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	2005-01-19	This study will determine whether adding more calories to the diet helps boys with growth problems grow better while being treated with Nutropin, a growth hormone that is used to help children grow taller. The Food and Drug Administration has approved Nutropin for use in children who are very short. This study will examine whether giving nutritional supplements in addition to Nutropin can help children grow better than with Nutropin alone. Boys between 7 and 10 years of age who are very short and below average in weight, but are otherwise healthy may be eligible for this study. Candidates must qualify for Nutropin treatments to boost their growth. Boys will be recruited for the study from the Nemours Children's Clinic in Jacksonville, FL, and from the National Institutes of Health in Bethesda, MD. Participants are randomly assigned to one of two treatment groups. One group is observed for 6 months and then receives a Nutropin injection every day for 12 months. The second group drinks 8 ounces of a high-calorie beverage called Pediasure every day for 6 months and then receives Nutropin plus Pediasure every day for 12 months. In addition to treatment, participants undergo the following tests and procedures at the schedule indicated: Baseline, 3, 6, 9, 12, 15 and 18 months - Clinical examination - Height measurement - Body composition assessment: Skin-fold thickness calipers are used in four places on the body to estimate body fat - Bioelectric impedance: A small amount of electrical current is used to calculate the percentage of body fat. Baseline, 6, 12, and 18 months - Blood tests - Bone age x-ray: x-ray of the left hand to measure growth potential - DEXA (dual energy x-ray absorptiometry) scan: x-ray scan to measure body fat, muscle, and bone mineral content. The subject lies on a flat table during the scan. Baseline, 6, and 12 months - Record of dietary intake: Parents are asked to write down everything the child eats and drinks for 3 days. Using this record, a dietitian calculates the daily caloric intake. - Total energy expenditure: This test determines how much energy the child uses. For the test, the child drinks water labeled with harmless isotopes (heavy oxygen and heavy hydrogen). For the next 10 days he collects urine in plastic tubes at home. At the end of the 10 days, the parents bring the urine to the clinic for analysis to determine how fast the labeled water leaves the body. This information is used to calculate how much energy the child expends each day. Participants' weight is measured at 2 and 4 weeks, and then monthly for the remainder of the 18-month study.
NCT00134420 ↗	Growth Hormone and Chromosome 18q- and Abnormal Growth	Completed	Genentech, Inc.	Phase 3	2001-02-01	We, the investigators at the University of Texas Health Science Center at San Antonio, want to learn if height and IQ (intelligence quotient) scores are improved by growth hormone (GH) treatment in children with chromosome 18 deletions and abnormal growth. Data from a previous study showed that growth hormone improved height in all children with 18q- and growth hormone deficiency. In addition, most of the study participants on growth hormone treatment showed an increase in IQ scores.
NCT00134420 ↗	Growth Hormone and Chromosome 18q- and Abnormal Growth	Completed	South Texas Veterans Health Care System	Phase 3	2001-02-01	We, the investigators at the University of Texas Health Science Center at San Antonio, want to learn if height and IQ (intelligence quotient) scores are improved by growth hormone (GH) treatment in children with chromosome 18 deletions and abnormal growth. Data from a previous study showed that growth hormone improved height in all children with 18q- and growth hormone deficiency. In addition, most of the study participants on growth hormone treatment showed an increase in IQ scores.
NCT00134420 ↗	Growth Hormone and Chromosome 18q- and Abnormal Growth	Completed	The University of Texas Health Science Center at San Antonio	Phase 3	2001-02-01	We, the investigators at the University of Texas Health Science Center at San Antonio, want to learn if height and IQ (intelligence quotient) scores are improved by growth hormone (GH) treatment in children with chromosome 18 deletions and abnormal growth. Data from a previous study showed that growth hormone improved height in all children with 18q- and growth hormone deficiency. In addition, most of the study participants on growth hormone treatment showed an increase in IQ scores.
NCT00209235 ↗	Albright Hereditary Osteodystrophy: Growth Hormone Trial and Cognitive/Behavioral Assessments	Recruiting	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2/Phase 3	2003-01-01	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
NCT00209235 ↗	Albright Hereditary Osteodystrophy: Growth Hormone Trial and Cognitive/Behavioral Assessments	Recruiting	Johns Hopkins University	Phase 2/Phase 3	2003-01-01	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

Clinical Trial Conditions for Nutropin Aq Pen

Condition Name

Condition Name for Nutropin Aq Pen
Intervention	Trials
Growth Hormone Deficiency	4
Cystic Fibrosis	2
Healthy	1
HIV Infection	1

Condition MeSH

Condition MeSH for Nutropin Aq Pen
Intervention	Trials
Dwarfism, Pituitary	5
Endocrine System Diseases	2
Fibrosis	2
Cystic Fibrosis	2

Clinical Trial Locations for Nutropin Aq Pen

Trials by Country

Trials by Country for Nutropin Aq Pen
Location	Trials
United States	48

Trials by US State

Trials by US State for Nutropin Aq Pen
Location	Trials
New York	5
Texas	4
Florida	4
Minnesota	3
New Jersey	2

Clinical Trial Progress for Nutropin Aq Pen

Clinical Trial Phase

Clinical Trial Phase for Nutropin Aq Pen
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	1
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for Nutropin Aq Pen
Clinical Trial Phase	Trials
Completed	4
Withdrawn	4
Terminated	2

Clinical Trial Sponsors for Nutropin Aq Pen

Sponsor Name

Sponsor Name for Nutropin Aq Pen
Sponsor	Trials
Genentech, Inc.	5
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	2
University of Texas Southwestern Medical Center	2

Sponsor Type

Sponsor Type for Nutropin Aq Pen
Sponsor	Trials
Other	11
Industry	7
NIH	2

Nutropin AQ Pen: Clinical Trials, Market Analysis, and Projections

Clinical Trials Overview

Indications and Patient Populations

Nutropin AQ, a somatropin formulation, is indicated for the treatment of pediatric patients with growth failure due to inadequate secretion of endogenous growth hormone (GH), growth failure associated with chronic kidney disease (CKD), and idiopathic short stature (ISS)[2][4].

Adverse Reactions and Safety Profile

Clinical trials and post-marketing surveillance have identified several adverse reactions associated with Nutropin AQ. Common adverse reactions include hypothyroidism, impaired glucose tolerance, headache, hypertonia, arthralgia, myalgia, peripheral oedema, and injection site reactions. More serious adverse reactions include neoplasms (both malignant and benign) and intracranial hypertension. Intracranial hypertension is typically associated with symptoms such as papilloedema, visual changes, headache, nausea, and vomiting, usually occurring within eight weeks of therapy initiation[1][4].

Specific Populations

• Pediatric Patients with Growth Hormone Deficiency: Common adverse reactions in this group include central nervous system neoplasms and other indication-specific reactions.
• Children with Turner Syndrome: Menorrhagia is a common adverse reaction.
• Children with Chronic Renal Insufficiency: These patients are more likely to develop intracranial hypertension, renal failure, peritonitis, and osteonecrosis[1].

Market Analysis

Current Market Situation

As of 2023, Nutropin AQ is facing a significant supply shortage in the United States due to a sustained increase in demand and fluctuations in supply across several growth hormone manufacturers. This shortage is expected to persist for several months, with potential return to supply stability in the first half of 2024[3].

Impact on Patients and Healthcare Providers

The shortage has critical implications for patients and healthcare providers, leading to delays in refilling prescriptions and necessitating discussions about alternative treatment options. Genentech recommends against starting treatment-naive patients on Nutropin AQ during this period and suggests considering alternative growth hormone formulations or switching to different formulations based on clinical judgment and market availability[3].

Market Demand and Competition

The demand for growth hormone therapies is high, driven by the need to treat various growth disorders in pediatric patients. The shortage of Nutropin AQ has highlighted the competitive landscape of growth hormone treatments, where other manufacturers may benefit from the temporary unavailability of this product. However, the loyalty of patients and healthcare providers to established brands like Nutropin AQ remains a significant factor in the market dynamics[3].

Projections and Future Outlook

Supply Chain Recovery

Genentech anticipates that the supply issues will be resolved by the first half of 2024, which should stabilize the market and restore availability of Nutropin AQ. Until then, the company is working to ensure that existing patients can continue their treatment with minimal disruption, while also exploring alternative solutions for new patients[3].

Market Growth

The growth hormone market is expected to continue growing due to increasing awareness and diagnosis of growth disorders, as well as advancements in treatment options. Despite the current shortage, Nutropin AQ is likely to remain a significant player in this market due to its established reputation and the trust of healthcare providers and patients.

Regulatory and Clinical Developments

Future clinical trials and regulatory approvals will be crucial in expanding the indications for Nutropin AQ and improving its safety profile. Continuous monitoring of post-marketing data will help in identifying and mitigating any long-term adverse effects, further solidifying the drug's position in the market.

Key Takeaways

• Clinical Trials: Nutropin AQ has been extensively studied in various pediatric populations, highlighting its efficacy but also its potential for serious adverse reactions.
• Market Analysis: The current shortage underscores the high demand for growth hormone therapies and the competitive nature of the market.
• Projections: The market is expected to stabilize once the supply issues are resolved, with Nutropin AQ likely to remain a key player due to its established reputation and ongoing clinical and regulatory developments.

FAQs

What are the common indications for Nutropin AQ?

Nutropin AQ is indicated for the treatment of pediatric patients with growth failure due to inadequate secretion of endogenous growth hormone, growth failure associated with chronic kidney disease, and idiopathic short stature[2].

What are the most serious adverse reactions associated with Nutropin AQ?

The most serious adverse reactions include neoplasms (both malignant and benign) and intracranial hypertension. Other significant reactions include impaired glucose tolerance, diabetes mellitus, and injection site reactions[1][4].

Why is there a current shortage of Nutropin AQ?

The shortage is due to a significant and sustained increase in demand combined with fluctuations in supply across several growth hormone manufacturers. This is expected to persist until the first half of 2024[3].

What alternatives can patients consider during the Nutropin AQ shortage?

Patients and healthcare providers may consider alternative growth hormone formulations or switch to different formulations based on clinical judgment and market availability[3].

How does the shortage impact patients and healthcare providers?

The shortage leads to delays in refilling prescriptions and necessitates discussions about alternative treatment options, causing stress and disruption to patient care[3].

Sources

1. European Medicines Agency - NutropinAq, INN-Somatropin - European Medicines Agency
2. Drugs.com - Nutropin AQ: Package Insert / Prescribing Information
3. Genentech - Nutropin Shortage
4. Genentech - Master Nutropin AQ USPI_NDA 20522_CLEAN
5. Genentech - Nutropin AQ NuSpin 5 Instructions for Use (IFU)