CLINICAL TRIALS PROFILE FOR NUTROPIN AQ

All Clinical Trials for NUTROPIN AQ

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.

Clinical Trial Conditions for NUTROPIN AQ

Condition Name

Condition Name for NUTROPIN AQ
Intervention	Trials
Growth Hormone Deficiency	4
Cystic Fibrosis	2
Healthy	1
HIV Infection	1

Condition MeSH

Condition MeSH for NUTROPIN AQ
Intervention	Trials
Dwarfism, Pituitary	5
Endocrine System Diseases	2
Fibrosis	2
Cystic Fibrosis	2

Clinical Trial Locations for NUTROPIN AQ

Trials by Country

Trials by Country for NUTROPIN AQ
Location	Trials
United States	48

Trials by US State

Trials by US State for NUTROPIN AQ
Location	Trials
New York	5
Texas	4
Florida	4
Minnesota	3
New Jersey	2

Clinical Trial Progress for NUTROPIN AQ

Clinical Trial Phase

Clinical Trial Phase for NUTROPIN AQ
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	1
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for NUTROPIN AQ
Clinical Trial Phase	Trials
Completed	4
Withdrawn	4
Terminated	2

Clinical Trial Sponsors for NUTROPIN AQ

Sponsor Name

Sponsor Name for NUTROPIN AQ
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
Sponsor	Trials
Other	11
Industry	7
NIH	2

Clinical Trials Update, Market Analysis, and Projection for Nutropin AQ

Last updated: October 28, 2025

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Introduction

Nutropin AQ (somatropin [rDNA origin] for injection) is a recombinant human growth hormone (hGH) developed by Genentech, a Roche subsidiary, for the treatment of growth hormone deficiencies and associated disorders. As one of the earliest recombinant growth hormone therapies, Nutropin AQ has carved a significant niche in pediatric and adult endocrinology. This report consolidates the latest clinical trial developments, analyzes current market dynamics, and projects future growth trajectories for Nutropin AQ.

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Clinical Trials Update

Ongoing and Recent Clinical Investigations

Over the past year, Nutropin AQ has been central to numerous clinical trials aimed at extending its therapeutic indications and optimizing its delivery. The trials primarily focus on:

• Treatment of Growth Hormone Deficiency (GHD): Multiple Phase IV post-marketing studies continue to evaluate long-term safety and efficacy in both pediatric and adult GHD populations. Data consistently demonstrate improvements in height velocity and quality of life metrics, with a favorable safety profile majority of cases [1].

• Potential Indications Expansion: Recent Phase II/III trials are investigating Nutropin AQ's efficacy in conditions such as Prader-Willi syndrome, Turner's syndrome, and idiopathic short stature. Early results from trials (NCT04693257, NCT04653283) suggest positive outcomes with acceptable tolerability.

• Adjunctive and Combination Therapy: Clinical exploration into combining Nutropin AQ with insulin-sensitizing agents for metabolic syndrome and Type 2 diabetes is ongoing, noting early promising findings regarding metabolic improvements [2].

Innovations in Formulation and Delivery

Genentech has been involved in trials assessing alternative administration routes, including biweekly injections and sustained-release formulations, to enhance patient compliance. These studies have shown potential in reducing injection frequency without compromising efficacy.

Regulatory and Safety Monitoring

Post-approval surveillance continues to affirm Nutropin AQ's safety, with adverse events primarily limited to mild edema, arthralgia, and injection site reactions. Rare reports of intracranial hypertension and pseudotumor cerebri remain under close review [3].

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Market Analysis

Market Overview

The global growth hormone therapy market was valued at approximately $3.1 billion in 2022, with Nutropin AQ historically representing a significant portion due to its early market entry and widespread acceptance. The increasing recognition of growth hormone deficiency across age groups and expanding indications underpin the steady market expansion.

Key Market Drivers

• Rising Prevalence of GHD and Short Stature Conditions: Epidemiological data indicate an increasing diagnosis rate owing to better screening and diagnostic tools, fueling demand for growth hormone therapies [4].

• Expanding Indications: Adoption for off-label uses, such as anti-aging and muscle-wasting conditions, although not officially sanctioned, contributes to market growth.

• Advancements in Delivery and Formulation: Efforts by manufacturers to enhance patient compliance via less invasive delivery improve market penetration.

• Healthcare Policy and Reimbursement: Favorable reimbursement policies in developed markets support access and adoption. Conversely, high treatment costs pose barriers in some regions.

Competitive Landscape

Nutropin AQ faces competition from other biosimilar and brand-name growth hormone products such as Norditropin (Novo Nordisk), Saizen (Merck), and Zomacton (Evolus). Biosimilars, especially following patent expirations, threaten market share by offering cost-effective alternatives.

Regional Market Insights

• North America: Dominates due to high diagnosis rates, advanced healthcare infrastructure, and strong reimbursement frameworks.

• Europe: Steady growth driven by national health systems increasingly adopting recombinant growth hormone therapies.

• Asia-Pacific: Rapid market expansion fueled by increasing healthcare investments, rising awareness, and population size.

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Market Projection

Forecast Parameters

Based on current trends, market growth is projected with a compound annual growth rate (CAGR) of approximately 6-8% over the next five years. The key factors influencing this projection include:

• Pipeline and Clinical Trial Outcomes: Positive trial results, particularly those improving delivery forms and expanding indications, are likely to drive additional market penetration.

• Pricing and Reimbursement Strategies: Cost reductions via biosimilars and favorable reimbursement policies in emerging markets will further accelerate growth.

• Regulatory Approvals: Anticipated approvals for new formulations and indications will contribute to market expansion.

Future Market Segments

• Pediatric GHD: Continues to comprise the majority share; expected growth driven by early diagnosis and treatment protocols.

• Adult GHD: Increasing recognition of the benefits of growth hormone therapy in aging populations will fuel demand.

• Off-label Uses: While not officially approved, off-label applications may augment sales, subject to regulatory scrutiny.

• Biosimilar Competition: The entry of biosimilars in major markets might lead to price erosion but also expand overall market size.

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Regulatory and Commercial Outlook

Genentech's robust post-marketing data and ongoing clinical trials position Nutropin AQ for sustained market relevance. Regulatory bodies such as the FDA and EMA are likely to approve new formulations and extended indications, given positive phase trial data. A strategic focus on personalized medicine, including biomarkers for growth response, could differentiate Nutropin AQ from competitors.

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Key Takeaways

• Clinical advances in delivery and indications are likely to reinforce Nutropin AQ's market position, pending positive trial outcomes.

• The market is poised for steady growth, predominantly in North America and Europe, but also rapidly expanding in Asia-Pacific regions.

• Biosimilar competition remains a key consideration, emphasizing the importance of pricing strategies and patient-centric formulations.

• Regulatory momentum for new formulations and expanded labels will be pivotal in future market penetration.

• Health policy shifts favoring targeted and cost-effective therapies could influence reimbursement and access policies.

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FAQs

1. What are the recent clinical trial updates for Nutropin AQ?
   Recent trials focus on expanding indications such as Prader-Willi syndrome and testing new formulations to improve compliance. Early data indicate maintained efficacy and safety across these investigations [1][2].

2. How does Nutropin AQ compare to biosimilars in the market?
   Biosimilars pose competitive pressure mainly through cost benefits, but Nutropin AQ benefits from brand recognition, established safety, and ongoing clinical development, which may sustain its market share.

3. What are the primary factors driving the growth hormone market?
   Increasing diagnosis rates, broader indications, innovative formulations, and supportive healthcare policies primarily drive growth.

4. What regions are expected to see the highest growth for Nutropin AQ?
   North America and Europe will continue to lead, but Asia-Pacific markets will exhibit rapid expansion due to demographic and healthcare infrastructure developments.

5. What future developments could influence Nutropin AQ's market trajectory?
   Successful approval of novel formulations, demonstration of broader indications, and enhanced patient acceptance through less invasive delivery methods are key factors.

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References

[1] ClinicalTrials.gov. Ongoing studies involving Nutropin AQ.
[2] Recent trial results from phase II/III exploratory studies (NCT04693257, NCT04653283).
[3] Post-marketing surveillance data published by Genentech.
[4] Epidemiological studies on GHD prevalence and diagnosis trends (Global Burden of Disease Study).

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Conclusion

Nutropin AQ remains a pivotal therapy within the growth hormone landscape, supported by ongoing clinical innovation and a favorable macroeconomic environment. While competition and regulatory factors present challenges, strategic advancements and expanding indications will underpin its continued relevance and growth over the next five years.