What is the mechanism of action of this treatment?

Growth Hormone Deficiency (GHD) is commonly treated with recombinant human growth hormone (rhGH) therapies, which work by mimicking the natural growth hormone produced by the pituitary gland. Daily growth hormone therapy, such as Genotropin, involves daily injections of rhGH to maintain consistent levels of the hormone in the body, promoting growth and development. Long-acting growth hormone treatments, like MOD-4023, are designed to be administered weekly, providing a more convenient option while still ensuring adequate hormone levels. These treatments are crucial for GHD patients as they help normalize growth, improve metabolic functions, and enhance overall quality of life by addressing the hormone deficiency.

What side effects are associated with this type of intervention?

Common side effects of growth hormone treatments like MOD-4023 (long-acting) and Genotropin (daily) include injection site reactions, headaches, and muscle or joint pain. Other potential side effects are edema (swelling due to fluid retention), increased blood sugar levels, and, rarely, development of antibodies against the growth hormone. Long-term use may also increase the risk of certain metabolic and cardiovascular issues. It is important for patients to be monitored regularly by their healthcare provider to manage and mitigate these side effects.

What prior FDA approvals exist?

The FDA has approved several treatments for Growth Hormone Deficiency (GHD), including daily growth hormone therapies like Genotropin (somatropin), which is administered daily to manage GHD in children and adults. Recently, there has been interest in long-acting growth hormone therapies, such as MOD-4023, which are designed to reduce the frequency of injections by providing sustained release of the hormone. These long-acting formulations aim to improve patient compliance and quality of life by reducing the burden of daily injections.

What other types of research exist?

Promising alternatives to MOD-4023 and Genotropin for GHD patients include vosoritide, a C-type natriuretic peptide analogue, which has shown efficacy in increasing growth velocity in children with achondroplasia. Additionally, TRF-budesonide has demonstrated potential in stabilizing eGFR and reducing proteinuria in patients with IgA nephropathy, indicating its systemic absorption and possible application in GHD. These therapies represent novel approaches that could be considered alongside traditional growth hormone treatments.

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Long-acting Growth Hormone

Long-acting hGH (MOD-4023) for Growth Hormone Deficiency

Phase 3

Waitlist Available

Research Sponsored by OPKO Health, Inc.

Eligibility Criteria Checklist

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

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up after 6 and 12 months

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Pivotal Trial

Summary

This trial compares a new growth hormone treatment (MOD-4023) to another treatment (Genotropin) in children who do not produce enough growth hormone. The goal is to see if the new treatment is as effective and safe as the other one. MOD-4023 is designed to improve compliance and reduce the inconvenience of frequent injections.

Who is the study for?

This trial is for pre-pubertal children aged ≥3 and <11 years for girls, <12 years for boys with growth hormone deficiency who haven't used growth hormone treatments before. They should have impaired height velocity and normal kidney function. Children with multiple hormonal deficiencies must be on stable replacement therapies.

What is being tested?

The study compares the safety and effectiveness of a weekly dose of MOD-4023 to daily Genotropin therapy in children with growth hormone deficiency. It's an open-label, randomized trial where participants will receive one treatment or the other.

What are the potential side effects?

Potential side effects may include reactions at the injection site, headaches, muscle pain, joint stiffness, flu-like symptoms, and development of antibodies against the medication which could reduce its effectiveness.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ after 6 and 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~after 6 and 12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and after 6 and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Annual Height Velocity

Secondary study objectives

Change in Bone Maturation (BM)

Change in Height Standard Deviation Score (SDS)

Height Velocity at 6 Months

+1 more

Other study objectives

Device

Side effects data

From 2020 Phase 3 trial • 44 Patients • NCT03874013

73%

Injection site pain

55%

Nasopharyngitis

27%

Influenza

18%

Pyrexia

18%

Bronchitis

18%

Gastroenteritis

14%

Vomiting

14%

Pharyngitis

Conjunctivitis allergic

Abdominal pain upper

Injection site erythema

Nausea

Stomatitis

Erythema infectiosum

Hand-foot-and-mouth disease

Rhinitis

Eczema

Febrile convulsion

Hypoparathyroidism

Traumatic fracture

Conjunctivitis

100%

80%

60%

40%

20%

Study treatment Arm

MOD-4023 Treatment Arm

Genotropin Treatment Arm

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: MOD-4023Experimental Treatment1 Intervention

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

Group II: GenotropinActive Control1 Intervention

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Somatrogon

FDA approved

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Growth Hormone Deficiency (GHD) is commonly treated with recombinant human growth hormone (rhGH) therapies, which work by mimicking the natural growth hormone produced by the pituitary gland. Daily growth hormone therapy, such as Genotropin, involves daily injections of rhGH to maintain consistent levels of the hormone in the body, promoting growth and development. Long-acting growth hormone treatments, like MOD-4023, are designed to be administered weekly, providing a more convenient option while still ensuring adequate hormone levels. These treatments are crucial for GHD patients as they help normalize growth, improve metabolic functions, and enhance overall quality of life by addressing the hormone deficiency.