What is the mechanism of action of this treatment?

Growth hormone (GH) therapy, such as the treatment being studied in the ALTU-238 trial, involves administering recombinant human growth hormone (rhGH) to patients with GH deficiency. This therapy stimulates growth and cell reproduction by activating GH receptors, which leads to the production of insulin-like growth factor 1 (IGF-1) in the liver and other tissues. IGF-1 is essential for bone growth, muscle mass development, and metabolic regulation. For patients with GH deficiency, this treatment is crucial as it helps normalize growth in children, improves body composition, and enhances overall quality of life by addressing the physical and metabolic issues caused by the deficiency.

What side effects are associated with this type of intervention?

Common side effects of growth hormone supplementation treatments, such as those similar to ALTU-238, include joint and muscle pain, swelling due to fluid retention, and increased risk of diabetes. Other potential side effects are carpal tunnel syndrome, increased intracranial pressure, and injection site reactions. Long-term use may also lead to an increased risk of certain cancers and cardiovascular issues.

What prior FDA approvals exist?

The FDA has approved several treatments for Human Growth Hormone Deficiency (GHD), including recombinant human growth hormone (rhGH) therapies such as somatropin. These treatments are designed to replace the deficient growth hormone in children and adults with GHD, promoting growth and development. Nutropin AQ, a daily rhGH injection, is one such FDA-approved therapy. The study of ALTU-238, a weekly growth hormone treatment, aims to provide a more convenient dosing schedule compared to daily injections like Nutropin AQ, potentially improving adherence and outcomes for patients with GHD.

What other types of research exist?

Promising novel alternatives to ALTU-238 for Human Growth Hormone Deficiency patients include vosoritide, a C-type natriuretic peptide analogue, which has shown efficacy in growth modulation. Additionally, advancements in gene therapy and other biologics targeting growth hormone pathways may offer new treatment options. Patients should discuss these emerging therapies with their healthcare provider to determine the best course of action.

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Growth Hormone

Weekly Growth Hormone for Growth Hormone Deficiency in Children

Phase 2

Waitlist Available

Research Sponsored by Altus Pharmaceuticals

Eligibility Criteria Checklist

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

Must have

If on thyroid hormone replacement therapy, the dose must be stable for at least 6 weeks prior to Screening and the free thyroxine level (T4), TSH, and cortisol must be within the normal range at the Screening visit

Chronologic age at Screening of 3 to 13 years (inclusive) for boys and 3 to 12 years (inclusive) for girls

Must not have

History of severe associated pathology affecting growth, including malnutrition, malabsorption, or bone dysplasia

History of or active benign intracranial hypertension

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trial is testing ALTU-238, a medication for children who can't produce enough growth hormone. It aims to help these children grow and develop by providing the hormone they lack. The study will also check if a regular dose is effective. Somatrogon is a long-acting recombinant human growth hormone being developed as a regular treatment for children with growth hormone deficiency (GHD).

Who is the study for?

This trial is for prepubertal children with growth hormone deficiency, aged 3-13 years for boys and 3-12 years for girls. They must have a low annualized height velocity and be diagnosed with GHD by specific tests. Children who've had prior growth hormone treatments or suffer from certain diseases like diabetes, genetic syndromes affecting growth, or severe liver/kidney disease cannot participate.

What is being tested?

The study is testing the safety and effectiveness of ALTU-238 as a weekly treatment compared to daily Nutropin AQ in children who naturally produce insufficient growth hormone. It aims to determine if ALTU-238 can effectively treat their condition with less frequent dosing.

What are the potential side effects?

Possible side effects of somatropin (the active ingredient in both ALTU-238 and Nutropin AQ) include injection site reactions, headaches, muscle pain, weakness, insulin resistance and could potentially contribute to hip problems in some cases.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My thyroid medication dose has been stable for 6 weeks and my thyroid and cortisol levels are normal.

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I am a boy aged 3-13 or a girl aged 3-12.

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I have not started puberty yet.

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My growth hormone levels are lower than normal for my age and sex.

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My child's bone age is 11 years or less if they are a boy, and 10 years or less if they are a girl.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a severe condition affecting my growth, like malnutrition or bone issues.

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I have or had increased pressure in my brain not caused by a tumor.

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I haven't had brain tumor treatment in the last year.

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I have had cancer that did not affect my brain.

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I have previously been treated with growth hormones or sex steroids.

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I have a condition like Turner, Noonan, or Prader-Willi syndrome that affects growth.

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I have a history of a condition like diabetes or chronic kidney disease that could affect growth.

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I have active brain cancer confirmed by recent scans.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2017 Phase 4 trial • 82 Patients • NCT02311894

21%

Headache

15%

Vomiting

10%

Upper respiratory tract infection

Injection site bruising

Pharyngitis streptococcal

Rash

Nasopharyngitis

Pyrexia

100%

80%

60%

40%

20%

Study treatment Arm

Somatropin

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.

Trial Design

4Treatment groups

Experimental Treatment

Active Control

Group I: 3Experimental Treatment1 Intervention

ALTU-238

Group II: 2Experimental Treatment1 Intervention

ALTU-238

Group III: 1Experimental Treatment1 Intervention

ALTU-238

Group IV: 4Active Control1 Intervention

Nutropin AQ

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Somatotropin

FDA approved

0 / 13Eligibility criteria met

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 (GH) therapy, such as the treatment being studied in the ALTU-238 trial, involves administering recombinant human growth hormone (rhGH) to patients with GH deficiency. This therapy stimulates growth and cell reproduction by activating GH receptors, which leads to the production of insulin-like growth factor 1 (IGF-1) in the liver and other tissues. IGF-1 is essential for bone growth, muscle mass development, and metabolic regulation. For patients with GH deficiency, this treatment is crucial as it helps normalize growth in children, improves body composition, and enhances overall quality of life by addressing the physical and metabolic issues caused by the deficiency.