Growth Hormone for Autism Spectrum Disorder

Overseen By Alexander Kolevzon

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Icahn School of Medicine at Mount Sinai

Disqualifiers: Neoplasia, Hypertension, Hepatic, Renal, others

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if growth hormone (Somatropin) can improve symptoms in children with Phelan-McDermid syndrome and idiopathic autism. Researchers will assess its impact on social skills, language, and repetitive behaviors. They will compare the hormone's effects to a placebo using a crossover design, where participants switch treatments halfway through. Children ages 2 to 12 with autism or PMS, who are stable on their current treatments, might be eligible to participate. The goal is to gather evidence on the feasibility and potential benefits of this treatment. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to early evidence of its potential.

Will I have to stop taking my current medications?

The trial requires that participants be on stable medication regimens for at least three months before joining, as long as the medications are safe to use with growth hormone. So, you may not need to stop your current medications if they are considered safe.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that growth hormone treatment is usually safe for children. One study on autism found that growth hormone improved social skills without causing major side effects. Another study on a genetic condition linked to autism also confirmed its safety. Most children did not experience serious side effects, and the treatment was generally well-received.

These results are encouraging, but this trial remains in its early stages. Early trials often focus on safety and determining the right dose. By participating, you help researchers understand how people handle the treatment. If you decide to join, the study team will closely monitor for any side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

Researchers are excited about using growth hormone as a potential treatment for Autism Spectrum Disorder (ASD) because it offers a novel approach compared to existing options. Unlike standard treatments, which often focus on managing symptoms with behavioral therapy or medications like antipsychotics, growth hormone targets the biological aspect of ASD by potentially influencing brain development and function. This treatment could open up new pathways for improving core symptoms of autism in ways current therapies haven’t addressed. By exploring the role of growth hormone, scientists hope to uncover benefits that could lead to more effective, biologically-based interventions for those with ASD.

What evidence suggests that growth hormone might be an effective treatment for autism?

Research has shown that growth hormone, one of the treatments in this trial, may improve social skills in children with autism. One study found that it significantly helped with social challenges in patients with a genetic condition linked to autism. Early results also indicate positive changes in brain activity related to sensory information processing. These findings suggest that growth hormone might assist with socializing, language, and repetitive behaviors in children with autism. The encouraging results from these studies support further research on using growth hormone as a treatment for autism and related conditions. Participants in this trial will receive growth hormone in one phase and saline in another, allowing for a comparison of effects. ^{1 2 6 7 8}

Who Is on the Research Team?

Alexander Kolevzon

Principal Investigator

Icahn School of Medicine at Mount Sinai

Are You a Good Fit for This Trial?

This trial is for children aged 2-12 with Phelan-McDermid syndrome or idiopathic autism. They must have open bone growth plates, be on stable treatments for three months, and not used growth hormone before. Kids with visual issues, severe health risks, allergies to growth hormone components, closed bone plates, or serious organ problems can't join.

Inclusion Criteria

I have been diagnosed with Autism Spectrum Disorder without known genetic causes.

My medication and mental health treatment have been stable for 3 months.

Open epiphyses on bone age x ray

Exclusion Criteria

My kidneys do not work well.

My growth plates have closed.

I have an enlarged heart or heart valve problems.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Phase 1

Participants receive growth hormone or placebo as a once daily subcutaneous injection for 12 weeks

12 weeks

Regular monitoring visits for anthropometric measures, laboratory parameters, and behavioral evaluations

Wash-out

A four week wash-out period between treatment phases

4 weeks

Treatment Phase 2

Participants crossover to receive the alternate treatment (growth hormone or placebo) for another 12 weeks

12 weeks

Regular monitoring visits for anthropometric measures, laboratory parameters, and behavioral evaluations

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Growth Hormone
• Saline

Trial Overview

The study tests if growth hormone can help kids with Phelan-McDermid syndrome and idiopathic autism improve social skills, language, and reduce repetitive behaviors. It's a double-blind test where half get the real medicine and half get saline (a placebo), without knowing which one they receive.

How Is the Trial Designed?

Group I: Growth Hormone then Saline Experimental Treatment 1 Intervention

12 weeks in each treatment phase (rhGH then placebo) and a four week wash-out period between phases.

Group II: Placebo (saline) then Growth Hormone Placebo Group 1 Intervention

12 weeks in each treatment phase (placebo then rhGH) and a four week wash-out period between phases.

Growth Hormone is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Somatropin for:

• Growth hormone deficiency
• Turner syndrome
• Prader-Willi syndrome
• Chronic kidney disease
• Short stature due to Noonan syndrome
• Short stature due to Turner syndrome
• HIV-associated wasting

Approved in European Union as Somatropin for:

• Growth hormone deficiency
• Turner syndrome
• Prader-Willi syndrome
• Chronic kidney disease
• Short stature due to Noonan syndrome
• Short stature due to Turner syndrome

Approved in Canada as Somatropin for:

• Growth hormone deficiency
• Turner syndrome
• Prader-Willi syndrome
• Chronic kidney disease
• Short stature due to Noonan syndrome
• Short stature due to Turner syndrome

Who Is Running the Clinical Trial?

Icahn School of Medicine at Mount Sinai

Lead Sponsor

Trials

933

Recruited

579,000+

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator

Trials

1,403

Recruited

655,000+

Published Research Related to This Trial

The PATRO Adults study is a large, long-term observational study designed to assess the safety and efficacy of somatropin (Omnitrope(®)) in adults with growth hormone deficiency, involving multiple centers across Europe.

This study will provide valuable insights into the long-term effects of Omnitrope(®) on various health factors, including diabetes development, tumor recurrence, and overall quality of life, while monitoring adverse events and safety outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rationale and design of PATRO Adults, a multicentre, noninterventional study of the long-term efficacy and safety of Omnitrope(®) for the treatment of adult patients with growth hormone deficiency. Beck-Peccoz, P., Minuto, F., Leal-Cerro, A., et al.[2021]

In a study of 67 Italian patients with growth hormone deficiency, Omnitrope® was found to be effective in improving health markers like insulin-like growth factor-1 levels and body composition over an average treatment duration of 45.4 months.

While 55.2% of patients experienced adverse events, none were deemed related to Omnitrope®, indicating that it is generally safe for long-term use in adults with growth hormone deficiency.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of Omnitrope&#174; in adults with growth hormone deficiency: Italian interim analysis of the PATRO Adults study. Ferone, D., Profka, E., Gasco, V., et al.[2018]

In a phase III study involving 70 prepubertal children with growth hormone deficiency, long-term treatment with Omnitrope (0.03 mg/kg/day) resulted in significant growth improvements over 4 years, including an average height increase of 31.1 cm.

Omnitrope was found to be safe and well tolerated, with significant increases in serum levels of IGF-1 and IGFBP-3, indicating effective stimulation of growth-related hormones.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of the recombinant human growth hormone Omnitrope® in the treatment of Spanish growth hormone deficient children: results of a phase III study. López-Siguero, J., Borrás Pérez, MV., Balser, S., et al.[2013]

In a 26-week phase 3 trial involving 92 adults with growth hormone deficiency, once-weekly somapacitan was found to be well tolerated, with no significant safety issues or severe injection site reactions reported.

Patients using somapacitan reported significantly higher treatment satisfaction regarding convenience compared to those using the once-daily Norditropin, indicating a preference for the less frequent dosing schedule.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and convenience of once-weekly somapacitan in adult GH deficiency: a 26-week randomized, controlled trial. Johannsson, G., Feldt-Rasmussen, U., Håkonsson, IH., et al.[2019]

Growth hormone (GH) therapy has significantly improved height outcomes in children with growth hormone deficiency (GHD), with growth velocity increasing from 3-4 cm/year before treatment to 10-12 cm/year during the first two years.

The transition from pituitary-derived GH to recombinant GH was accelerated due to safety concerns, and while higher doses (0.33 mg/kg/week) can enhance height outcomes, they come at a high cost and require careful monitoring for effectiveness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Growth hormone therapy. Bajpai, A., Menon, PS.[2023]

The PATRO Children study, involving 1837 patients across 184 sites in 10 European countries, is monitoring the long-term safety and efficacy of somatropin (Omnitrope(®)) in children requiring growth hormone treatment, with initial results showing no confirmed cases of diabetes or malignancies.

Efficacy data from the study are consistent with previous findings, indicating that Omnitrope(®) is safe for use in children, including those with specific conditions like Prader-Willi syndrome, and will provide further insights into its long-term safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Design of, and first data from, PATRO Children, a multicentre, noninterventional study of the long-term efficacy and safety of Omnitrope(®) in children requiring growth hormone treatment. Pfäffle, R., Schwab, KO., Marginean, O., et al.[2021]

Valtropin, a new biosimilar recombinant human growth hormone, demonstrated comparable efficacy to Humatrope in increasing height velocity in children with growth hormone deficiency, with a height velocity of 11.3 cm/year for Valtropin versus 10.5 cm/year for Humatrope over one year.

Both treatments showed similar safety profiles, with no significant differences in adverse events or bone maturation acceleration, indicating that Valtropin is a safe and effective option for treating children with growth hormone deficiency.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized, double-blind study to assess the efficacy and safety of valtropin, a biosimilar growth hormone, in children with growth hormone deficiency. Peterkova, V., Arslanoglu, I., Bolshova-Zubkovskaya, E., et al.[2007]

Norditropin® (somatropin) significantly increases height in Japanese children with short stature due to Noonan syndrome, with a greater effect observed at the higher dose of 0.066 mg/kg/day compared to 0.033 mg/kg/day over a 104-week treatment period.

The treatment demonstrated a favorable safety profile, with most adverse events being mild and unlikely related to the medication, and no withdrawals due to adverse events were reported.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of two doses of Norditropin&#174; (somatropin) in short stature due to Noonan syndrome: a 2-year randomized, double-blind, multicenter trial in Japanese patients. Ozono, K., Ogata, T., Horikawa, R., et al.[2022]

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