Growth Hormone for Autism Spectrum Disorder

Recruiting in New York (>99 mi)

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

32 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial will test if growth hormone can help children with Phelan-McDermid syndrome and idiopathic autism. Researchers hope it will improve their social skills, language, and reduce repetitive behaviors. The study will involve 45 children who will receive daily injections for a few months. Growth hormone has been previously tested in children with Phelan-McDermid syndrome, showing good tolerance without serious issues.

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.

What data supports the effectiveness of the drug Growth Hormone for Autism Spectrum Disorder?

While there is no direct evidence for Growth Hormone's effectiveness in Autism Spectrum Disorder, studies show that Growth Hormone therapy can significantly increase growth rates in children with growth hormone deficiency and other conditions like Turner's syndrome, suggesting potential benefits in growth-related outcomes. ^{1 2 3 4 5}

Is growth hormone treatment generally safe for humans?

Growth hormone treatments like Omnitrope and Norditropin have been studied for safety in both children and adults with growth hormone deficiency and other conditions. These studies generally show a favorable safety profile, with most side effects being mild and not directly related to the treatment. Long-term studies in children and adults have not shown significant safety concerns. ^{4 6 7 8 9}

How does the drug Growth Hormone differ from other treatments for Autism Spectrum Disorder?

Growth Hormone (Somatropin) is unique because it is typically used for growth hormone deficiency and is administered as a subcutaneous injection, potentially offering a novel approach for Autism Spectrum Disorder by targeting growth-related pathways, unlike standard behavioral therapies. ^{4 6 10 11 12}

Research Team

Alexander Kolevzon

Principal Investigator

Icahn School of Medicine at Mount Sinai

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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 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]

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]

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]

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Long-term results of GH therapy in GH-deficient children treated before 1 year of age. [2019]

2. Israel pubmed.ncbi.nlm.nih.gov

Growth hormone therapy: emerging dilemmas. [2012]

3. Germany pubmed.ncbi.nlm.nih.gov

Efficacy and safety of Valtropin in the treatment of short stature in girls with Turner's syndrome. [2019]

4. Switzerland 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. [2007]

5. India pubmed.ncbi.nlm.nih.gov

Growth hormone therapy. [2023]

6. United States 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. [2021]

7. United States 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. [2021]

8. Japan 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. [2022]

9. Italy 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. [2018]

10. United States 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. [2013]

11. Switzerland pubmed.ncbi.nlm.nih.gov

Lonapegsomatropin: Pediatric First Approval. [2022]

12. England pubmed.ncbi.nlm.nih.gov

Safety and convenience of once-weekly somapacitan in adult GH deficiency: a 26-week randomized, controlled trial. [2019]