Carbetocin Nasal Spray for Prader-Willi Syndrome

Recruiting at 29 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: ACADIA Pharmaceuticals Inc.

Disqualifiers: Cerebrovascular disease, Epilepsy, Psychotic disorder, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 36 months

160 Participants Needed

Trial Summary

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the study team for guidance.

What data supports the effectiveness of the drug Carbetocin Nasal Spray for Prader-Willi Syndrome?

Research shows that intranasal carbetocin, which is similar to the hormone oxytocin, helps reduce excessive eating, anxiety, and distress in people with Prader-Willi syndrome. This was demonstrated in a study where adolescents with the condition experienced these benefits. ^{1 2 3 4 5}

How is the drug Carbetocin nasal spray unique for treating Prader-Willi syndrome?

Carbetocin nasal spray is unique because it is an oxytocin analog (a substance similar to the hormone oxytocin) that is administered intranasally (through the nose) and specifically targets symptoms like excessive hunger, anxiety, and distress in Prader-Willi syndrome, unlike other treatments that may focus primarily on weight management. ^{1 2 3 6 7}

What is the purpose of this trial?

To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS

Eligibility Criteria

This trial is for individuals with Prader-Willi Syndrome, particularly those who experience hyperphagia or binge eating. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may need to have a confirmed diagnosis of the condition.

Inclusion Criteria

Has completed the Week12/EOT visit of the antecedent, Study ACP-101-302

Met all entry criteria for the antecedent study

May benefit from long-term treatment with open-label carbetocin in the judgment of the Investigator

Exclusion Criteria

History of suicide attempt or inpatient psychiatric hospitalization

Has a clinically significant abnormality in vital signs at Baseline

Has an average QTcF interval of >450 ms on the Baseline ECG performed before the first dose of carbetocin is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive carbetocin nasal spray 3.2 mg three times daily (TID) to evaluate long-term safety and tolerability

36 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Carbetocin

Trial Overview The study is testing the long-term safety and tolerability of a nasal spray containing carbetocin (3.2 mg taken three times daily) in subjects with Prader-Willi Syndrome to see if it can help manage their symptoms.

Participant Groups

Group I: Drug: Carbetocin Experimental Treatment 1 Intervention

Carbetocin nasal spray 3.2 mg three times daily (TID)

Who Is Running the Clinical Trial?

ACADIA Pharmaceuticals Inc.

Lead Sponsor

Trials

49

Recruited

11,700+

Founded

1993

Headquarters

San Diego, USA

Known For

Neurological Disorders

Top Products

Nuplazid (pimavanserin), Daybue (trofinetide)

Findings from Research

In a randomized, double-blind trial involving 37 adolescents with Prader-Willi syndrome, intranasal carbetocin significantly reduced hyperphagia symptoms compared to placebo, indicating its potential efficacy in managing this condition.

Carbetocin was well tolerated, with a similar incidence of adverse events between the treatment and placebo groups, suggesting it is a safe option for improving behavioral symptoms associated with Prader-Willi syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intranasal carbetocin reduces hyperphagia in individuals with Prader-Willi syndrome. Dykens, EM., Miller, J., Angulo, M., et al.[2019]

In a phase 3 trial involving 130 participants with Prader-Willi syndrome, intranasal carbetocin was found to be well tolerated, with the 3.2 mg dose showing clinically meaningful improvements in hyperphagia and anxiousness compared to placebo.

Although the primary endpoints did not reach statistical significance, the 3.2 mg dose demonstrated nominally significant improvements in hyperphagia, anxiousness, and distress behaviors, with benefits sustained during long-term follow-up.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial. Roof, E., Deal, CL., McCandless, SE., et al.[2023]

A review of 14 studies and a case series of 10 adolescents with Prader-Willi syndrome (PWS) indicated that anti-obesity medications (AOMs) can help manage hyperphagia, with 60% of participants showing decreased or stable BMI z-scores after treatment.

No significant side effects were reported in the case series, suggesting that AOMs may be safe for use in youth with PWS, but further research is needed to establish comprehensive treatment guidelines.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Anti-Obesity Medication Use in Children and Adolescents with Prader-Willi Syndrome: Case Review and Literature Search. Goldman, VE., Naguib, MN., Vidmar, AP.[2023]

Recombinant human growth hormone (rhGH) treatment in 20 children with Prader-Willi syndrome led to significant catch-up growth, with many reaching adult heights that were markedly taller than historical controls after 10 years.

While rhGH treatment correlated with a reduction in body fat percentage, patients remained severely obese, and their health-related quality of life was still significantly lower than that of the normal population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term effects of growth hormone therapy on patients with Prader-Willi syndrome. Sipilä, I., Sintonen, H., Hietanen, H., et al.[2015]

Exenatide, a GLP-1 agonist, was effectively used to manage diabetes in a 19-year-old female with Prader-Willi syndrome, highlighting its potential in this specific population.

The drug demonstrated beneficial effects in reducing weight and suppressing appetite, suggesting it could be a promising treatment strategy for obesity and diabetes associated with Prader-Willi syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treating diabetes mellitus in Prader-Willi syndrome with Exenatide. Seetho, IW., Jones, G., Thomson, GA., et al.[2018]

Chronic administration of diazoxide in a mouse model of Prader-Willi syndrome (PWS) significantly reduced body weight and fat mass while improving metabolic parameters like blood glucose levels and endurance capacity.

These results suggest that diazoxide may be an effective treatment for obesity in children with PWS, indicating potential pharmacological targets for managing obesity-related disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Chronic diazoxide treatment decreases fat mass and improves endurance capacity in an obese mouse model of Prader-Willi syndrome. Bischof, JM., Wevrick, R.[2019]

A case study of a 20-year-old female with Prader-Willi syndrome (PWS) showed that the SGLT2 inhibitor empagliflozin significantly improved her glycemic control and reduced body weight after previous treatment with the GLP-1 analog liraglutide.

The findings suggest that SGLT2 inhibitors could be a promising treatment option for managing diabetes in young PWS patients, particularly those with preserved pancreatic insulin secretion.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of sodium-glucose cotransporter 2 inhibitor with glucagon-like peptide-1 receptor agonist for the glycemic control of a patient with Prader-Willi syndrome: a case report. Sano, H., Kudo, E., Yamazaki, T., et al.[2020]

References

1. United States pubmed.ncbi.nlm.nih.gov

Intranasal carbetocin reduces hyperphagia in individuals with Prader-Willi syndrome. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Intranasal Carbetocin Reduces Hyperphagia, Anxiousness, and Distress in Prader-Willi Syndrome: CARE-PWS Phase 3 Trial. [2023]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Anti-Obesity Medication Use in Children and Adolescents with Prader-Willi Syndrome: Case Review and Literature Search. [2023]

4. Norway pubmed.ncbi.nlm.nih.gov

Long-term effects of growth hormone therapy on patients with Prader-Willi syndrome. [2015]

5. Ireland pubmed.ncbi.nlm.nih.gov

Treating diabetes mellitus in Prader-Willi syndrome with Exenatide. [2018]

6. United States pubmed.ncbi.nlm.nih.gov

Chronic diazoxide treatment decreases fat mass and improves endurance capacity in an obese mouse model of Prader-Willi syndrome. [2019]

7. Japan pubmed.ncbi.nlm.nih.gov

Efficacy of sodium-glucose cotransporter 2 inhibitor with glucagon-like peptide-1 receptor agonist for the glycemic control of a patient with Prader-Willi syndrome: a case report. [2020]