Carbetocin Nasal Spray for Prader-Willi Syndrome

Not currently recruiting at 33 trial locations

Overseen By Sandy Filosi

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: ACADIA Pharmaceuticals Inc.

Must not be taking: Prostaglandins, Oxytocin, Desmopressin, others

Disqualifiers: Schaaf-Yang syndrome, Cardiovascular disorder, Epilepsy, others

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: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a nasal spray treatment, carbetocin, for individuals with Prader-Willi syndrome (PWS), specifically targeting those with constant hunger and excessive food-seeking behavior. Participants will receive either the carbetocin spray or a placebo to compare effects over 12 weeks. Suitable candidates include individuals aged 5 to 30 with PWS and a notably high appetite that remains unsatisfied. If the condition frequently disrupts daily life with food-seeking behavior, this trial may be appropriate. As a Phase 3 trial, this study represents the final step before FDA approval, offering a chance to contribute to a potentially groundbreaking treatment for PWS.

Will I have to stop taking my current medications?

The trial requires participants to stop using certain medications, such as nasal saline, other nasal irrigation, and intranasal medications during the study. Additionally, you must not have used prostaglandins, oxytocin, desmopressin, or tesofensine within specific timeframes before the study. If you are on a GLP-1 agonist, you must have been taking it for more than 6 months before the screening.

Is there any evidence suggesting that carbetocin nasal spray is likely to be safe for humans?

Research has shown that carbetocin nasal spray is generally safe for people with Prader-Willi syndrome. In studies, a 3.2 mg dose taken three times a day showed promising results. Participants experienced improvements in eating habits, anxiety, and stress levels. This suggests the treatment is both effective and safe. These studies reported no major safety issues. For those considering joining a trial, this information may reassure about the treatment's safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Prader-Willi syndrome?

Carbetocin nasal spray is unique because it offers a new delivery method for treating Prader-Willi Syndrome. Unlike the standard treatments, which often involve behavioral therapies and medications like growth hormone and appetite suppressants, Carbetocin is administered as a nasal spray, making it potentially more convenient and faster-acting. Researchers are excited about its ability to mimic oxytocin, a hormone linked to social bonding and appetite regulation, which could address some of the core symptoms of Prader-Willi Syndrome more directly.

What evidence suggests that carbetocin nasal spray might be an effective treatment for hyperphagia in Prader-Willi syndrome?

Research has shown that carbetocin nasal spray might help reduce excessive eating and related behaviors in people with Prader-Willi syndrome (PWS). In this trial, participants will receive either a 3.2 mg dose of carbetocin nasal spray three times daily or a placebo. Earlier studies found that patients who took a 3.2 mg dose experienced noticeable improvements in their eating habits and anxiety levels compared to those who took a placebo. However, some recent findings suggested that carbetocin did not significantly reduce excessive eating in all cases. This mixed evidence indicates that while carbetocin is promising, its effectiveness may vary among individuals with PWS. ^{1 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for males and females aged 5-30 with Prader-Willi syndrome, experiencing intense hunger and food-seeking behavior. Participants must have a caregiver able to follow study procedures. Exclusions include other cognitive impairments, recent nasal surgery, severe respiratory diseases, use of certain medications like prostaglandins or oxytocin recently, and new dietary interventions within the last month.

Inclusion Criteria

I am between 5 and 30 years old.

I have Prader-Willi syndrome with a confirmed genetic mutation.

I often feel very hungry and seek food but don't feel full easily.

Exclusion Criteria

Active psychotic symptoms, a history of psychotic symptoms, or a psychotic disorder

History of suicide attempt or inpatient psychiatric hospitalization

New food-related interventions, including environment or dietary restrictions, within 1 month of the Screening visit

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive carbetocin nasal spray or placebo for 12 weeks

12 weeks

Regular visits for administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Carbetocin

Trial Overview The study tests Carbetocin nasal spray against a placebo over 12 weeks to see if it can reduce excessive eating (hyperphagia) in those with Prader-Willi syndrome. It's randomized and double-blind, meaning neither participants nor researchers know who gets the real treatment versus the placebo.

How Is the Trial Designed?

Group I: Carbetocin Experimental Treatment 1 Intervention

Carbetocin nasal spray 3.2 mg three times daily (TID)

Group II: Placebo Placebo Group 1 Intervention

Placebo

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)

Published Research Related to This Trial

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]

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]

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]

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]

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]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36633570/

Intranasal Carbetocin Reduces Hyperphagia, Anxiousness ... Carbetocin was well tolerated, and the 3.2-mg dose was associated with clinically meaningful improvements in hyperphagia and anxiousness and distress behaviors.

2. academic.oup.com academic.oup.com/jcem/article/108/7/1696/6986298

Intranasal Carbetocin Reduces Hyperphagia, Anxiousness ... Efficacy results over the 14 days of the trial demonstrated significant improvements in hyperphagia and other behavioral symptoms in PWS vs the placebo arm, ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06173531

NCT06173531 | Study of Carbetocin Nasal Spray for the ... 12-week, randomized, double-blind, placebo-controlled, parallel-group study of carbetocin nasal spray for the treatment of hyperphagia in Prader-Willi syndrome ...

4. pwsausa.org pwsausa.org/acadia-shares-results-of-phase-3-carbetocin-trial-primary-endpoint-not-met/

Acadia Shares Results of Phase 3 Carbetocin Trial In other words, The trial results showed that the carbetocin drug did not make a meaningful enough difference in reducing hyperphagia compared ...

5. horizonscandb.pcori.org horizonscandb.pcori.org/report/topics/417

Carbetocin (ACP-101) to treat hyperphagia associated with ... Carbetocin is thought to help improve appetite control and reduce food intake. It might also reduce obsessive-compulsive symptoms and anxiety associated with ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06420297

NCT06420297 | OLE Study of Carbetocin Nasal Spray for ... This is a long-term, OLE study to evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS.

7. fpwr.org fpwr.org/blog/pws-clinical-trial-webinar-carbetocin

PWS Clinical Trial Webinar: Carbetocin A new study is now enrolling patients ages 7 to 18 years old to determine the safety and efficacy of Carbetocin for the treatment of symptoms related to PWS.

8. acadia.com acadia.com/en-us/media/news-releases/acadia-pharmaceuticals-initiates-pivotal-phase-3-study

Acadia Pharmaceuticals Initiates Pivotal Phase 3 Study of ... Carbetocin nasal spray is an investigational drug being developed for the treatment of hyperphagia in Prader-Willi syndrome (PWS).