Setmelanotide to Treat Obesity in a Patient With Pseudohypoparathyroidism Type 1a (PHP1a)

NCT07496463 | Enrolling By Invitation Phase 2 FDA Drug

• 1 other identifier: 2026P000332
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

The investigators plan to test the efficacy and safey of 6-months of open-label setmelanotide to treat obesity in a single patient with pseudohypoparathyroidism type 1a due to a GNAS mutation.

Conditions

Pseudohypoparathyroidism Type 1a; Obesity

Keywords

pseudohypoparathyroidism type 1a; obesity; setmelanotide

Outcome Measures

Primary Outcomes (1)

• Greater than or Equal to 5% Weight loss

  Greater than or equal to 5% weight loss from baseline

  Baseline to 6 Months

Secondary Outcomes (4)

• Percent Weight Loss

  Baseline to 3 Months, Baseline to 6 Months

• Trunk Fat Mass

  Baseline to 6 Months

• Hemoglobin A1c

  Baseline to 3 Months, Baseline to 6 Months

• Serum Triglycerides

  Baseline to 3 Months, Baseline to 6 Months

Other Outcomes (5)

• Eating Behaviors

  Baseline to 3 Months, Baseline to 6 Months

• Total Daily Calorie Intake

  Baseline to 3 Months, Baseline to 6 Months

• Daily Step Count

  Baseline to 3 Months, Baseline to 6 Months

Study Arms (1)

Patient with pseudohypoparathyroidism type 1a

OTHER

A preselected patient with obesity related to pseudohypoparathyroidism type 1a.

Drug: Setmelanotide

Interventions

Setmelanotide DRUG

Setmelanotide will be prescribed at the standard initial dose of 2 mg SC daily. The treatment will be uptitrated at the 2-Week visit to 3 mg SC daily if tolerated. We will continue this maximal dose for a period of 6 months.

Patient with pseudohypoparathyroidism type 1a

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Known patient with PHP1a (confirmed GNAS mutation)
• Optimized therapy for diabetes and dyslipidemia

You may not qualify if:

• \- Use of medications that may affect endpoints that are changed within 3 months prior to Baseline or that are likely to require a change in dose during the open-label treatment period

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Related Links

• Related Info

MeSH Terms

Conditions

Pseudohypoparathyroidism; Obesity

Interventions

setmelanotide

Condition Hierarchy (Ancestors)

Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Calcium Metabolism Disorders; Overweight; Overnutrition; Nutrition Disorders; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor of Medicine

Model Details: Single patient open-label study

Study Record Dates

• First Submitted: March 22, 2026
• First Posted: March 27, 2026
• Study Start: July 21, 2026
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): April 30, 2027
• Last Updated: July 23, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: IPD and supporting information will be available upon completion of the study for at least 3 years.
• Access Criteria: IPD and supporting information may be requested from the study PI. Each request will be considered on a case-by-case basis and granted if appropriately justified.

Locations

US (1)