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A Phase 3, Double Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Setmelanotide in Patients with Acquired Hypothalamic Obesity

A Phase 3, Double Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Setmelanotide in Patients with Acquired Hypothalamic Obesity

Flaherty Jim

Rhythm Pharmaceuticals, Inc.

222 Berkeley Street 12th Floor Boston, MA 02116

clinicaltrials@rhythmtx.com

Sogame Kaori

CMIC Co., Ltd.

1-1-1, Shibaura, Minato-ku, Tokyo

+81-90-6493-9787

ClinicalTrialInformation@cmic.co.jp

Not Recruiting

Mar. 29, 2024

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

1. Patient has documented evidence of acquired HO
2. Aged 4 years and older
3. Documented weight gain associated with the hypothalamic injury either, and a body mass index (BMI) of >-30 kg/m2 for patients >-18 years of age, or BMI>-95th percentile for age and sex for patients 4 to <18 years of age
4. A highly effective form of contraception must be used/practiced throughout the trial and for 90 days
5. If receiving hormone replacement therapy, the dose of such therapy has remained stable for at least 2 months prior to Screening

Other protocol defined Inclusion criteria may apply.

1. Diagnosis of Prader-Willi syndrome (PWS) or Rapid-onset obesity with hypoventilation, hypothalamic, autonomic dysregulation, neuroendocrine tumor syndrome (ROHHADNET)
2. Weight loss >2% in the previous 3 months for patients aged >-18 years or >2% reduction in BMI for patients aged 4 to <18 years
3. Bariatric surgery or procedure within the last 2 years
4. Diagnosis of severe psychiatric disorders, or any suicidal ideation, attempts, or any suicidal
5. Current, clinically significant pulmonary, cardiac, metabolic, or oncologic disease considered
6. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion)
7. History or close family history of skin cancer or melanoma
8. Participation in any clinical trial with an investigational drug/device within 3 months, prior to the first trial dose
9. Previously enrolled in a clinical trial involving setmelanotide or any previous exposure to setmelanotide
10. Inability to comply with once daily (QD) injection regimen
11. Pregnant and/or breastfeeding
12. Patients with obesity attributable to other genetic or syndromic conditions (eg, PPL [POMC, PCSK1, LEPR, collectively], BBS) prior to the hypothalamic injury
13. Hypersensitivity to setmelanotide and/or any excipients contained in the investigational drug

Other protocol defined Exclusion criteria may apply.

Both

Acquired hypothalamic obesity (HO)

Patients who are eligible to enter the trial will be randomized in a blinded manner on Day 1 in a 2:1 ratio, stratified by age group (>-18 years old, >-12 and <18 years old, <12 years old) and subpopulation (Japanese vs non-Japanese), to receive either setmelanotide (2) or placebo (1).
Setmelanotide 0.5 mg QD or placebo equivalent will be subcutaneous injection (SC) administered as the starting dose for all patients. After the initial dose of 0.5 mg, patients will be escalated each week in increments of either 0.5 or 1.0 mg to achieve the individual patient's therapeutic regimen.
Patients will receive trial treatment (setmelanotide or placebo, per randomization) for 52 weeks on a therapeutic regimen (up to approximately 60 weeks).
Patients who complete 52 weeks of treatment on a therapeutic regimen and remain enrolled may initiate open- label setmelanotide via Bridging visits every 12 weeks and last until either an LTE study or a post-trial access program is available, commercial drug is available in the region, or the trial ends. Since the patient's initial assignment to either setmelanotide or placebo will continue to be blinded to all parties, open-label dosing in Bridging visits will follow the setmelanotide dosing schedule for treatment on a therapeutic.

Mean % change in BMI from Baseline after approximately 52 weeks on a therapeutic regimen of setmelanotide compared to placebo

+81-52-851-5511

Mar. 11, 2024

No

Canada/Germany/Netherlands/United Kingdom/United States