Hypothalamic Obesity | About the Horizon-HO Study

What is the purpose of the Horizon-HO study?

The Horizon-HO study is a clinical research study to find out if a study drug can help people with hypothalamic obesity have a healthier body weight. The study will also find out how much of the study drug can be taken without causing problems (side effects).

What will happen during the study?

Participation in the Horizon-HO study may last about a year and a half. It includes the following parts:

Screening | Up to 3 weeks

Participants visit the study doctor’s office and have study tests and procedures to find out if they can be in the study. This will include a physical exam, a full-body scan, a heart test, blood and urine tests, questionnaires, and various other tests and procedures.

Part 1 | Double-Blind | 36 to 38 weeks (about 9 months)

Participants are assigned at random (like rolling dice) to receive a low, medium, or high dose of the study drug or placebo. Placebo looks like the study drug but does not have active drug ingredients in it.
Double-Blind means that neither you nor study doctors and staff know whether you are receiving study drug or placebo. Study doctors can find out if necessary for emergency purposes.
Participants visit the study doctor’s office about once a month for the first 4 months and then once about every 10 weeks thereafter. Some visits may also be completed remotely using telemedicine.
After 36 weeks, participants may either continue to Part 2 or stop participating and slowly stop taking the study drug over 2 weeks.

Part 2 | Optional Open-Label | 38 weeks (about 9 months)

Participants who decide to stay in the study receive the study drug. There will be no chance of receiving placebo.
Open-Label means that everyone knows that you are receiving the study drug (and not placebo).
Participants visit the study doctor’s office about once a month for the first 4 months and then once about every 10 weeks thereafter.
At the end of Part 2, or if participants leave the study early, they will be asked to slowly stop taking the study drug over 2 weeks.

Who can join the Horizon-HO study?

People who meet the following study requirements may be able to join:

Male or female, 16 years of age or older
Diagnosed with hypothalamic obesity caused by damage to the hypothalamus
Note: Any therapy directly related to the damage must be completed at least 6 months before Screening.
Stable body weight
If participants are taking any of the below, they must be on a stable dose:
pituitary replacement
growth hormone
antidiabetic medications
lipid-lowering medications

Other study requirements will apply.

Participant Journey

1. Read & sign the Informed Consent Form

You will read and sign the Informed Consent Form (ICF) to show that you agree to be in the study.

2. Go to a Screening visit

At the Screening visit, you will have study tests and procedures to find out if you can be in the study.

3. Get assigned to a study group

If Screening tests confirm you can be in the study, you will be assigned at random (like rolling dice) to a group that receives the study drug or a placebo (which has no active study drug in it).

4. Begin Part 1 (Double-Blind)

You will take the study drug or placebo every day and visit the study doctor’s office about once a month for the first 4 months and then about every 10 weeks thereafter.

5. Begin Part 2 (Open-Label Extension), if applicable

You will take the study drug every day (with no chance of receiving placebo) and attend study visits about once a month for the first 4 months and then every 10 weeks thereafter until the end of the study.