Active not recruitingPHASE3INTERVENTIONAL

Evaluation of the Safety and Efficacy of Eneboparatide (AZP-3601) in Patients With Chronic Hypoparathyroidism

This study is investigating a new treatment, eneboparatide, for adults aged 18-80 who have chronic hypoparathyroidism. The main goal is to see how safe the medicine is and if it helps people manage their condition better. For the first 24 weeks, some participants will receive eneboparatide and others a dummy medicine (placebo). Neither you nor your doctor will know which you are receiving. The medicine is given by injection using a special pen, and the dose will be adjusted for each person. After this, all participants will receive eneboparatide for a further 132 weeks. This research could lead to new ways to help people with hypoparathyroidism.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Alexion Pharmaceuticals, Inc.

Enrolment target

165

Start

07 Jun 2023

Estimated completion

16 Jun 2027

Chronic Hypoparathyroidism Endocrine System Diseases Parathyroid Diseases

What is this study about?

This study is testing a new medicine called eneboparatide (also known as AZP-3601) for people who have chronic hypoparathyroidism. This is a condition where your body doesn't make enough parathyroid hormone, which is important for controlling calcium levels in your blood. When calcium levels are too low, it can lead to various health problems.

The main purpose of this study is to see if eneboparatide is safe for people to use and if it can effectively help manage their hypoparathyroidism. This means checking if it helps bring calcium levels back to a healthy range and if it reduces the need for other medications like calcitriol and calcium supplements, which are often used to treat the condition.

Finding new treatments is important because current therapies might not work for everyone or can have side effects. This research could provide a new option that helps people with chronic hypoparathyroidism live more comfortably and with better-controlled calcium levels, improving their overall quality of life.

Key takeaways

This study evaluates a new treatment, eneboparatide, for chronic hypoparathyroidism.
It aims to check both the safety and effectiveness of the medicine.
Participation involves injections and regular check-ups over about 3 years.
For the first 24 weeks, some people receive the new medicine, others a placebo (dummy treatment).
All participants will receive eneboparatide after the initial 24 weeks.
You can stop participating at any time.

Who may be eligible?

This study is looking for both men and women between the ages of 18 and 80 who have had chronic hypoparathyroidism for at least a year. To be considered, you should have consistently low levels of parathyroid hormone and calcium, and currently be taking specific amounts of calcitriol or alphacalcidol, along with at least 1000mg of calcium supplements daily.

Before starting the main part of the study, your calcium levels will need to be carefully adjusted and stable within a specific range. You'll also need to have healthy kidney function and certain vitamin and mineral levels in your blood. You should be able to give yourself daily injections using a pre-filled pen, or have someone who can help you do this.

There are also some reasons why you might not be able to join. These include having certain significant abnormal blood test results, very high blood pressure, or mental health issues that might make it difficult to understand or fully participate in the study. Women who could become pregnant must agree to use effective birth control throughout the study.

Quick self-check

Are you between 18 and 80 years old?
Have you been diagnosed with chronic hypoparathyroidism for at least 12 months?
Are you currently taking calcitriol (or alphacalcidol) and at least 1000mg of calcium supplements daily?
Are your kidneys generally healthy (eGFR of 30 or higher)?
Can you give yourself daily injections, or do you have someone who can help you?
If you are a woman who could become pregnant, are you willing to use effective birth control throughout the study?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, it will last for a total of about 3 years. For the first 24 weeks, you'll be randomly assigned to receive either the new medicine, eneboparatide, or a dummy medicine (placebo). Neither you nor your study doctor will know which one you are getting. Both medicines are given as a daily injection under the skin using a pre-filled pen, and your dose will be adjusted specifically for you.

After these first 24 weeks, everyone in the study, including those who were on the placebo, will receive eneboparatide for an additional 132 weeks. Throughout the study, you'll have regular visits to the clinic for medical check-ups, blood tests, and to discuss how you're feeling. These assessments will help the researchers understand how the medicine is working and monitor your health. You will be taught how to administer the injections yourself.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving a new medicine that could help manage your hypoparathyroidism if current treatments aren't working well enough. However, there are also potential risks, including side effects from the study medicine or the frequent blood tests. As with any new medicine, not all side effects are known yet. You will be closely monitored by the study team, and you are free to withdraw from the study at any time and for any reason, without it affecting your usual medical care.

Locations (54)

Harbor UCLA Medical Center Endocrinology
Torrance, United States
Denver Endocrinology Diabetes and Thyroid Center
Denver, United States
University of Chicago - Medical Center
Chicago, United States
North Shore University Health System
Evanston, United States
Indiana University (IU) Health University Hospital
Indianapolis, United States
Massachusetts General Hospital
Boston, United States
Mayo Clinic
Rochester, United States
Northern Nevada Endocrinology
Reno, United States
Colombia University Irving Medical Center
New York, United States
Physician's East Endocrinology
Greenville, United States
The Ohio State University Wexner Medical Center
Columbus, United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, United States

+42 more sites — see the official record for the full list.

Common questions

What is chronic hypoparathyroidism?

It's a long-term condition where your body doesn't produce enough parathyroid hormone, which leads to low calcium levels in your blood.

What is eneboparatide?

It's a new medicine being tested in this study to see if it can help manage chronic hypoparathyroidism.

Will I know if I'm getting the real medicine or a dummy medicine?

For the first 24 weeks, neither you nor your doctor will know. This is called a 'blinded' study and helps make sure the results are fair. After that, everyone will receive eneboparatide.

How will the medicine be given?

It will be given as a daily injection under your skin using a special pen, similar to an insulin pen.

How long will the study last?

The study will last for approximately 3 years in total, with an initial 24-week period followed by a longer 132-week period.