Announcing the sunRIZE Clinical Study in Congenital Hyperinsulinism

Trial ID: NCT04538989 RZ358
It’s a new day full of potential for people facing congenital hyperinsulinism (cHI), with the introduction of a new kind of treatment, known as RZ358. That’s why this clinical study is called sunRIZE. RZ358 has shown promise in previous studies of cHI patients and is now entering its latest phase of clinical research. sunRIZE is a global, Phase 3 clinical study, which will evaluate the results of RZ358 in people with cHI whose current treatments are not controlling their blood sugar levels well enough. The study is open to people diagnosed with cHI between 3 months to 45 years of age.

About the sunRIZE Study:

Because there are very few treatments available to safely treat cHI, researchers are looking for better ways to care for people with cHI. Clinical studies like sunRIZE are essential to discovering new treatment options.

The sunRIZE study is being done to see if adding RZ358 to standard medicines (also called “standard‑of‑care” or SOC) helps in controlling blood sugar levels among people with cHI. The study is also investigating other effects RZ358 may have in the body, and the impact RZ358 may have on the daily lives of participants.

All participants will continue to receive their SOC throughout the first part of the study. For participants over 1 year old, most will be randomly selected to get RZ358 (either a low or a high dose) and some will receive a placebo (a medicine that looks like RZ358 but does not have any active drug in it). None of the participants or the study doctors or staff will know during the study which treatment the participants are getting. With this method, researchers will be able to compare just how well RZ358 works when given together with SOC versus SOC by itself.

sunRIZE for Infants

For caregivers that are interested in having their infant participate in the sunRIZE study, a separate treatment group will allow patients aged 3 months to 1 yr to contribute to advancing the understanding of RZ358 treatment effects on cHI. All infant participants in this treatment group will be given SOC plus RZ358 (starting at the low‑dose of RZ358 and possibly increased to high‑dose of RZ358, if needed). Participants’ guardians, as well as study doctors and staff, will know that the infant is getting RZ358 treatment and how much RZ358 they are receiving.

Understanding how RZ358 works in infants is particularly important since nearly all cHI patients are diagnosed in the first year of life.

Estimated Enrollment

56 Participants

Phase

3

Eligibility Criteria

Participants must:

Inclusion Criteria

Participants must

  • Have been diagnosed with cHI by their doctor
  • Be between 3 months and 45 years of age
  • Have at least 3 hypoglycemia events per week along with other key glucose measures
  • Complete an evaluation involving medical tests and assessments

Participants must not:

Exclusion Criteria

Participants must not have

  • Have a diagnosis of diabetes.
  • Be pregnant or breastfeeding.
  • Have any medical conditions that would make it unsafe for them to take part in the study, such as HIV, hepatitis, alcoholism, or drug abuse.

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About the Condition/ Disease Overview:

Congenital hyperinsulinism (cHI) is a rare disease in which the pancreas produces too much insulin. Insulin helps control blood sugar levels in the body. Because of high levels of insulin, people with cHI can have frequent episodes of dangerously low blood sugar levels, also called “hypoglycemia”. cHI is the most frequent cause of severe, persistent hypoglycemia in newborns, infants, and children. If not recognized early and treated properly, these episodes of hypoglycemia can lead to serious problems such as seizures, brain damage and death.

“I just have so many questions and concerns about our family’s future with this diagnosis. It’s been a week at the hospital and I’m still nowhere near being able to accept this. I scour literature and the Facebook group and try to find something in anyone’s story that can encourage me but everything I see just drives home the fact we will never lead a normal life and so many of my hopes and dreams for our family and for our little one are shattered.” Banerjee, 2022 – https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8858501/

About RZ358

RZ358 has been specifically developed to treat cHI. It is a type of medicine called a monoclonal antibody—meaning it is a protein created in a research laboratory that can act like the body’s own targeting system to work at a very specific point inside the body. Different monoclonal antibodies have already provided breakthrough treatments in many health conditions.

RZ358 targets the insulin receptor to turn down insulin’s signal to take sugar from the bloodstream, which is particularly helpful to keep the sugar levels in a safer range to protect the brain.

RZ358 is given intravenously (small tube placed into a vein, also called an IV). It takes approximately 30 minutes for the medicine to get through the IV and into the body. The amount of RZ358 given depends on a participant’s weight and to which treatment group they will be randomly assigned.

“Participating in the [RIZE Phase 2b] study was the best thing that could have happened because my daughter was able to regulate her glucose levels much better.”

What Will Happen in This Study

Screening Visit

If you are interested in joining this study (or if you would like your child to take part in the study), you will need to visit one of the study centers nearest to your home.

A study doctor will explain the study to you. If you agree to join or if you agree on behalf of your child, the doctors and site staff will check your or your child’s: overall health, blood sugar levels, and other health conditions through simple tests. Study doctors will also ask you questions about your or your child’s: cHI symptoms, medical history, and medication history to make sure you or your child is eligible to take part in the study. The screening period described above may last for up to 35 days.

If you or your child meet all the criteria of the study and decide to join the study, you or your child will continue the usually prescribed cHI treatment and be given either RZ358 or placebo.

Study Visit

Participants will receive a total of 7 doses of the study treatment over 24 weeks – 3 doses every other week, then 4 monthly doses. Participants will visit a clinical study center at least 8 times during the treatment period. During study site visits, the study doctors will:

  • Check the participant’s blood sugar level.
  • Give the participant an examination.
  • Collect blood and urine samples.
  • Ask about the participant’s cHI symptoms, how cHI symptoms affect the participant’s quality of life, and other relevant medical questions

At the end of the study, participants will have the option, with the input of the study doctor, to continue into the extended portion of the study and receive RZ358 for a longer time (up to 3 years). For participants that choose to stop the study after the first 24-week portion, there will be 2 monthly visits to the study center after getting the last dose of the assigned study treatment (RZ358 or placebo).

Where You Can Join

The study will be offered at the sites shown below. Travel support may be provided to the nearest site for qualified participants. Speak with site staff to learn more about available travel support.

Location
University of Saskatchewan, Jim Pattison Children’s Hospital
Status
Recruiting
Location
University Children’s Hospital – Dusseldorf
Status
Recruiting
Location
Hospital Necker – Enfants Malades
Status
Recruiting
Location
Great Ormond Street Hospital
Status
Not yet Recruiting
Location
National Children’s Hospital Vietnam
Status
Not yet Recruiting
Location
Vall d’Hebron
Status
Recruiting
Location
Hospital Universitario Virgen del Rocio - PPDS
Status
Recruiting
Location
King Faisal Specialist Hospital & Research Center
Status
Not yet Recruiting
Location
National Guard Hospital Affairs
Status
Not yet Recruiting
Location
Hacettepe Universitesi Tip Fakultesi Hastanesi
Status
Not yet Recruiting
Location
Royal Manchester Children’s Hospital
Status
Not yet Recruiting
Location
Institute for Experimental Pediatric Endocrinology – Charite
Status
Recruiting
Location
Sheik Shabout Medical Center
Status
Not yet Recruiting
Location
Sidra Medicine
Status
Not yet Recruiting
Location
Sultan Qaboos University Hospital
Status
Not yet Recruiting
Location
Edmond & Lilly Safra’s Children’s Hospital
Status
Recruiting
Location
Centre Hospitalier Universitaire Lyon
Status
Recruiting
Location
Odense University Hospital, Hans Christian Anderson Children’s Hospital
Status
Recruiting
Location
Medical University of Varna UMHAT “St. Marina”
Status
Not yet Recruiting
Location
M. Iashvili children’s Central Hospital
Status
Recruiting
Location
Medical University of Varna UMHAT “St. Marina”
Status
Recruiting
Location
University of Athens, Aghia Sophia Children’s Hospital
Status
Recruiting

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Frequently Asked Questions (FAQs)