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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Arm Efficacy and Safety Study of RZ358 in Patients with Congenital Hyperinsulinism
EU CTIS ID: 2023-503240-13-00
What this study is testing
To assess the glycemic efficacy of RZ358 by hypoglycemia events on point of care self-monitored blood glucose over 24 weeks of treatment
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Provide written informed consent and, as applicable, assent, before any study specific procedures are performed.
- At screening, aged ≥3 months (corrected for gestational age for children under 9 months) and ≤45 years.
- An established clinical diagnosis of congenital HI, with or without identification of a known monogenic variant by genetic testing.
- Participant has failed to achieve adequate glycemic control with appropriate and reasonable trials of locally accepted and available SOC medical therapies (e.g., diazoxide and SSAs) per the judgment of the investigator.
- Experiencing ≥3 hypoglycemia events (<70 mg/dL [<3.9 mmol/L]) per week by screening SMBG and/or according to the investigator’s evaluation, AND Average daily percent time with hypoglycemia (<70 mg/dL [<3.9 mmol/L]) of ≥8% of the monitored screening CGM time.
- Hepatic ultrasound at screening without clinically significant findings, including clinically significant gallstones as judged by the investigator (e.g., large size, obstructive, or biliary colic), or evidence of peliosis hepatitis.
You likely can't join if
- Participants meeting any of the following criteria will be excluded from the study: 1. Any out-of-range laboratory value at screening that is assessed as clinically significant by the investigator, other than glucose, or otherwise is not stable and documented as part of the participant’s known medical history.
- Major surgery, not including gastrostomy or other enteral catheter insertions, central or peripheral catheter insertion, or similar procedures, within 3 months before screening or anticipated during the study period.
- Treatment with an investigational drug or device within 30 days or 5 half lives of the investigational drug of screening, whichever is longer. Participation in registries and purely diagnostic studies is allowed.
- Female participants who are pregnant, planning to become pregnant during the study or within 3 months after last administration of study drug, have recently delivered (within 3 months before screening), or are breastfeeding.
- Male participants who are planning a pregnancy with a female partner during study or within 3 months after last administration of study drug.
- Use of mammalian target of rapamycin (mTOR) inhibitors (e.g., sirolimus, everolimus) within 2 weeks prior to screening and during the study.
See the full eligibility criteria
Who can join
- Provide written informed consent and, as applicable, assent, before any study specific procedures are performed.
- At screening, aged ≥3 months (corrected for gestational age for children under 9 months) and ≤45 years.
- An established clinical diagnosis of congenital HI, with or without identification of a known monogenic variant by genetic testing.
- Participant has failed to achieve adequate glycemic control with appropriate and reasonable trials of locally accepted and available SOC medical therapies (e.g., diazoxide and SSAs) per the judgment of the investigator.
- Experiencing ≥3 hypoglycemia events (<70 mg/dL [<3.9 mmol/L]) per week by screening SMBG and/or according to the investigator’s evaluation, AND Average daily percent time with hypoglycemia (<70 mg/dL [<3.9 mmol/L]) of ≥8% of the monitored screening CGM time.
- Hepatic ultrasound at screening without clinically significant findings, including clinically significant gallstones as judged by the investigator (e.g., large size, obstructive, or biliary colic), or evidence of peliosis hepatitis.
- For female participants of childbearing potential*, a negative serum or urine pregnancy test within 7 days before dosing. • *Females of childbearing potential are defined as fertile following menarche and until becoming postmenopausal or permanently sterile (Postmenopausal is defined as absence of vaginal bleeding or spotting for at least 1 year. Permanently sterile is defined as having had a hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.)
- For female participants of childbearing potential, a willingness to use highly effective** contraceptive measures adequate to prevent a new pregnancy for the duration of the study, AND/including for at least 3 months after receiving the last dose of study drug. For women with reproductive potential who use a hormonal method of contraception, concurrent use of a second (barrier) method is recommended. • **Highly effective methods of birth control are defined as those that result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly (e.g., implants, injectables, oral contraceptives, some intrauterine devices, bilateral tubal occlusion, true sexual abstinence in line with the preferred and usual lifestyle of the participant, or vasectomized partner.)
- For sexually active males, a willingness to use contraceptive measures, e.g., a condom, for the duration of the study, AND/including for at least 3 months after receiving the last dose of study drug. In addition, males must agree not to donate sperm over the same study period.
What rules you out
- Participants meeting any of the following criteria will be excluded from the study: 1. Any out-of-range laboratory value at screening that is assessed as clinically significant by the investigator, other than glucose, or otherwise is not stable and documented as part of the participant’s known medical history.
- Major surgery, not including gastrostomy or other enteral catheter insertions, central or peripheral catheter insertion, or similar procedures, within 3 months before screening or anticipated during the study period.
- Treatment with an investigational drug or device within 30 days or 5 half lives of the investigational drug of screening, whichever is longer. Participation in registries and purely diagnostic studies is allowed.
- Female participants who are pregnant, planning to become pregnant during the study or within 3 months after last administration of study drug, have recently delivered (within 3 months before screening), or are breastfeeding.
- Male participants who are planning a pregnancy with a female partner during study or within 3 months after last administration of study drug.
- Use of mammalian target of rapamycin (mTOR) inhibitors (e.g., sirolimus, everolimus) within 2 weeks prior to screening and during the study.
- Initiation, reduction, or discontinuation of medications used for the chronic management of hypoglycemia (e.g., diazoxide, SSAs, continuous glucagon) within 2 weeks of screening.
- Initiation of long-acting SSAs within 3 months of screening or receipt of the most recent dose of a long-acting SSAs (e.g., octreotide long-acting release [LAR], lanreotide) ≤10 days prior to the beginning of screening glycemic evaluation with SMBG and CGM.
- Initiation or discontinuation of enteral (tube) feeding treatments or nutritional supplementation within 2 weeks of screening.
- Alanine transaminase (ALT), aspartate transaminase (AST), total bilirubin (TB), alkaline phosphatase (ALP), and gamma glutamyl transferase (GGT) ≥1.5 × the upper limit of normal (ULN) for the age-specific reference range, regardless of assessed significance.
- Body mass index (BMI) ≥35 kg/m2 for participants aged 18 years and above, or BMI ≥99% (percentile) per Centers for Disease Control and Prevention growth charts for participants >12 and <18 years of age (no BMI exclusion for participants ≤12 years of age).
- A known clinical diagnosis of diabetes or pre-diabetes, or a history of insulin dependency within 3 months of screening.
- Average daily percent time with hyperglycemia ≥250 mg/dL (≥13.9 mmol/L) ≥5% of the monitored screening CGM time.
- History of malignancy within 3 years before screening, other than carcinoma in situ of the cervix or adequately treated non-metastatic squamous or basal cell carcinoma of the skin.
- History of seropositivity (indicative of infection) for human immunodeficiency virus antibody, hepatitis B, or hepatitis C antibody (excluding immunization patterns).
- Known allergy or sensitivity to RZ358 or any component of the drug.
- Any organ condition, concomitant disease (e.g., psychiatric illness, severe alcoholism, or drug abuse, cardiac, hepatic, or kidney disease), or other abnormality that itself, or the treatment of which, could interfere with the conduct of the study (e.g., may affect absorption, distribution, metabolism, or elimination of the study drug) or that, in the opinion of the investigator and/or Sponsor’s medical monitor would pose an unacceptable risk to the participant in the study.
The study team makes the final eligibility decision.
Where it's taking place
- Georgia
- United Kingdom
- United Arab Emirates
- United States
- Vietnam
- Turkey
- Israel
- Saudi Arabia
- Australia
- Qatar
- Oman
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Georgia; United Kingdom; United Arab Emirates; United States; Vietnam; Turkey and 5 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.