An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism

Rezolute

Status and phase: Completed; Phase 2

Conditions: Congenital Hyperinsulinism

Treatments: Drug: RZ358 Sequential Group Cohort 3; Drug: RZ358 Sequential Group Cohort 4; Drug: RZ358 Sequential Group Cohort 2; Drug: RZ358 Sequential Group Cohort 1

Study type: Interventional

Funder types: Other

Identifiers

NCT04538989

RZ358-606

Details and patient eligibility

About

The objective of this trial is to evaluate the safety, tolerability and glucose-raising effects of RZ358 in patients with Congenital Hyperinsulinism (HI).

Full description

There is a significant unmet medical need to develop new therapies aimed at preventing chronic recurrent hypoglycemia in congenital HI, the most common cause of persistent hypoglycemia in children. RZ358 is a human mAb that allosterically attenuates excessive insulin action on target cells. Therefore, RZ358 is ideally suited as a potential therapy for hyperinsulinism, and it is being developed to treat the hypoglycemia associated with diseases such as congenital HI. This is a Phase 2, multicenter, open label clinical study designed to assess the safety and efficacy of four progressively higher doses of RZ358 in separate groups of patients with hyperinsulinemic hypoglycemia due to Congenital HI, not adequately controlled with or without current standard of care. A screening period of up to 5 weeks will evaluate eligibility. Once enrolled, RZ358 will be administered bi-weekly over 8 weeks, and then patients will complete a post-treatment follow-up period of 13 weeks.

Enrollment: 23 patients

Sex: All

Ages: 2 to 45 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female age 2-45 years old (except age 12-45 in US) with an established clinical diagnosis of congenital hyperinsulinism
• Able to provide written informed consent or, as applicable, assent
• Confirmed hypoglycemia as assessed by CGM, SMBG, and clinical evaluation, during Screening
• Willingness to use contraception if of child-bearing potential

Exclusion criteria

• Out of range blood work for study entry
• Body Mass index outside of study entry criteria
• History of malignancy
• Clinically significant diseases, seropositivity for HIV, hepatitis B or C antibody
• Use of systemic corticosteroids within 30 days before Screening
• Known or suspected allergy to the study drug
• Recent use of an investigational drug or treatment, or participation in an investigational study
• Pregnant or lactating women
• History of drug abuse or excessive alcohol use

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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23 participants in 4 patient groups

RZ358 Cohort 1

Experimental group

Treatment:

Drug: RZ358 Sequential Group Cohort 1

RZ358 Cohort 2

Experimental group

Treatment:

Drug: RZ358 Sequential Group Cohort 2

RZ358 Cohort 3

Experimental group

Treatment:

Drug: RZ358 Sequential Group Cohort 3

RZ358 Cohort 4

Experimental group

Treatment:

Drug: RZ358 Sequential Group Cohort 4