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Trial record 1 of 1 for:    Rezolute
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An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism

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ClinicalTrials.gov Identifier: NCT04538989
Recruitment Status : Completed
First Posted : September 4, 2020
Last Update Posted : September 8, 2022
Sponsor:
Information provided by (Responsible Party):
Rezolute

Tracking Information
First Submitted Date  ICMJE August 21, 2020
First Posted Date  ICMJE September 4, 2020
Last Update Posted Date September 8, 2022
Actual Study Start Date  ICMJE February 24, 2020
Actual Primary Completion Date April 5, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 15, 2021)
  • Glycemic efficacy: Target glucose control [ Time Frame: 8 weeks ]
    Change from Baseline in Percent Time in Glucose Target Range by Continuous Glucose Monitor (CGM)
  • Repeat dose safety and tolerability of RZ358 [ Time Frame: Through 21 Weeks ]
    Occurrence of a safety signal as assessed by the incidence of treatment-emergent AEs, SAEs, and AEs leading to study drug discontinuation.
  • Repeat dose pharmacokinetics of RZ358 [ Time Frame: Through 8 weeks ]
    Change from baseline in RZ358 drug exposure as assessed by Population-PK modeling of maximum concentrations (Cmax) and Area under the Concentration-Time Curve (AUC).
Original Primary Outcome Measures  ICMJE
 (submitted: August 31, 2020)
  • Glycemic efficacy: Target glucose control [ Time Frame: 8 weeks ]
    Change from Baseline in Percent Time in Glucose Target Range by Continuous Glucose Monitor (CGM)
  • Glycemic efficacy: Target glucose control [ Time Frame: 4 weeks ]
    Change from Baseline in Percent Time in Glucose Target Range by Continuous Glucose Monitor (CGM)
  • Repeat dose safety and tolerability of RZ358 [ Time Frame: Through 27 Weeks ]
    Occurrence of a safety signal as assessed by the incidence of treatment-emergent AEs, SAEs, and AEs leading to study drug discontinuation.
  • Repeat dose pharmacokinetics of RZ358 [ Time Frame: Through 8 weeks ]
    Change from baseline in RZ358 drug exposure as assessed by Population-PK modeling of maximum concentrations (Cmax) and Area under the Concentration-Time Curve (AUC).
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 31, 2020)
  • Glycemic efficacy: Occurrence of hypoglycemia [ Time Frame: 8 weeks ]
    Change from Baseline in the Incidence of Hypoglycemia by Self-Monitored Blood Glucose (SMBG)
  • Glycemic efficacy: Duration of Hypoglycemia [ Time Frame: 8 weeks ]
    Change from Baseline in the Absolute (minutes) and Percent Time with Hypoglycemia by CGM
  • Glycemic efficacy: Occurrence of Hypoglycemia [ Time Frame: 8 weeks ]
    Change from Baseline in the Incidence of Hypoglycemia by CGM
  • Overnight Target Glucose Control [ Time Frame: 8 weeks ]
    Change from Baseline in Percent Time in Overnight (midnight to 8 am) Glucose Target Range by CGM
  • Glycemic Efficacy: Ability to Complete a Fast without Hypoglycemia [ Time Frame: 8 weeks ]
    Change from Baseline in Incidence of Hypoglycemia by SMBG, During a 12-hour Fasting Challenge
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Open-Label Multiple Dose Study of RZ358 in Patients With Congenital Hyperinsulinism
Official Title  ICMJE An Open-Label Multiple-Dose Study of RZ358 in Patients With Congenital Hyperinsulinism
Brief Summary The objective of this trial is to evaluate the safety, tolerability and glucose-raising effects of RZ358 in patients with Congenital Hyperinsulinism (HI).
Detailed 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Congenital Hyperinsulinism
Intervention  ICMJE
  • Drug: RZ358 Sequential Group Cohort 1
    IV infusion for 8 weeks (3 mg/kg bi-weekly for 8 weeks)
  • Drug: RZ358 Sequential Group Cohort 2
    IV infusion for 8 weeks (6 mg/kg bi-weekly for 8 weeks)
  • Drug: RZ358 Sequential Group Cohort 3
    IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks)
  • Drug: RZ358 Sequential Group Cohort 4
    IV infusion for 8 weeks (bi-weekly fixed dose-titration from 3 to 9 mg/kg for the first 4 weeks, followed by a fixed 9 mg/kg dose amount thereafter for the remaining 4 weeks)
Study Arms  ICMJE
  • Experimental: RZ358 Cohort 1
    Intervention: Drug: RZ358 Sequential Group Cohort 1
  • Experimental: RZ358 Cohort 2
    Intervention: Drug: RZ358 Sequential Group Cohort 2
  • Experimental: RZ358 Cohort 3
    Intervention: Drug: RZ358 Sequential Group Cohort 3
  • Experimental: RZ358 Cohort 4
    Intervention: Drug: RZ358 Sequential Group Cohort 4
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 7, 2022)
23
Original Estimated Enrollment  ICMJE
 (submitted: August 31, 2020)
32
Actual Study Completion Date  ICMJE August 19, 2022
Actual Primary Completion Date April 5, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 45 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Bulgaria,   Canada,   Denmark,   Georgia,   Germany,   Israel,   Russian Federation,   Spain,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04538989
Other Study ID Numbers  ICMJE RZ358-606
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Rezolute
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Rezolute
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Rezolute
Verification Date June 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP