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The objective of this trial is to evaluate the safety, tolerability and glucose-raising effects of RZ358 in patients with Congenital Hyperinsulinism (HI).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| RZ358 Cohort 1 | Experimental |
| |
| RZ358 Cohort 2 | Experimental |
| |
| RZ358 Cohort 3 | Experimental |
| |
| RZ358 Cohort 4 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| RZ358 Sequential Group Cohort 1 | Drug | IV infusion for 8 weeks (3 mg/kg bi-weekly for 8 weeks) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Median of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average daily percent time within the glucose target range 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline and End of Treatment (EOT) is reported. | 8 weeks |
| Median Percent Change of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM From Baseline (BL) | The average daily percent time within the glucose target range (70-180 mg/dL) is compared from baseline to end of treatment (EOT) and the median percent change of that difference is reported. | 8 weeks |
| Repeat Dose Pharmacokinetics of RZ358 | All patients who received RZ358 and for whom the primary PK data was considered to be sufficient and interpretable were to be included in the PK analyses. Individual and mean plasma concentration data is summarized descriptively at the specified timepoints. The results of this study may be combined with those of other studies for analysis and modeling (e.g., population PK and PK-PD), and therefore the PK parameters are reported separately, as part of an iterative population PK approach. | Pre dose Weeks 1,3,5,7, 1-hr post dose Week 1 and Week 7, and Follow up on Days 14, Day 28, Day 42, and Day 105 |
| Measure | Description | Time Frame |
|---|---|---|
| Median of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG at Baseline (BL) and End of Treatment (EOT) | The median of average weekly number of overall hypoglycemia events (<70 mg/dL [3.9mmol/L], moderate hypoglycemia events (<60 mg/dL [3.3 mmol/L] and severe hypoglycemia events (<50 mg/dL [2.8mmol/L]) by SMBG at baseline (BL) and end of treatment (EOT) is reported. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States | ||
| Cook Children's Medical Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40107271 | Derived | Demirbilek H, Melikyan M, Iotova V, Galcheva S, Ozbek MN, Dastamani A, Kheladze N, Mazor-Aronovitch K, Clemente M, Empting S, Mohnike K, Christesen HT, Thornton PS, De Leon DD, Hood D, O'Boyle E, Roberts BK. Global, multi-center, repeat-dose, phase 2 study of RZ358 (ersodetug), an insulin receptor antibody, for congenital hyperinsulinism. Med. 2025 Jun 13;6(6):100611. doi: 10.1016/j.medj.2025.100611. Epub 2025 Mar 18. |
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NA; open-label study
Sites were recruited based on cHI professional expertise and sufficient patient panels and advocacy referrals.
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| ID | Title | Description |
|---|---|---|
| FG000 | RZ358 Cohort 1 | RZ358 Sequential Group Cohort 1: IV infusion for 8 weeks (3 mg/kg bi-weekly for 8 weeks) |
| FG001 | RZ358 Cohort 2 | RZ358 Sequential Group Cohort 2: IV infusion for 8 weeks (6 mg/kg bi-weekly for 8 weeks) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 15, 2021 | Aug 27, 2024 |
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| RZ358 Sequential Group Cohort 2 | Drug | IV infusion for 8 weeks (6 mg/kg bi-weekly for 8 weeks) |
|
| RZ358 Sequential Group Cohort 3 | Drug | IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks) |
|
| RZ358 Sequential Group Cohort 4 | Drug | 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) |
|
| 8 weeks |
| Median Percent Change of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG From Baseline (BL) | The average weekly number overall hypoglycemia events (<70 mg/dL [3.9mmol/L], moderate hypoglycemia events (<60 mg/dL [3.3 mmol/L] and severe hypoglycemia events (<50 mg/dL [2.8mmol/L]) by SMBG is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported. | 8 weeks |
| Median of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average daily percent time in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L]), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia at baseline (BL) and end of treatment (EOT) is reported. | 8 weeks |
| Median Percent Change of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) | The average daily percent time in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L]), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported. | 8 weeks |
| Median of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average duration (minutes) in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L] ), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia by CGM at baseline (BL) and end of treatment (EOT) is reported. | 8 weeks |
| Median Percent Change of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) | The average duration (minutes) in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L] ), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia by CGM is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported. | 8 weeks |
| Occurrence of Hypoglycemia During Fasting Challenge | The occurrence of hypoglycemia during a 12 Hour fasting challenge. | 8 Weeks |
| Median of Average Hypoglycemia Events Per Day at Each of the Specified Glucose Thresholds by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average hypoglycemia events per day at each of the specified glucose thresholds [<70, <60, and <50 mg/dL (3.9, 3.3, and 2.8 mmol/L)], by CGM at baseline (BL) and end of treatment (EOT) is reported. | 8 weeks |
| Median Net Change of Average Hypoglycemia Events Per Day at Each of the Specified Glucose Thresholds by CGM From Baseline (BL) | The average hypoglycemia events per day at each of the specified glucose thresholds [<70, <60, and <50 mg/dL (3.9, 3.3, and 2.8 mmol/L)], by CGM is compared from baseline (BL) to end of treatment (EOT) and the median net changes of those differences are reported. | 8 weeks |
| Median of Average 8-hour Overnight Percent Time in Glucose Target Range of 70-180mg/dL by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average 8-hour overnight (12am-8am) percent time within the glucose target range (70-180 mg/dL) at baseline (BL) and end of treatment (EOT) is reported. | 8 weeks |
| Median Percent Change of Average 8-hour Overnight Percent Time in Glucose Target Range of 70-180mg/dL by CGM From Baseline (BL) | The average 8-hour overnight (12am-8am) percent time within the glucose target range (70-180 mg/dL) is compared from baseline (BL) to end of treeatment (EOT) and the median percent change of that difference is reported. | 8 weeks |
| Fort Worth |
| Texas |
| 76104 |
| United States |
| SHAT Children diseases "Prof. Dr. Ivan Mitov" | Sofia | Bulgaria |
| Medical University of Varna UMHAT "St. Marina" | Varna | 9010 | Bulgaria |
| Research Institute of the McGill University Health Centre | Monteral | Qubec | H4A 3J1 | Canada |
| Odense University Hospital | Odense | 5000 | Denmark |
| LTD "Pediatric Surgery Centre" | Tbilisi | 0122 | Georgia |
| Magdeburg University Clinic Center (Otto-von-Guericke Universität) | Magdeburg | 39120 | Germany |
| Edmond & Lilly Safra's Children Hospital | Ramat Gan | Tel-Hashomer | 5265601 | Israel |
| Hadassah Har Hazofim MC - Division of Pediatric Endocrinology | Jerusalem | 90000 | Israel |
| Endocrinology research center | Moscow | 117036 | Russia |
| Hospital Universitari Vall d' Hebron | Barcelona | 08035 | Spain |
| Adana Cukurova University Balcalı Hospital | Sarıçam | Adana | Turkey (Türkiye) |
| Hacettepe University | Çankaya | Ankara | 06800 | Turkey (Türkiye) |
| SBÜ Gazi Yaşargil Eğitim ve Araştirma Hastanesi | Kayapınar | Diyarbakır | 21070 | Turkey (Türkiye) |
| Erzurum City Hospital | Yakutiye | Erzurum | Turkey (Türkiye) |
| Great Ormond Street Hospital | London | United Kingdom |
| FG002 | RZ358 Cohort 3 | RZ358 Sequential Group Cohort 3: IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks) |
| FG003 | RZ358 Cohort 4 | 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) |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | RZ358 Cohort 1 | RZ358 Sequential Group Cohort 1: IV infusion for 8 weeks (3 mg/kg bi-weekly for 8 weeks) |
| BG001 | RZ358 Cohort 2 | RZ358 Sequential Group Cohort 2: IV infusion for 8 weeks (6 mg/kg bi-weekly for 8 weeks) |
| BG002 | RZ358 Cohort 3 | RZ358 Sequential Group Cohort 3: IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks) |
| BG003 | RZ358 Cohort 4 | 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) |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Median of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average daily percent time within the glucose target range 70-180 mg/dL (3.9-10 mmol/L) by CGM at Baseline and End of Treatment (EOT) is reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Daily Percent Time | 8 weeks |
|
|
| |||||||||||||||||||||||||||||||||||||
| Primary | Median Percent Change of Average Daily Percent Time Within a Glucose Target Range of 70-180 mg/dL (3.9-10 mmol/L) by CGM From Baseline (BL) | The average daily percent time within the glucose target range (70-180 mg/dL) is compared from baseline to end of treatment (EOT) and the median percent change of that difference is reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Percent Change | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Primary | Repeat Dose Pharmacokinetics of RZ358 | All patients who received RZ358 and for whom the primary PK data was considered to be sufficient and interpretable were to be included in the PK analyses. Individual and mean plasma concentration data is summarized descriptively at the specified timepoints. The results of this study may be combined with those of other studies for analysis and modeling (e.g., population PK and PK-PD), and therefore the PK parameters are reported separately, as part of an iterative population PK approach. | PK Population; Data from Cohort 1, 2 and 4 was not collected during Pre-dose Week 1. | Posted | Mean | Full Range | ng/mL | Pre dose Weeks 1,3,5,7, 1-hr post dose Week 1 and Week 7, and Follow up on Days 14, Day 28, Day 42, and Day 105 |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG at Baseline (BL) and End of Treatment (EOT) | The median of average weekly number of overall hypoglycemia events (<70 mg/dL [3.9mmol/L], moderate hypoglycemia events (<60 mg/dL [3.3 mmol/L] and severe hypoglycemia events (<50 mg/dL [2.8mmol/L]) by SMBG at baseline (BL) and end of treatment (EOT) is reported. | Per Protocol Population; For efficacy outcomes, the PP Population included 20 participants for BGM-based glycemic endpoints due to endpoint-specific exclusions. One participant in Cohort 1 and one in Cohort 4 were excluded from the PP population BGM-based analyses because they failed to meet pre-specified minimum testing frequency by glucometer. One participant in Cohort 3 was also excluded from the PP population BGM analyses for stopping background SOC therapy after starting RZ358 in the study | Posted | Median | 95% Confidence Interval | Hypoglycemia Events | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median Percent Change of Average Weekly Overall, Moderate, and Severe Hypoglycemia Events by SMBG From Baseline (BL) | The average weekly number overall hypoglycemia events (<70 mg/dL [3.9mmol/L], moderate hypoglycemia events (<60 mg/dL [3.3 mmol/L] and severe hypoglycemia events (<50 mg/dL [2.8mmol/L]) by SMBG is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported. | Per Protocol Population; For efficacy outcomes, the PP Population included 20 participants for BGM-based glycemic endpoints due to endpoint-specific exclusions. One participant in Cohort 1 and one in Cohort 4 were excluded from the PP population BGM-based analyses because they failed to meet pre-specified minimum testing frequency by glucometer. One participant in Cohort 3 was also excluded from the PP population BGM analyses for stopping background SOC therapy after starting RZ358 in the study | Posted | Median | 95% Confidence Interval | Percent Change | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average daily percent time in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L]), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia at baseline (BL) and end of treatment (EOT) is reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Daily Percent Time | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median Percent Change of Average Daily Percent Time With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) | The average daily percent time in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L]), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Percent Change | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average duration (minutes) in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L] ), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia by CGM at baseline (BL) and end of treatment (EOT) is reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Daily Minutes in Hypoglycemia | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median Percent Change of Average Daily Duration (Minutes) With Overall, Moderate, and Severe Hypoglycemia by CGM From Baseline (BL) | The average duration (minutes) in overall (<70 mg/dL [<3.9 mmol/L]), moderate (<60 mg/dL [3.3 mmol/L] ), and severe (<50 mg/dL [2.8 mmol/L]) hypoglycemia by CGM is compared from baseline (BL) to end of treatment (EOT) and the median percent changes of those differences are reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Percent Change | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Occurrence of Hypoglycemia During Fasting Challenge | The occurrence of hypoglycemia during a 12 Hour fasting challenge. | Per Protocol Population | Posted | Number | participants | 8 Weeks |
| ||||||||||||||||||||||||||||||||||||||||
| Secondary | Median of Average Hypoglycemia Events Per Day at Each of the Specified Glucose Thresholds by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average hypoglycemia events per day at each of the specified glucose thresholds [<70, <60, and <50 mg/dL (3.9, 3.3, and 2.8 mmol/L)], by CGM at baseline (BL) and end of treatment (EOT) is reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Hypoglycemia Events Per Day | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median Net Change of Average Hypoglycemia Events Per Day at Each of the Specified Glucose Thresholds by CGM From Baseline (BL) | The average hypoglycemia events per day at each of the specified glucose thresholds [<70, <60, and <50 mg/dL (3.9, 3.3, and 2.8 mmol/L)], by CGM is compared from baseline (BL) to end of treatment (EOT) and the median net changes of those differences are reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Events Per Day | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median of Average 8-hour Overnight Percent Time in Glucose Target Range of 70-180mg/dL by CGM at Baseline (BL) and End of Treatment (EOT) | The median of average 8-hour overnight (12am-8am) percent time within the glucose target range (70-180 mg/dL) at baseline (BL) and end of treatment (EOT) is reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | 8-hour Overnight Percent Time | 8 weeks |
| |||||||||||||||||||||||||||||||||||||||
| Secondary | Median Percent Change of Average 8-hour Overnight Percent Time in Glucose Target Range of 70-180mg/dL by CGM From Baseline (BL) | The average 8-hour overnight (12am-8am) percent time within the glucose target range (70-180 mg/dL) is compared from baseline (BL) to end of treeatment (EOT) and the median percent change of that difference is reported. | Per Protocol Population | Posted | Median | 95% Confidence Interval | Percent Change | 8 weeks |
|
8 weeks
Any untoward medical occurrence [i.e., any unfavorable and unintended sign (including abnormal lab findings), symptom or disease] which is either new or pre-existing but worsened in severity or frequency, whether or not it has a causal relationship to the study investigational (medicinal) products or procedures. Includes events w/ onset from the time of providing written informed consent until the EOS visit. AE may or may not be temporally or causally associated w/ the use of a study drug.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | RZ358 Cohort 1 | RZ358 Sequential Group Cohort 1: IV infusion for 8 weeks (3 mg/kg bi-weekly for 8 weeks) | 0 | 4 | 0 | 4 | 2 | 4 |
| EG001 | RZ358 Cohort 2 | RZ358 Sequential Group Cohort 2: IV infusion for 8 weeks (6 mg/kg bi-weekly for 8 weeks) | 0 | 8 | 2 | 8 | 7 | 8 |
| EG002 | RZ358 Cohort 3 | RZ358 Sequential Group Cohort 3: IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks) | 0 | 8 | 1 | 8 | 5 | 8 |
| EG003 | RZ358 Cohort 4 | 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) | 0 | 3 | 0 | 3 | 2 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypoglycemia with LOC | Endocrine disorders | Systematic Assessment | Unrelated to the study drug. Patient had a medical history of hypoglycemia with LOC due to her background congenital HI. |
| |
| Tonic-Clonic Seizure | Nervous system disorders | Systematic Assessment | Unrelated to study drug. Patient had a medical history of seizures. |
| |
| Eosinophilic Leukocytosis | Blood and lymphatic system disorders | Systematic Assessment | Unrelated to study drug. Patient had medical history of pre-existing eosinophilia predominant leukocytosis. |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Phlebitis | Vascular disorders | Non-systematic Assessment |
| ||
| Extravasation | General disorders | Non-systematic Assessment |
| ||
| Fatigue | General disorders | Non-systematic Assessment |
| ||
| Infusion Site Reaction | General disorders | Non-systematic Assessment |
| ||
| Medical Device Pain | General disorders | Non-systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
| ||
| Respiratory Disorder | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
| ||
| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
| ||
| Arthropod sting | Injury, poisoning and procedural complications | Non-systematic Assessment |
| ||
| Contusion | Injury, poisoning and procedural complications | Non-systematic Assessment |
| ||
| Dizziness | Nervous system disorders | Non-systematic Assessment |
| ||
| Generalized tonic-clonic seizure | Nervous system disorders | Non-systematic Assessment |
| ||
| Headache | Nervous system disorders | Non-systematic Assessment |
| ||
| Paraesthesia | Nervous system disorders | Non-systematic Assessment |
| ||
| Psychomotor Hyperactivity | Nervous system disorders | Non-systematic Assessment |
| ||
| Presyncope | Nervous system disorders | Non-systematic Assessment |
| ||
| Anaemia | Blood and lymphatic system disorders | Non-systematic Assessment |
| ||
| Leukocytosis | Blood and lymphatic system disorders | Non-systematic Assessment |
| ||
| Chalazion | Eye disorders | Non-systematic Assessment |
| ||
| Upper Abdominal Pain | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| Acanthosis | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Dermatitis Contact | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Urticaria | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Leukocyturia | Renal and urinary disorders | Non-systematic Assessment |
| ||
| Pain in extremity | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
| ||
| Sacral Pain | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
| ||
| Covid-19 | Infections and infestations | Non-systematic Assessment |
| ||
| Conjunctivitis | Infections and infestations | Non-systematic Assessment |
| ||
| Influenza | Infections and infestations | Non-systematic Assessment |
| ||
| Otitis Media | Infections and infestations | Non-systematic Assessment |
| ||
| Respiratory tract infection, viral | Infections and infestations | Non-systematic Assessment |
| ||
| Upper Respiratory Infection | Infections and infestations | Non-systematic Assessment |
| ||
| Hypoglycemia | Endocrine disorders | Non-systematic Assessment |
| ||
| Hypoglycemia | Metabolism and nutrition disorders | Non-systematic Assessment |
|
Limitations include a small sample size (low statistical power), an open-label design (potential for bias due to lack of blinding), a short treatment period in a chronic disease, and a lack of ethnic diversity in study participants who have a rare pediatric disease. Additionally, the COVID-19 pandemic impacted the study by causing a delay in projected enrollment and study timelines. However, due to excess precaution taken, there was no impact on the overall execution/outcomes of the study.
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer (Dr. Brian Roberts) | Rezolute | 650-206-4507 | brian@rezolutebio.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 17, 2022 | Aug 27, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D044903 | Congenital Hyperinsulinism |
| D007003 | Hypoglycemia |
| D044882 | Glucose Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D006946 | Hyperinsulinism |
| ID | Term |
|---|---|
| D007232 | Infant, Newborn, Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| United States |
|
| Denmark |
|
| United Kingdom |
|
| Georgia |
|
| Israel |
|
| Bulgaria |
|
| Germany |
|
| Russia |
|
| Spain |
|
| Median of Average Daily Percent Time in 70-180 mg/dL at 8 weeks (EOT) |
|
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
| OG003 |
| RZ358 Cohort 4 |
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) |
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
RZ358 Sequential Group Cohort 3: IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks)
| OG003 | RZ358 Cohort 4 | 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) |
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
RZ358 Sequential Group Cohort 3: IV infusion for 8 weeks (9 mg/kg bi-weekly for 8 weeks) |
| OG003 | RZ358 Cohort 4 | 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) |
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
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| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
RZ358 Total Participants |
|
|
| OG004 | RZ358 Pooled | RZ358 Total Participants |
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|
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
| OG004 | RZ358 Pooled | RZ358 Total Participants |
|
|
| OG004 | RZ358 Pooled | RZ358 Total Participants |
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