RecruitingPhase 1Phase 2NCT05478720

DON in Pediatric Cerebral Malaria

DON in Pediatric Cerebral Malaria: A Phase I/IIa Dose-Escalation Safety Study


Sponsor

Douglas Postels, MD, MS

Enrollment

152 participants

Start Date

Aug 16, 2022

Study Type

INTERVENTIONAL

Conditions

Summary

The goal of this clinical trial is to evaluate the safety of a single intravenous dose of DON in healthy adults, adults with uncomplicated malaria, and children 12 months-14 years old with clinically defined Cerebral Malaria. The main objectives are: * Determine the pharmacokinetic (PK) profile of a single dose of DON in children with CM * Determine if administration of a single intravenous dose of DON as an adjunctive therapy in children with CM is associated with improved intracerebral blood flow dynamics on transcranial doppler (TCD) * Determine if administration of a single intravenous dose of DON as an adjunctive therapy in children with CM is associated with a reduction in brain volume score on magnetic resonance imaging (MRI) * Determine if administration of a single intravenous dose of DON as an adjunctive therapy in children with cerebral malaria is associated with changes in electroencephalogram (EEG) pattern * Exploratory: Explore the metabolic mechanisms of action of adjunctive DON in children with CM Healthy adult participants will receive: * anti-emetic ondansetron * one dose of DON Adults with uncomplicated malaria will receive: * anti-emetic ondansetron * one dose of DON * artemisinin-combination therapies per Malawi Ministry of Health guidelines Pediatric participants will receive: * one dose of DON * anti-emetic ondansetron and per Malawi Ministry of Health guidelines: * enteral lumefantrine-artemether therapy, and * artesunate therapy


Eligibility

Min Age: 12 Months

Inclusion Criteria33

  • For Healthy Adults (Arm 1):
  • years and older
  • Informed consent obtained and ICF signed
  • Temperature ≤ 37.5 °C
  • BMI 18.5-25 kg/m2
  • Creatinine ≤ 110 mmol/L (≤ 1.2 mg/dL; males) or ≤ 90 mmol/L (≤ 1.0 mg/dL; females)
  • Hemoglobin ≥ 7 g/dL or hematocrit/ packed-cell volume (PCV) ≥ 20%
  • Thick or thin blood smear negative for asexual forms of P. falciparum
  • Negative pregnancy test for persons of child-bearing potential
  • For Adults with Uncomplicated Malaria (Arm 2):
  • years and older
  • Informed consent obtained and ICF signed
  • Temperature ≥ 38 °C or history of fever in the past 24 hours
  • Thick or thin blood smear positive for asexual forms of P. falciparum (parasite count and speciation documented)
  • Hemoglobin ≥ 7 g/dL or hematocrit/ PCV ≥ 20%
  • BMI 18.5-25 kg/m2
  • Creatinine ≤ 110 mmol/L (≤ 1.2 mg/dL; males) or ≤ 90 mmol/L (≤ 1.0 mg/dL; females)
  • Glasgow coma score of 15
  • Respiratory rate ≤ 20 breaths/ minute
  • Oxygen saturation ≥ 90% on room air
  • Negative pregnancy test for person of child-bearing potential
  • For Children with Cerebral Malaria (Arm 3):
  • Age 12 months-14 years old
  • Informed consent obtained and ICF signed by parent or guardian
  • Temperature ≥ 38 °C or history of fever in the last 24 hours
  • Thick or thin blood smear positive for asexual forms of P. falciparum
  • Blantyre coma score ≤ 2
  • No other explanation for coma by history or physical exam
  • Hematocrit or PCV ≥ 18%
  • Negative pregnancy test for persons of child-bearing potential
  • Creatinine ≤ 1.5 mg/dL
  • Aspartate aminotransferase (AST) < 280 IU/L
  • Alanine aminotransferase (ALT) < 195 IU/L

Exclusion Criteria10

  • Pregnancy or lactation (participants of child-bearing potential ages 9-59 years will undergo pregnancy testing prior to administration of the intervention)
  • Participants attempting to become pregnant
  • Currently taking highly active antiretroviral therapy (HAART)
  • Currently taking anti-tuberculosis medications
  • Allergy to ondansetron
  • Cloudy cerebrospinal fluid (indicative of a probable bacterial central nervous system infection)
  • Malnutrition defined as a more than or equal to two standard deviations below the mean weight for height and/ or MUAC ≤ 12.5 cm (due to inability to adequately care for children with severe malnutrition on the PRW)
  • Allergy to ondansetron or ceftriaxone
  • Coma for > 72 hours
  • Have taken a CYP3A4 inhibitor within 7 days of enrollment

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Interventions

DRUG6-diazo-5-oxo-L-norleucine (DON)

Single intravenous dose ranging from 0.1-10 mg/kg per dose

DRUGPlacebo

Single intravenous dose of saline

DRUG6-diazo-5-oxo-L-norleucine (DON)

Single intravenous dose ranging from 0.1-1.0 mg/kg per dose


Locations(2)

Ndirande Research Clinic

Blantyre, Malawi

Queen Elizabeth Central Hospital

Blantyre, Malawi

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NCT05478720