search
Back to results

Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study

Primary Purpose

Human African Trypanosomiasis (HAT)

Status
Completed
Phase
Phase 2
Locations
Congo, The Democratic Republic of the
Study Type
Interventional
Intervention
fexinidazole
Sponsored by
Drugs for Neglected Diseases
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Human African Trypanosomiasis (HAT)

Eligibility Criteria

6 Years - 14 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Informed consent form signed by one of the parents or the child's legal representative
  • Child assent to participate in the study collected in the presence of an impartial witness
  • Age between 6 and 15 years old
  • > 20 Kg bodyweight
  • Boy or girl
  • Able to ingest at least one complete meal per day (or at least one Plumpy'Nut® sachet)
  • Able to swallow 600mg fexinidazole tablets
  • Karnofsky index > 50
  • Presence of trypanosomes in blood and/or lymph and/or CSF
  • Permanent address and ability to comply with follow-up visit schedule

Exclusion Criteria:

  • Child refusing to be included in the trial
  • Bodyweight < 20 Kg
  • Severe malnutrition, defined as having a BMI below 16 (- 2 Z-score of the norm according to WHO 2007 Growth reference data)
  • Inability to take oral medication
  • Pregnancy or breastfeeding
  • Clinically relevant medical condition other than HAT that, in the Investigator's opinion, may jeopardize subject safety or interfere with participation in the study, including but not limited to significant liver or cardiovascular disease, active documented or suspected infection, central nervous system (CNS) trauma or seizure disorders, coma or altered consciousness
  • Severely deteriorated general condition, such as cardiovascular shock, respiratory distress, or terminal illness
  • Any medical condition (except HAT-specific symptoms) hindering communication with the Investigator as required for the completion of this study
  • Any contraindication to imidazole products (known hypersensitivity to imidazoles)
  • History of HAT treatment in the past 2 years
  • Patients previously enrolled in the study or having already received fexinidazole.
  • Expected follow-up difficulties (migrants, refugees, itinerant vendors, etc.).
  • Current alcohol or drug abuse
  • Clinically significant abnormal laboratory findings, including ASAT and/or ALAT > 2 times ULN // Total bilirubin > 1.5 times ULN // Severe leukopenia (< 2000/mm3) // Potassium (K+) < 3.5 mmol // Any other clinically significant abnormal laboratory value (see details in Investigator Manual)
  • Pregnancy confirmed by a positive urine pregnancy test obtained within 24 hours (h) prior to start of study treatment (see Section 5.8.3 Contraception; p36) for girls over 12 years old and over
  • ECG abnormalities assessed by a central cardiologist
  • QTcF≥ 450 ms, as measured automatically (if first measurement is abnormal, a second assessment will be done at least 10-20 min later, with the patient in resting position).
  • Patients not tested for malaria and/or not treated adequately for this infection
  • Patients not treated adequately for soil transmitted helminthic diseases

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Fexinidazole

Arm Description

Outcomes

Primary Outcome Measures

Outcome (success or failure) at the test of cure (ToC)
Success at 12 months, adapted from the WHO criteria (51), is defined as a cured patient: Patient alive; AND no evidence of trypanosomes in any body fluid; AND WBC ≤20/µL in CSF.

Secondary Outcome Measures

Full Information

First Posted
June 19, 2014
Last Updated
June 23, 2020
Sponsor
Drugs for Neglected Diseases
search

1. Study Identification

Unique Protocol Identification Number
NCT02184689
Brief Title
Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study
Official Title
Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study
Study Type
Interventional

2. Study Status

Record Verification Date
June 2020
Overall Recruitment Status
Completed
Study Start Date
May 3, 2014 (Actual)
Primary Completion Date
December 14, 2016 (Actual)
Study Completion Date
June 27, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Drugs for Neglected Diseases

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The aim of the study is to assess the efficacy and safety of an oral regimen of fexinidazole (once daily for 10 days) in the treatment of stage 1 and stage 2 T.b. gambiense sleeping sickness in children at least 6 years old and over 20 kg bodyweight.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Human African Trypanosomiasis (HAT)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
125 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Fexinidazole
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
fexinidazole
Primary Outcome Measure Information:
Title
Outcome (success or failure) at the test of cure (ToC)
Description
Success at 12 months, adapted from the WHO criteria (51), is defined as a cured patient: Patient alive; AND no evidence of trypanosomes in any body fluid; AND WBC ≤20/µL in CSF.
Time Frame
12 months after the end of treatment

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Informed consent form signed by one of the parents or the child's legal representative Child assent to participate in the study collected in the presence of an impartial witness Age between 6 and 15 years old > 20 Kg bodyweight Boy or girl Able to ingest at least one complete meal per day (or at least one Plumpy'Nut® sachet) Able to swallow 600mg fexinidazole tablets Karnofsky index > 50 Presence of trypanosomes in blood and/or lymph and/or CSF Permanent address and ability to comply with follow-up visit schedule Exclusion Criteria: Child refusing to be included in the trial Bodyweight < 20 Kg Severe malnutrition, defined as having a BMI below 16 (- 2 Z-score of the norm according to WHO 2007 Growth reference data) Inability to take oral medication Pregnancy or breastfeeding Clinically relevant medical condition other than HAT that, in the Investigator's opinion, may jeopardize subject safety or interfere with participation in the study, including but not limited to significant liver or cardiovascular disease, active documented or suspected infection, central nervous system (CNS) trauma or seizure disorders, coma or altered consciousness Severely deteriorated general condition, such as cardiovascular shock, respiratory distress, or terminal illness Any medical condition (except HAT-specific symptoms) hindering communication with the Investigator as required for the completion of this study Any contraindication to imidazole products (known hypersensitivity to imidazoles) History of HAT treatment in the past 2 years Patients previously enrolled in the study or having already received fexinidazole. Expected follow-up difficulties (migrants, refugees, itinerant vendors, etc.). Current alcohol or drug abuse Clinically significant abnormal laboratory findings, including ASAT and/or ALAT > 2 times ULN // Total bilirubin > 1.5 times ULN // Severe leukopenia (< 2000/mm3) // Potassium (K+) < 3.5 mmol // Any other clinically significant abnormal laboratory value (see details in Investigator Manual) Pregnancy confirmed by a positive urine pregnancy test obtained within 24 hours (h) prior to start of study treatment (see Section 5.8.3 Contraception; p36) for girls over 12 years old and over ECG abnormalities assessed by a central cardiologist QTcF≥ 450 ms, as measured automatically (if first measurement is abnormal, a second assessment will be done at least 10-20 min later, with the patient in resting position). Patients not tested for malaria and/or not treated adequately for this infection Patients not treated adequately for soil transmitted helminthic diseases
Facility Information:
City
Kinshasa
Country
Congo, The Democratic Republic of the

12. IPD Sharing Statement

Citations:
PubMed Identifier
36179736
Citation
Kande Betu Kumesu V, Mutombo Kalonji W, Bardonneau C, Valverde Mordt O, Ngolo Tete D, Blesson S, Simon F, Delhomme S, Bernhard S, Nganzobo Ngima P, Mahenzi Mbembo H, Fina Lubaki JP, Lumeya Vuvu S, Kuziena Mindele W, Ilunga Wa Kyhi M, Mandula Mokenge G, Kaninda Badibabi L, Kasongo Bonama A, Kavunga Lukula P, Lumbala C, Scherrer B, Strub-Wourgaft N, Tarral A. Safety and efficacy of oral fexinidazole in children with gambiense human African trypanosomiasis: a multicentre, single-arm, open-label, phase 2-3 trial. Lancet Glob Health. 2022 Nov;10(11):e1665-e1674. doi: 10.1016/S2214-109X(22)00338-2. Epub 2022 Sep 27.
Results Reference
derived

Learn more about this trial

Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study

We'll reach out to this number within 24 hrs