Human African Trypanosomiasis (HAT)Phase 2/3NCT02184689
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
A Phase 2/3 study testing fexinidazole in people with Human African Trypanosomiasis (HAT), run by Drugs for Neglected Diseases at 1 site in Democratic Republic of the Congo.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 14 years
- Conditions the study is for
- Human African Trypanosomiasis (HAT)
- What is being tested?
- fexinidazole
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 125 participants
- Where
- 1 siteDemocratic Republic of the Congo
- Study dates
- 3 May 2014to 27 Jun 2017
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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)
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
Democratic Republic of the Congo1 site
Study site
Kinshasa
About this study
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.
- Conditions
- Human African Trypanosomiasis (HAT)
- Being tested
- fexinidazole
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
fexinidazoleDrug
| Study group | Role | What they receive |
|---|---|---|
| Fexinidazole | Experimental | Not described Receives: Drug: fexinidazole |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 months after the end of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2014 (estimated)
- Study start3 May 2014
- Primary completion14 Dec 2016
- Study completion27 Jun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Human African Trypanosomiasis (HAT)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 125 participants (actual)
Sponsor and investigators
- Lead sponsor
- Drugs for Neglected Diseases
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02184689
- Sponsor's protocol ID
- DNDiHATFEX006
- Status last verified
- Jun 2020
- First submitted
- 19 Jun 2014
- Last updated
- 24 Jun 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 36179736
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02184689
- Acronym / sponsor ID
- DNDiHATFEX006
- Conditions
- Human African Trypanosomiasis (HAT)
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 125 participants (actual)
- Lead sponsor
- Drugs for Neglected DiseasesUniversity, hospital or other
- Study start
- 3 May 2014 (actual)
- Primary completion
- 14 Dec 2016 (actual)
- Study completion
- 27 Jun 2017 (actual)
- First posted
- 9 Jul 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fexinidazole | Experimental | Drug: fexinidazole |
Who may qualify
- Ages
- 6 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (19 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| fexinidazole | Drug | Fexinidazole |
Primary outcome
Outcome (success or failure) at the test of cure (ToC)
Time frame: 12 months after the end of treatment
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2020.
- Derived from this study: PubMed 36179736Kande 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.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing fexinidazole in people with Human African Trypanosomiasis (HAT), run by Drugs for Neglected Diseases at 1 site in Democratic Republic of the Congo.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Democratic Republic of the Congo. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Outcome (success or failure) at the test of cure (ToC) (time frame: 12 months after the end of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02184689, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2020.