Human African Trypanosomiasis (HAT)Phase 2/3NCT02169557
Efficacy and Safety of Fexinidazole in Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort 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 purpose of this study is to demonstrate the treatment success of fexinidazole, at one year follow-up visit, in HAT stage 1 and early stage 2 patients. Read more about this study
- Who is it for?
- Ages 15 years and older
- 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
- 230 participants
- Where
- 1 siteDemocratic Republic of the Congo
- Study dates
- 30 Apr 2014to 25 Apr 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent
- 15 years old or more
- Male or female
- Ability to ingest at least one complete meal per day (or at least one Plumpy'Nut® sachet)
- Karnofsky index > 50
- Presence of trypanosomes in blood or lymph
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- Absence of trypanosomes in CSF
- Permanent address and ability to comply with the follow-up visit schedule
- Patient agreeing to be hospitalized to receive the treatment
Who cannot join
- Severely malnutrition, defined as BMI < 16.
- Inability to take oral medication.
- Pregnancy or breastfeeding (a urine pregnancy test will be performed in all women of childbearing age within 24 h prior to treatment).
- Clinically relevant medical condition other than HAT that, in the Investigator's opinion, may jeopardize patient safety or interfere with participation in the study, including but not limited to significant liver or cardiovascular disease, documented or suspected active infection (including AIDS), CNS trauma or seizure disorders, coma or altered consciousness.
- Severely deteriorated general condition, e.g. cardiovascular shock, respiratory distress syndrome, or terminal illness.
- Any medical condition (except HAT specific symptoms) hindering communication with the Investigator as required for the completion of this study.
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- 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 value, such as:
- Alanine aminotransferase (ALAT) and/or aspartate aminotransferase (ASAT) > 2 times ULN
- Total bilirubin (TBIL) > 1.5 times ULN
- Severe leukopenia (< 2000/mm3)
- Potassium (K+) < 3.5 mmol/L
- Any clinically significant abnormal value (see details in Investigator Manual)
- Pregnancy confirmed by a positive urine pregnancy test obtained within 24 h prior to start of study treatment (see Section 5.8.3 Contraception; p35) QTcF ≥ 450 ms as measured automatically (if the 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 purpose of this study is to demonstrate the treatment success of fexinidazole, at one year follow-up visit, in HAT stage 1 and early stage 2 patients.
Read the detailed description
Primary Objective
-To demonstrate that the success rate of fexinidazole at one year follow-up in stage 1 and early stage 2 patients is greater than 80%. An 80 % success rate is considered as unacceptable.
Secondary Objectives
- To verify whether the success rate of fexinidazole treatment depends on the stage of the disease (stage 1 versus early stage 2); and, if the difference between the 2 stages is significant, to show that the success rate is greater than 80% and compatible with the historical success rate of NECT in early stage 2 patients and with the historical success rate of pentamidine in stage 1 patients.
- To verify whether the success rate of fexinidazole treatment depends on the number of WBCs in CSF before treatment initiation.
- To assess changes in the success rate over time.
- To evaluate the safety of fexinidazole and determine whether its safety profile is comparable to the historical safety profile of pentamidine.
- 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) visit 12 months after the end of treatment (EOT).
Measured: 12 months after end of treatment (day 11)
Secondary outcomes (1)
Success or failure at each visit between the End of treatment and 18 months visit.
Measured: End of treatment (day 11) to last follow-up visit (18 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2014 (estimated)
- Study start30 Apr 2014
- Primary completion9 Oct 2016
- Study completion25 Apr 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
- 230 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
- NCT02169557
- Sponsor's protocol ID
- DNDiHATFEX005
- Status last verified
- Jun 2020
- First submitted
- 15 May 2014
- Last updated
- 24 Jun 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kande Betu Ku Mesu V, Mutombo Kalonji W, Bardonneau C, Valverde Mordt O, Ngolo Tete D, Blesson S, Simon F, Delhomme S, Bernhard S, Mahenzi Mbembo H, Mpia Moke C, Lumeya Vuvu S, Mudji E'kitiak J, Akwaso Masa F, Mukendi Ilunga M, Mpoyi Muamba Nzambi D, Mayala Malu T, Kapongo Tshilumbwa S, Botalema Bolengi F, Nkieri Matsho M, Lumbala C, Scherrer B, Strub-Wourgaft N, Tarral A. Oral fexinidazole for stage 1 or early stage 2 African Trypanosoma brucei gambiense trypanosomiasis: a prospective, multicentre, open-label, cohort study. Lancet Glob Health. 2021 Jul;9(7):e999-e1008. doi: 10.1016/S2214-109X(21)00208-4.
PubMed 34143998
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 Patients With Stage 1 or Early Stage 2 Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open-label Cohort Study, plug-in to the Pivotal Study
- ClinicalTrials.gov ID
- NCT02169557
- Acronym / sponsor ID
- DNDiHATFEX005
- Conditions
- Human African Trypanosomiasis (HAT)
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 230 participants (actual)
- Lead sponsor
- Drugs for Neglected DiseasesUniversity, hospital or other
- Study start
- 30 Apr 2014 (actual)
- Primary completion
- 9 Oct 2016 (actual)
- Study completion
- 25 Apr 2017 (actual)
- First posted
- 23 Jun 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 purpose of this study is to demonstrate the treatment success of fexinidazole, at one year follow-up visit, in HAT stage 1 and early stage 2 patients.
Detailed description
Primary Objective
-To demonstrate that the success rate of fexinidazole at one year follow-up in stage 1 and early stage 2 patients is greater than 80%. An 80 % success rate is considered as unacceptable.
Secondary Objectives
- To verify whether the success rate of fexinidazole treatment depends on the stage of the disease (stage 1 versus early stage 2); and, if the difference between the 2 stages is significant, to show that the success rate is greater than 80% and compatible with the historical success rate of NECT in early stage 2 patients and with the historical success rate of pentamidine in stage 1 patients.
- To verify whether the success rate of fexinidazole treatment depends on the number of WBCs in CSF before treatment initiation.
- To assess changes in the success rate over time.
- To evaluate the safety of fexinidazole and determine whether its safety profile is comparable to the historical safety profile of pentamidine.
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
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Signed informed consent
- 15 years old or more
- Male or female
- Ability to ingest at least one complete meal per day (or at least one Plumpy'Nut® sachet)
- Karnofsky index > 50
Key exclusion criteria (20 listed)
- Severely malnutrition, defined as BMI < 16.
- Inability to take oral medication.
- Pregnancy or breastfeeding (a urine pregnancy test will be performed in all women of childbearing age within 24 h prior to treatment).
- Clinically relevant medical condition other than HAT that, in the Investigator's opinion, may jeopardize patient safety or interfere with participation in the study, including but not limited to significant liver or cardiovascular disease, documented or suspected active infection (including AIDS), CNS trauma or seizure disorders, coma or altered consciousness.
- Severely deteriorated general condition, e.g. cardiovascular shock, respiratory distress syndrome, or terminal illness.
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) visit 12 months after the end of treatment (EOT).
Time frame: 12 months after end of treatment (day 11)
1 secondary outcome
Success or failure at each visit between the End of treatment and 18 months visit.
Time frame: End of treatment (day 11) to last follow-up visit (18 months)
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 34143998Kande Betu Ku Mesu V, Mutombo Kalonji W, Bardonneau C, Valverde Mordt O, Ngolo Tete D, Blesson S, Simon F, Delhomme S, Bernhard S, Mahenzi Mbembo H, Mpia Moke C, Lumeya Vuvu S, Mudji E'kitiak J, Akwaso Masa F, Mukendi Ilunga M, Mpoyi Muamba Nzambi D, Mayala Malu T, Kapongo Tshilumbwa S, Botalema Bolengi F, Nkieri Matsho M, Lumbala C, Scherrer B, Strub-Wourgaft N, Tarral A. Oral fexinidazole for stage 1 or early stage 2 African Trypanosoma brucei gambiense trypanosomiasis: a prospective, multicentre, open-label, cohort study. Lancet Glob Health. 2021 Jul;9(7):e999-e1008. doi: 10.1016/S2214-109X(21)00208-4.
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 9 inclusion and 20 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) visit 12 months after the end of treatment (EOT). (time frame: 12 months after end of treatment (day 11)). 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 NCT02169557, 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.