Trypanosoma Brucei Rhodesiense; InfectionNCT06060600
A Retrospective Analysis of Suramin Treatment for Stage 1 TBR
An observational study of Trypanosoma Brucei Rhodesiense; Infection, run by Paxmedica at 1 site in United States.
Trial summary
The study will include TBR HAT patients treated with suramin between 2000 and 2020 at three sites in Uganda and Malawi A natural history cohort composed of source data from approximately 200 patients from a published epidemiological study will be used as a comparator. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Trypanosoma Brucei Rhodesiense; Infection
- How many people
- 345 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jan 2023to 2 Aug 2023
- Phase
- Observational
- 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 Any age
- All sexes
- Child, Adult, Older adult
Study population: Patient charts will be retrieved from three major TBR HAT treatment centers. The study will include approximately 145 patients with S1TBR HAT who have sufficient clinical data for analysis. The study will also include a natural history cohort of the eligible subset of the approximately 200 patients from the 1900-1920 TBR HAT epidemic who were hospitalized in the years 1901 through 1910; some of these patients received experimental treatments such as arsenic which may have had some temporary clinical improvement, but none received any treatment with demonstrated efficacy.
Who may be able to join
- Suramin-treated cohort:
- Patient records must meet all the following criteria to be included in the suramin-treated cohort:
- Male or female of any age; age or sex must be included.
- Treatment with at least four full doses of suramin (not including the test dose).
- Onset date or duration of symptoms associated with S1 TBR HAT is available.
- An outcome is required; any mention of a clinical outcome is acceptable.
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- Must live in an area endemic for TBR HAT
- Documented HAT diagnosis
- Positive parasitology for HAT (observed in blood sample or a standard test).
- Natural History cohort:
- Treatment records from a cohort of approximately 200 patients who were hospitalized between 1901 and 1910 during the 1900-1920 HAT epidemic
- Treatment records must have sufficient information for analysis including:
- Demographic data: age or sex must be included
- Diagnosis: HAT diagnosis is confirmed by blood or lymph gland fluid analysis and parasites observed or HAT symptoms during the epidemic. For example, if the records state that a lymph node biopsy was performed, any result of the biopsy (e.g., documentation that trypanosomes were observed), a documentation of the HAT diagnosis, or mention of HAT symptoms such as sleepiness or excess sleeping are all acceptable. Symptoms alone are not sufficient, but a notation of biopsy and mention of HAT symptoms is acceptable.
- Outcome: An outcome is required; any mention of a clinical outcome is acceptable.
Who cannot join
- Patient records that meet any of the following criteria will be excluded from the suramin-treated cohort:
- Reported duration of symptoms for more than 2 months at time of presentation at a healthcare facility.
- Stage 2 TBR HAT as determined by examination of cerebrospinal fluid (CSF) using WHO criteria, which classify patients with the presence of trypanosomes in the CSF and/or a WBC count >5 cells/mm3 as Stage 2 TBR HAT at time of presentation to a healthcare facility.
- Evidence of Stage 2 TBR HAT symptoms at time of presentation to a healthcare facility.
- Required medication treatment for Stage 2 illness (melarsoprol) prior to time of presentation to a healthcare facility.
- Known to have had Trypanosoma Brucei Gambiense (TBG) HAT or became ill while travelling from an area known to be endemic for TBG HAT
- Duration of HAT symptoms for more than 6 months. Survival for more than 6 months with TBR HAT is unlikely.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Retrospective Analysis of Suramin Treatment for Stage 1 Trypanosoma Brucei Rhodesiense Human African Trypanosomiasis (S1 TBR HAT) in Uganda and Malawi
The study will include TBR HAT patients treated with suramin between 2000 and 2020 at three sites in Uganda and Malawi A natural history cohort composed of source data from approximately 200 patients from a published epidemiological study will be used as a comparator.
This study's objectives are to evaluate the efficacy and safety of suramin in the Stage 1 treatment of TBR HAT.
Read the detailed description
The study will include TBR HAT patients treated with suramin between 2000 and 2020 at three sites in Uganda and Malawi (e.g., the retrospective suramin-treated cohort). The study will include all the approximately 145 patients who are deemed eligible through chart review and who have sufficient data. A natural history cohort composed of source data from approximately 200 patients from a published epidemiological study will be used as a comparator.
The primary objective is to determine whether standard of care treatment with suramin, as currently practiced in Uganda and Malawi, leads to better health outcomes in patients with S1 TBR HAT than observed in a natural history cohort with source data from a published epidemiologic study.
- Medical subject headings
- Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Natural History Cohort: | * Treatment records from a cohort of approximately 200 patients who were hospitalized between 1901 and 1910 during the 1900-1920 HAT epidemic * Treatment records must have sufficient information for analysis including: * Demographic data: age or sex must be included * Diagnosis: HAT diagnosis is confirmed by blood or lymph gland fluid analysis and parasites observed or HAT symptoms during the epidemic. For example, if the records state that a lymph node biopsy was performed, any result of the biopsy (e.g., documentation that trypanosomes were observed), a documentation of the HAT diagnosis, or mention of HAT symptoms such as sleepiness or excess sleeping are all acceptable. Symptoms alone are not sufficient, but a notation of biopsy and mention of HAT symptoms is acceptable. * Outcome: An outcome is required; any mention of a clinical outcome is acceptable. | |
| Retrospective cohort | The hospital records of TBR HAT patients will be examined for date of symptom onset (if available) and hospital admission, race, sex, age, geographic area of origin, parasites, concomitant medications and illnesses, co-infections, and disease stage. Patients are normally screened for HAT and other tropical diseases using standard parasitological World Health Organization (WHO) criteria. Briefly, blood is obtained from the patients and checked for the presence of trypanosomes using direct wet smear and capillary centrifugation technique methods. Disease stage determination is by examination of CSF using the WHO criteria which classify patients with the presence of trypanosomes in the CSF and/or a WBC count >5 cells/mm3 as Stage 2 TBR HAT. Stage 2 patients would be disqualified from inclusion in the study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary efficacy endpoint is survival of patients treated with suramin compared to the natural history cohort.
The primary efficacy analysis will compare the proportion alive and not meeting any of the following criteria
- Death (both cohorts).
- Progression from Stage 1 to Stage 2 TBR HAT as defined by meeting any of the following:
- presence of TBR trypanosomes in the cerebrospinal fluid (CSF)
- abnormal symptoms .
- presence of TBR HAT symptoms for more than 2 months
- Use of melarsoprol for clinical worsening or treatment failure
- Moribund status
Measured: 30 Days
Secondary outcomes (1)
The secondary objective is to describe the safety and tolerability of suramin.
- Incidence of adverse events.
Measured: 30 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2023
- Study start2 Jan 2023
- Primary completion2 Aug 2023
- Study completion2 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Trypanosoma Brucei Rhodesiense; InfectionStudies 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
- Observational
- Observational model
- Ecologic or community
- Time perspective
- Retrospective
- Enrolment
- 345 participants (actual)
Sponsor and investigators
- Lead sponsor
- Paxmedica
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- jennifer L bonfrisco, Study director, Paxmedica
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06060600
- Sponsor's protocol ID
- PAX-HAT-301
- Status last verified
- Sep 2023
- First submitted
- 12 Sep 2023
- Last updated
- 29 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Retrospective Analysis of Suramin Treatment for Stage 1 Trypanosoma Brucei Rhodesiense Human African Trypanosomiasis (S1 TBR HAT) in Uganda and Malawi
- ClinicalTrials.gov ID
- NCT06060600
- Acronym / sponsor ID
- PAX-HAT-301
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 345 participants (actual)
- Lead sponsor
- PaxmedicaIndustry (pharma, biotech, devices)
- Study start
- 2 Jan 2023 (actual)
- Primary completion
- 2 Aug 2023 (actual)
- Study completion
- 2 Aug 2023 (actual)
- First posted
- 29 Sep 2023 (actual)
- 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 study will include TBR HAT patients treated with suramin between 2000 and 2020 at three sites in Uganda and Malawi A natural history cohort composed of source data from approximately 200 patients from a published epidemiological study will be used as a comparator.
This study's objectives are to evaluate the efficacy and safety of suramin in the Stage 1 treatment of TBR HAT.
Detailed description
The study will include TBR HAT patients treated with suramin between 2000 and 2020 at three sites in Uganda and Malawi (e.g., the retrospective suramin-treated cohort). The study will include all the approximately 145 patients who are deemed eligible through chart review and who have sufficient data. A natural history cohort composed of source data from approximately 200 patients from a published epidemiological study will be used as a comparator.
The primary objective is to determine whether standard of care treatment with suramin, as currently practiced in Uganda and Malawi, leads to better health outcomes in patients with S1 TBR HAT than observed in a natural history cohort with source data from a published epidemiologic study.
Study design
- Observational model
- Ecologic or community
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Natural History Cohort: | – | –* Treatment records from a cohort of approximately 200 patients who were hospitalized between 1901 and 1910 during the 1900-1920 HAT epidemic * Treatment records must have sufficient information for analysis including: * Demographic data: age or sex must be included * Diagnosis: HAT diagnosis is confirmed by blood or lymph gland fluid analysis and parasites observed or HAT symptoms during the epidemic. For example, if the records state that a lymph node biopsy was performed, any result of the biopsy (e.g., documentation that trypanosomes were observed), a documentation of the HAT diagnosis, or mention of HAT symptoms such as sleepiness or excess sleeping are all acceptable. Symptoms alone are not sufficient, but a notation of biopsy and mention of HAT symptoms is acceptable. * Outcome: An outcome is required; any mention of a clinical outcome is acceptable. |
| Retrospective cohort | – | –The hospital records of TBR HAT patients will be examined for date of symptom onset (if available) and hospital admission, race, sex, age, geographic area of origin, parasites, concomitant medications and illnesses, co-infections, and disease stage. Patients are normally screened for HAT and other tropical diseases using standard parasitological World Health Organization (WHO) criteria. Briefly, blood is obtained from the patients and checked for the presence of trypanosomes using direct wet smear and capillary centrifugation technique methods. Disease stage determination is by examination of CSF using the WHO criteria which classify patients with the presence of trypanosomes in the CSF and/or a WBC count >5 cells/mm3 as Stage 2 TBR HAT. Stage 2 patients would be disqualified from inclusion in the study. |
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Patient charts will be retrieved from three major TBR HAT treatment centers. The study will include approximately 145 patients with S1TBR HAT who have sufficient clinical data for analysis. The study will also include a natural history cohort of the eligible subset of the approximately 200 patients from the 1900-1920 TBR HAT epidemic who were hospitalized in the years 1901 through 1910; some of these patients received experimental treatments such as arsenic which may have had some temporary clinical improvement, but none received any treatment with demonstrated efficacy.
Key inclusion criteria (15 listed)
- Suramin-treated cohort:
- Treatment records from a cohort of approximately 200 patients who were hospitalized between 1901 and 1910 during the 1900-1920 HAT epidemic
- Treatment records must have sufficient information for analysis including:
Key exclusion criteria (7 listed)
- Patient records that meet any of the following criteria will be excluded from the suramin-treated cohort:
- Reported duration of symptoms for more than 2 months at time of presentation at a healthcare facility.
- Stage 2 TBR HAT as determined by examination of cerebrospinal fluid (CSF) using WHO criteria, which classify patients with the presence of trypanosomes in the CSF and/or a WBC count >5 cells/mm3 as Stage 2 TBR HAT at time of presentation to a healthcare facility.
- Evidence of Stage 2 TBR HAT symptoms at time of presentation to a healthcare facility.
- Required medication treatment for Stage 2 illness (melarsoprol) prior to time of presentation to a healthcare facility.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
The primary efficacy endpoint is survival of patients treated with suramin compared to the natural history cohort.
Time frame: 30 Days
The primary efficacy analysis will compare the proportion alive and not meeting any of the following criteria * Death (both cohorts). * Progression from Stage 1 to Stage 2 TBR HAT as defined by meeting any of the following: * presence of TBR trypanosomes in the cerebrospinal fluid (CSF) * abnormal symptoms . * presence of TBR HAT symptoms for more than 2 months * Use of melarsoprol for clinical worsening or treatment failure * Moribund status
1 secondary outcome
The secondary objective is to describe the safety and tolerability of suramin.
Time frame: 30 Days
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 29 Sep 2023.
- Individual participant dataSharing planned: Undecided
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.
jennifer L bonfrisco study director
Paxmedica
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Trypanosoma Brucei Rhodesiense; Infection, run by Paxmedica at 1 site in United States.
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 15 inclusion and 7 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 29 Sep 2023. 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 United States. 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.
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?
The primary efficacy endpoint is survival of patients treated with suramin compared to the natural history cohort. (time frame: 30 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Sep 2023.