Hereditary Hemorrhagic TelangiectasiaTangRONCT03572556
Prospective Descriptive Study of the Angiogenic T Cell Population in Subjects With Hereditary Hemorrhagic Telangiectasia (HHT)
An observational study of Hereditary Hemorrhagic Telangiectasia, run by Centre Hospitalier Universitaire Dijon at 1 site in France.
Trial summary
Hereditary hemorrhagic telangiectasia (HHT) results from genetic deregulation of angiogenesis. It is characterized by mucocutaneous telangiectasia responsible for recurrent epistaxis affecting quality of life (anaemia, iron deficiency, social distress). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Hereditary Hemorrhagic Telangiectasia
- What is being tested?
- Blood samples and Epistaxis charts
- How many people
- 60 participants
- Where
- 1 siteFrance
- Study dates
- 28 Jun 2018to 3 May 2020
- 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 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: outpatient
Who may be able to join
- Person who has given consent
- Adult
- Person capable of understanding spoken and written French
- "Patient" group:
- Certain HHT (3 or 4 Curacao criteria - Appendix 2):
- Recurring epistaxis
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- Telangiectasia of the skin or mouth
- Family hereditary context
- Arteriovenous visceral malformations
- Causal mutation identified
- Person capable of completing monthly epistaxis charts
- "Control" group :
- Control subjects will be matched to patients for age (+/- 6 years) and sex.
Who cannot join
- Person not affiliated to a national health insurance scheme
- Pregnant or breastfeeding woman
- Protected adult
- Hemoglobin levels less than 9 g/dl in the last 15 days
- Progressive or recent infectious disease, autoimmune disease or cancer (less than 6 months)
- Immunosuppressive treatment in progress or recent (less than 6 months), including systemic steroid therapy. The use of inhaled or topical steroids is not an exclusion criterion.
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- Treatment in progress or stopped less than 6 months ago or to be introduced within the next 3 months of the following medications:
- bevacizumab
- tranexamic acid
- dipeptidyl peptidase 4 inhibitors (diabetic patient)
- beta-blockers (hypertensive patient)
Trial locations and contacts
1 site in 1 country.
About this study
Hereditary hemorrhagic telangiectasia (HHT) results from genetic deregulation of angiogenesis. It is characterized by mucocutaneous telangiectasia responsible for recurrent epistaxis affecting quality of life (anaemia, iron deficiency, social distress). More rarely, HHT is complicated by the appearance of pulmonary, hepatic or cerebral arteriovenous malformations that can lead to serious complications: cerebrovascular accidents, cerebral abscesses, high output heart failure, and massive hemoptysis (1). The intensity of symptoms increases with age but with significant individual variability, even for the same mutation in the same family. Thus, while the mutations responsible for the disease have been identified, the pathophysiology is not fully understood because these mutations do not explain the great diversity of clinical presentations. Other factors not yet identified probably play an important role. Angiogenic T cells (TANG) are a newly individualized T cell population, defined by a CD4+CXCR4+CD31+ phenotype, which plays a key role in differentiating endothelial progenitors (2).
In an earlier study, the investigators showed that patients with HHT had a decrease in CD4+ and CD8+ LT compared to a cohort of healthy subjects (3).
They hypothesize that the lymphopenia mainly involves TANG, whose quantification could make it possible to assess the individual level of angiogenesis during HHT. The evaluation of the TANG levels could thus make it possible to personalize HHT management.
- Conditions
- Hereditary Hemorrhagic Telangiectasia
- Being tested
- Blood samples, Epistaxis charts
- Medical subject headings
- Telangiectasia, Hereditary Hemorrhagic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood samplesBiological
- 5 mL dry tube to separate serum
- Two 6 mL EDTA tubes for plasma separation
- Eight 6 mL heparinized tubes for flow cytometry (quantification of TANG such as CD3+CD31+CXCR4+ and CEC) and quantification of angiogenesis markers.
Epistaxis chartsOther
Three monthly epistaxis charts to be completed
| Study group | Role | What they receive |
|---|---|---|
| Patients | Hereditary hemorrhagic telangiectasia patients Receives: Biological: Blood samples; Other: Epistaxis charts | |
| Controls | Matched for age (+/- 5 ans) and sex. Receives: Biological: Blood samples |
What is being measured
The primary outcome is the main question the study is designed to answer.
Average monthly duration (in minutes) of epistaxis over the 3 months following inclusion
Measured: Through study completion, an average of 3 months
Number/mm3 of circulating TANG (CD3+CXCR4+CD31+) at inclusion.
Measured: At inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2018
- Study start28 Jun 2018
- Primary completion9 Mar 2020
- Study completion3 May 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Hemorrhagic TelangiectasiaStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire DijonThe sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire Dijon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03572556
- Sponsor's protocol ID
- GUILHEM AMRO/AOI 2017
- Acronym
- TangRO
- Status last verified
- Nov 2019
- First submitted
- 8 Jun 2018
- Last updated
- 20 Nov 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Descriptive Study of the Angiogenic T Cell Population in Subjects With Hereditary Hemorrhagic Telangiectasia (HHT)
- ClinicalTrials.gov ID
- NCT03572556
- Acronym / sponsor ID
- TangRO / GUILHEM AMRO/AOI 2017
- Conditions
- Hereditary Hemorrhagic Telangiectasia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire DijonUniversity, hospital or other
- Study start
- 28 Jun 2018 (actual)
- Primary completion
- 9 Mar 2020 (actual)
- Study completion
- 3 May 2020 (actual)
- First posted
- 28 Jun 2018 (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.
Hereditary hemorrhagic telangiectasia (HHT) results from genetic deregulation of angiogenesis. It is characterized by mucocutaneous telangiectasia responsible for recurrent epistaxis affecting quality of life (anaemia, iron deficiency, social distress). More rarely, HHT is complicated by the appearance of pulmonary, hepatic or cerebral arteriovenous malformations that can lead to serious complications: cerebrovascular accidents, cerebral abscesses, high output heart failure, and massive hemoptysis (1). The intensity of symptoms increases with age but with significant individual variability, even for the same mutation in the same family. Thus, while the mutations responsible for the disease have been identified, the pathophysiology is not fully understood because these mutations do not explain the great diversity of clinical presentations. Other factors not yet identified probably play an important role. Angiogenic T cells (TANG) are a newly individualized T cell population, defined by a CD4+CXCR4+CD31+ phenotype, which plays a key role in differentiating endothelial progenitors (2).
In an earlier study, the investigators showed that patients with HHT had a decrease in CD4+ and CD8+ LT compared to a cohort of healthy subjects (3).
They hypothesize that the lymphopenia mainly involves TANG, whose quantification could make it possible to assess the individual level of angiogenesis during HHT. The evaluation of the TANG levels could thus make it possible to personalize HHT management.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients | – | Biological: Blood samples, Other: Epistaxis chartsHereditary hemorrhagic telangiectasia patients |
| Controls | – | Biological: Blood samplesMatched for age (+/- 5 ans) and sex. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- outpatient
Key inclusion criteria (13 listed)
- Person who has given consent
- Adult
- Person capable of understanding spoken and written French
- Certain HHT (3 or 4 Curacao criteria - Appendix 2):
- Recurring epistaxis
Key exclusion criteria (11 listed)
- Person not affiliated to a national health insurance scheme
- Pregnant or breastfeeding woman
- Protected adult
- Hemoglobin levels less than 9 g/dl in the last 15 days
- Progressive or recent infectious disease, autoimmune disease or cancer (less than 6 months)
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 |
|---|---|---|
| Blood samples* 5 mL dry tube to separate serum * Two 6 mL EDTA tubes for plasma separation * Eight 6 mL heparinized tubes for flow cytometry (quantification of TANG such as CD3+CD31+CXCR4+ and CEC) and quantification of angiogenesis markers. | Biological | Controls, Patients |
| Epistaxis chartsThree monthly epistaxis charts to be completed | Other | Patients |
Primary outcome
Average monthly duration (in minutes) of epistaxis over the 3 months following inclusion
Time frame: Through study completion, an average of 3 months
Number/mm3 of circulating TANG (CD3+CXCR4+CD31+) at inclusion.
Time frame: At inclusion
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 20 Nov 2020.
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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?
An observational study of Hereditary Hemorrhagic Telangiectasia, run by Centre Hospitalier Universitaire Dijon at 1 site in France.
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 13 inclusion and 11 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 20 Nov 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 France. 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?
Average monthly duration (in minutes) of epistaxis over the 3 months following inclusion (time frame: Through study completion, an average of 3 months). The study lists 2 primary outcomes in all. 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 20 Nov 2020.