ThrombopeniaFAGPLAQNCT06979765
Research for Plasma Biomarkers Associated With Fatigue in Thrombocytopenic Patients
A study testing blood sampling and self-administrated questionaires in people with Thrombopenia, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Thrombocytopenia is a clinical problem defined by a platelet count lower than 150×10⁹/L. It can be linked to various pathologies of central origin, such as decreased platelet production in the bone marrow, or peripheral origin with increased platelet destruction through autoimmune mechanisms, increased splenic… Read more about this study
- Who is it for?
- Ages 8 years and olderHealthy volunteers can take part
- Conditions the study is for
- Thrombopenia
- What is being tested?
- blood sampling and self-administrated questionaires
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 280 participants
- Where
- 1 siteFrance
- Study dates
- 26 Mar 2025to 26 Jun 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 8 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Patients with constitutional or autoimmune (chronic or persistent ITP with last treatment administration ≥ 3 weeks) thrombocytopenia (platelet count < 150×109/L) already diagnosed
- patient not being treated and not receiving serotonin reuptake inhibitor (SSRI) or norepinephrine (SNRI) or antithrombotic treatments (antiplatelet or anticoagulant) in the 10 days prior to inclusion
- affiliation to social security (beneficiary or assignee)
- patient followed in consultation in one of the recruiting haematology departments
- Patient (or parent) having received a detailed explanation of the research project and having consent by signing the consent form before any research-specific procedure
- Age- (± 5 years) and sex-matched healthy adult controls
Show all 15Show fewer
- Non-thrombocytopenic patients and not receiving antithrombotic, SNRI or SSRI therapy or if applicable, last treatment ≥ 10 days
- affiliation to social security (beneficiary or assignee)
- adults who received a detailed explanation of the research project and having consent by signing the consent form before any research-specific procedure
- Non inclusion criteria (adults and minors):
- Adult patients under legal protection (guardianship or curatorship) Thrombocytopenic patients treated with antithrombotics, serotonin reuptake inhibitors (SSRIs) or noradrenaline reuptake inhibitors (SNRIs)
- Minor patients weighing less than 20 kg
- Non inclusion criteria for healthy adult controls:
- Healthy adult volunteers under legal protection (guardianship, curatorship or safeguard of justice).
- Pregnant women
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hôpital Bichat-Claude Bernard
Paris, 75018
Nadine Ajzenberg, 01 40.25.62.73, nadine.ajzenberg@aphp.fr
Georges Jourdi, 01.49.95.64.11, georges.jourdi@aphp.fr
About this study
Thrombocytopenia is a clinical problem defined by a platelet count lower than 150×10⁹/L. It can be linked to various pathologies of central origin, such as decreased platelet production in the bone marrow, or peripheral origin with increased platelet destruction through autoimmune mechanisms, increased splenic sequestration, or excessive platelet consumption. Significant fatigue is often reported in association with thrombocytopenia, but its underlying pathophysiology remains unclear. One hypothesis is the role played by neurotrophic factors contained in platelets and released into the circulation following their activation, in particular the Brain-Derived Neurotrophic Factor (BDNF), which promotes the survival, growth, differentiation, and plasticity of neurons in both the central and peripheral nervous systems. Consequently, BDNF plays a key role in long-term memory, intellectual abilities, and neuroprotection.
In this context, this project aims to confirm whether platelet-origin neurotrophic biomarkers could explain the fatigue experienced by thrombocytopenic patients and whether it depends on the etiology of the thrombocytopenia.
Read the detailed description
Thrombocytopenia is defined as a platelet count below 150×109/L. The mechanisms leading to thrombocytopenia are multiple, and may be linked to :
- reduced platelet production in the bone marrow;
- increased destruction of peripheral platelets;
- increased splenic sequestration. In the event of a vascular breach, platelets contribute to hemostasis by sealing the lesion, thereby stopping bleeding. In thrombocytopenic patients, the best-known clinical signs are excessive mucocutaneous bleeding. Patients with severe thrombocytopenia (< 20×109 /L) may be at risk of life-threatening bleeding (cerebral bleeding).
In the case of autoimmune thrombocytopenia, cognitive disorders have been reported, detected by appropriate questionnaires, the pathophysiological mechanism of which remains unclear. In a study of 1871 patients with thrombocytopenia, 39% of patients in the UK and 22% in the USA reported severe asthenia. Asthenia appears to be related to thrombocytopenia, but the mechanism has not been identified either. Asthenia is a recognized symptom in other autoimmune pathologies, such as primary biliary cirrhosis (autoimmune liver disease), in which asthenia has been shown to be mainly associated with autonomic nervous system dysfunction.
While thrombocytopenia is primarily associated with bleeding risk, at least half of thrombocytopenic patients report fatigue and impaired mental and emotional health and social functioning, even though anemia is corrected and the association with autoimmune disease does not explain fatigue in all thrombocytopenic patients.
The hypothesis is that thrombocytopenia is associated with a decrease in circulating neurotrophic factor levels through reduced platelet granule secretion, which may explain the fatigue.
- Conditions
- Thrombopenia
- Being tested
- blood sampling, self-administrated questionaires
- Keywords
- Thrombopenia
- Medical subject headings
- Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
blood samplingBiological
3 blood tubes will be taken during a standard check-up
self-administrated questionairesOther
4 self administrated questionaires and scales at the inclusion
| Study group | Role | What they receive |
|---|---|---|
| Patients | Other | Minor patients (8 years of age or older) and adult patients seen consecutively in a hematology consultation as part of the follow-up of their thrombocytopenia. Receives: Biological: blood sampling; Other: self-administrated questionaires |
| Control group | Other | Control group (n=80) who will consist of healthy adult subjects (without pathology and without treatment) matched by age (± 5 years) and sex to adult patients included in the study given the possible influence of age and sex on circulating BDNF concentrations Receives: Biological: blood sampling; Other: self-administrated questionaires |
What is being measured
The primary outcome is the main question the study is designed to answer.
Blood BDNF levels in thrombocytopenic adult and pediatric patients
Measurement of BDNF levels in thrombocytopenic patients Adults and children with excessive fatigue
Measured: At the inclusion
Fatigue questionnaire scores for adult and pediatric patients
Fatigue questionnaire scores for adult and pediatric patients with excessive fatigue
Measured: At the inclusion
BDNF blood level in Healthy volunteers
Measurement of BDNF blood level in Healthy volunteers
Measured: At the inclusion
Secondary outcomes (22)
Measurement of pro-BDNF blood level in thrombopenic adult and pediatric patients
Measured: At the inclusion
measurement of p75NTR blood level in thrombopenic adult and pediatric patients
Measured: At the inclusion
measurement of TrkB blood level in thrombopenic adult and pediatric patients
Measured: At the inclusion
measurement of MMP9 blood level in thrombopenic adult and pediatric patients
Measured: At the inclusion
measurement of Serotonin blood level in thrombopenic adult and pediatric patients
Measured: At the inclusion
measurement of Dopamin blood level in thrombopenic adult and pediatric patients
Measured: At the inclusion
measurement of P-selectin blood level in thrombopenic adult and pediatric patients
Measured: At the inclusion
measurement of CD40 ligand blood level in thrombopenic adult and pediatric patients
Measured: At the inclusion
anxiety questionnaire sub score of adult and pediatric patients
Measured: At the inclusion
Depression questionnaire sub score of adult and pediatric patients
Measured: At the inclusion
cognition questionnaire sub score of adult and pediatric patients
Measured: At the inclusion
measurement of pro-BDNF blood level in Healthy volunteers
Measured: At the inclusion
measurement of p75NTR blood level in Healthy volunteers
Measured: At the inclusion
measurement of TrkB blood level in Healthy volunteers
Measured: At the inclusion
measurement of MMP9 blood level in Healthy volunteers
Measured: At the inclusion
measurement of Serotonin blood level in Healthy volunteers
Measured: At the inclusion
measurement of Dopamin blood level in Healthy volunteers
Measured: At the inclusion
measurement of P-selectin blood level in Healthy volunteers
Measured: At the inclusion
measurement of CD40 ligand blood level in Healthy volunteers
Measured: At the inclusion
anxiety questionnaire sub score in Healthy volunteers
Measured: At the inclusion
Depression questionnaire sub score in Healthy volunteers
Measured: At the inclusion
cognition questionnaire sub score in Healthy volunteers
Measured: At the inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2025
- Study start26 Mar 2025
- Primary completion26 Mar 2028 (estimated)
- Study completion26 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ThrombopeniaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 280 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Nadine Ajzenberg, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06979765
- Sponsor's protocol ID
- APHP240854
- Other IDs
- 2024-A01225-42
- Acronym
- FAGPLAQ
- Status last verified
- Jan 2026
- First submitted
- 12 May 2025
- Last updated
- 3 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Research for Plasma Biomarkers Associated With Fatigue in Thrombocytopenic Patients
- ClinicalTrials.gov ID
- NCT06979765
- Acronym / sponsor ID
- FAGPLAQ / APHP240854
- Conditions
- Thrombopenia
- Keywords
- Thrombopenia
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 280 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 26 Mar 2025 (actual)
- Primary completion
- 26 Mar 2028 (estimated)
- Study completion
- 26 Jun 2028 (estimated)
- First posted
- 20 May 2025 (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.
Thrombocytopenia is a clinical problem defined by a platelet count lower than 150×10⁹/L. It can be linked to various pathologies of central origin, such as decreased platelet production in the bone marrow, or peripheral origin with increased platelet destruction through autoimmune mechanisms, increased splenic sequestration, or excessive platelet consumption. Significant fatigue is often reported in association with thrombocytopenia, but its underlying pathophysiology remains unclear. One hypothesis is the role played by neurotrophic factors contained in platelets and released into the circulation following their activation, in particular the Brain-Derived Neurotrophic Factor (BDNF), which promotes the survival, growth, differentiation, and plasticity of neurons in both the central and peripheral nervous systems. Consequently, BDNF plays a key role in long-term memory, intellectual abilities, and neuroprotection.
In this context, this project aims to confirm whether platelet-origin neurotrophic biomarkers could explain the fatigue experienced by thrombocytopenic patients and whether it depends on the etiology of the thrombocytopenia.
Detailed description
Thrombocytopenia is defined as a platelet count below 150×109/L. The mechanisms leading to thrombocytopenia are multiple, and may be linked to :
- reduced platelet production in the bone marrow;
- increased destruction of peripheral platelets;
- increased splenic sequestration. In the event of a vascular breach, platelets contribute to hemostasis by sealing the lesion, thereby stopping bleeding. In thrombocytopenic patients, the best-known clinical signs are excessive mucocutaneous bleeding. Patients with severe thrombocytopenia (< 20×109 /L) may be at risk of life-threatening bleeding (cerebral bleeding).
In the case of autoimmune thrombocytopenia, cognitive disorders have been reported, detected by appropriate questionnaires, the pathophysiological mechanism of which remains unclear. In a study of 1871 patients with thrombocytopenia, 39% of patients in the UK and 22% in the USA reported severe asthenia. Asthenia appears to be related to thrombocytopenia, but the mechanism has not been identified either. Asthenia is a recognized symptom in other autoimmune pathologies, such as primary biliary cirrhosis (autoimmune liver disease), in which asthenia has been shown to be mainly associated with autonomic nervous system dysfunction.
While thrombocytopenia is primarily associated with bleeding risk, at least half of thrombocytopenic patients report fatigue and impaired mental and emotional health and social functioning, even though anemia is corrected and the association with autoimmune disease does not explain fatigue in all thrombocytopenic patients.
The hypothesis is that thrombocytopenia is associated with a decrease in circulating neurotrophic factor levels through reduced platelet granule secretion, which may explain the fatigue.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients | Other | Biological: blood sampling, Other: self-administrated questionairesMinor patients (8 years of age or older) and adult patients seen consecutively in a hematology consultation as part of the follow-up of their thrombocytopenia. |
| Control group | Other | Biological: blood sampling, Other: self-administrated questionairesControl group (n=80) who will consist of healthy adult subjects (without pathology and without treatment) matched by age (± 5 years) and sex to adult patients included in the study given the possible influence of age and sex on circulating BDNF concentrations |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- Patients with constitutional or autoimmune (chronic or persistent ITP with last treatment administration ≥ 3 weeks) thrombocytopenia (platelet count < 150×109/L) already diagnosed
- patient not being treated and not receiving serotonin reuptake inhibitor (SSRI) or norepinephrine (SNRI) or antithrombotic treatments (antiplatelet or anticoagulant) in the 10 days prior to inclusion
- affiliation to social security (beneficiary or assignee)
- patient followed in consultation in one of the recruiting haematology departments
- Patient (or parent) having received a detailed explanation of the research project and having consent by signing the consent form before any research-specific procedure
Key exclusion criteria (0 listed)
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 sampling3 blood tubes will be taken during a standard check-up | Biological | Control group, Patients |
| self-administrated questionaires4 self administrated questionaires and scales at the inclusion | Other | Control group, Patients |
Primary outcome
Blood BDNF levels in thrombocytopenic adult and pediatric patients
Time frame: At the inclusion
Measurement of BDNF levels in thrombocytopenic patients Adults and children with excessive fatigue
Fatigue questionnaire scores for adult and pediatric patients
Time frame: At the inclusion
Fatigue questionnaire scores for adult and pediatric patients with excessive fatigue
BDNF blood level in Healthy volunteers
Time frame: At the inclusion
Measurement of BDNF blood level in Healthy volunteers
22 secondary outcomes
Measurement of pro-BDNF blood level in thrombopenic adult and pediatric patients
Time frame: At the inclusion
measurement of p75NTR blood level in thrombopenic adult and pediatric patients
Time frame: At the inclusion
measurement of TrkB blood level in thrombopenic adult and pediatric patients
Time frame: At the inclusion
measurement of MMP9 blood level in thrombopenic adult and pediatric patients
Time frame: At the inclusion
measurement of Serotonin blood level in thrombopenic adult and pediatric patients
Time frame: At the inclusion
measurement of Dopamin blood level in thrombopenic adult and pediatric patients
Time frame: At the inclusion
measurement of P-selectin blood level in thrombopenic adult and pediatric patients
Time frame: At the inclusion
measurement of CD40 ligand blood level in thrombopenic adult and pediatric patients
Time frame: At the inclusion
anxiety questionnaire sub score of adult and pediatric patients
Time frame: At the inclusion
Depression questionnaire sub score of adult and pediatric patients
Time frame: At the inclusion
cognition questionnaire sub score of adult and pediatric patients
Time frame: At the inclusion
measurement of pro-BDNF blood level in Healthy volunteers
Time frame: At the inclusion
measurement of p75NTR blood level in Healthy volunteers
Time frame: At the inclusion
measurement of TrkB blood level in Healthy volunteers
Time frame: At the inclusion
measurement of MMP9 blood level in Healthy volunteers
Time frame: At the inclusion
measurement of Serotonin blood level in Healthy volunteers
Time frame: At the inclusion
measurement of Dopamin blood level in Healthy volunteers
Time frame: At the inclusion
measurement of P-selectin blood level in Healthy volunteers
Time frame: At the inclusion
measurement of CD40 ligand blood level in Healthy volunteers
Time frame: At the inclusion
anxiety questionnaire sub score in Healthy volunteers
Time frame: At the inclusion
Depression questionnaire sub score in Healthy volunteers
Time frame: At the inclusion
cognition questionnaire sub score in Healthy volunteers
Time frame: At the inclusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2026.
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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.
Nadine Ajzenberg principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing blood sampling and self-administrated questionaires in people with Thrombopenia, run by Assistance Publique - Hôpitaux de Paris 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 15 inclusion and 0 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: looking for participants now, as of the sponsor's last update on 3 Feb 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Nadine Ajzenberg (01 40.25.62.73, nadine.ajzenberg@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Blood BDNF levels in thrombocytopenic adult and pediatric patients (time frame: At the inclusion). The study lists 3 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 3 Feb 2026.