Immune ThrombocytopeniaPhase 1/2NCT06776510
A Clinical Study of NAD in the Treatment of Immune Thrombocytopenia
A Phase 1/2 study testing nicotinamide adenine dinucleotid/nicotinamide mononucleotide in people with Immune Thrombocytopenia and Treatment, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
To evaluate the safety and efficacy of nicotinamide adenine dinucleotide in the treatment of immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including Anti-CD20 Antibody and/or TPO-RA. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia, Treatment
- What is being tested?
- nicotinamide adenine dinucleotid/nicotinamide mononucleotide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 15 Jan 2025to 1 Mar 2026
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 and above, male or female;
- Conform to the diagnostic criteria of immune Thrombocytopenia (ITP) ≥3 months;
- Failure to achieve response or relapse after corticosteroid therapy, and failure to achieve response or relapse after previous second-line treatments such as TPO/TPORAs therapy, or are unable to afford the cost of the treatment;
- The platelet count of <30 X 10^9/L measured within 2 days prior to inclusion (During the screening visit and/or before receiving the study drug, platelet counts must be less than 30×10^9/L on at least two consecutive occasions, with a minimum interval of 1 day between the two tests.);
- ECOG physical state score ≤ 2 points;
- Subjects on stable dose maintenance therapy are allowed to be included (concomitant medications may include corticosteroids (≤0.5 mg/kg of prednisone or equivalent steroids) or TPO receptor agonists, etc.), but at the time of enrollment, only one concomitant medication with a stable dose is permitted. The concomitant medication must have been on a stable dose for at least 4 weeks prior to the first dose of the study drug;
- For female patients of childbearing potential, a negative pregnancy test result is required. Both female patients of childbearing potential and male patients must use highly effective contraception during the study and for 4 months/6 months after discontinuing treatment;
- Signed and dated written informed consent
Who cannot join
- Those who are allergic to Nicotinamide adenine dinucleotide or excipients, or who have previously received CD38 monoclonal antibody treatment with no efficacy.
- Those with autoimmune hemolytic anemia, or various types of secondary and genetic thrombocytopenia, such as leukemia, lymphoma, multiple myeloma, aplastic anemia, myelodysplastic syndrome, Evans syndrome, common variable immunodeficiency, systemic lupus erythematosus, cirrhosis, antiphospholipid antibody syndrome, pseudo-thrombocytopenia, drug-induced thrombocytopenia (e.g., quinine, heparin, antimicrobial drugs, anticonvulsants, etc.).
- A history of any thrombosis or embolism events within 12 months prior to the first dose, or the presence of extensive and severe bleeding, such as hemoptysis, upper gastrointestinal bleeding, intracranial hemorrhage, sepsis, or other irregular bleeding.
- Participation in any other clinical trial involving investigational drugs (including vaccine studies) or exposure to other investigational drugs within 4 weeks or 5 half-lives (whichever is longer) prior to the first dose.
- Use of anticoagulants or any drugs with antiplatelet effects (e.g., aspirin) within 2 weeks prior to the first dose.
- Receiving emergency treatment for ITP within 2 weeks prior to the first dose (e.g., methylprednisolone, platelet transfusion, intravenous immunoglobulin, or TPO receptor agonist treatment).
Show all 19Show fewer
- Receiving azathioprine, danazol, cyclosporine A, tacrolimus, sirolimus, or similar drugs within 4 weeks prior to the first dose; receiving CD20 monoclonal antibodies such as rituximab, cyclophosphamide, vincristine, or similar drugs within 3 months prior to the first dose.
- Splenectomy within 6 months prior to the first dose.
- Receiving a live vaccine within 4 weeks prior to the first dose, or planning to receive any live vaccine during the study period.
- A history of undergoing allogeneic stem cell transplantation or organ transplantation.
- A history of clinically significant diseases that, in the investigator's opinion, may pose a risk to the subject's safety or affect the assessment of safety or efficacy during the study if the disease/condition worsens.
- Subjects who have had malignant tumors within the past 5 years prior to screening (excluding completely cured carcinoma in situ of the cervix and non-metastatic squamous cell carcinoma or basal cell carcinoma of the skin).
- Exhibiting clinically significant laboratory abnormalities at screening: a) Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ the upper limit of normal (ULN); b) Total bilirubin ≥ 1.2 times ULN. ;c) Creatinine or blood urea nitrogen (BUN) ≥ ULN.
- Positive for HIV antibodies or syphilis antibodies.
- Positive for hepatitis B surface antigen (HBsAg) at screening, or positive for hepatitis B core antibody with a positive HBV-DNA result by polymerase chain reaction (PCR) testing, or positive for hepatitis C virus (HCV) antibodies.
- Women who are pregnant or breastfeeding, or planning to become pregnant or breastfeed during the study; and men whose partners are planning to become pregnant during the study.
- Subjects with mental disorders who are unable to provide informed consent or participate in the trial and follow-up properly.
- Subjects with unresolved toxicity symptoms caused by prior treatments before participating in the trial.
- Any other conditions deemed unsuitable for participation in this study as assessed by the investigator.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, 300020
Yunfei Chen, MD, +8618502220788, chenyunfei@ihcams.ac.cn
About this study
Official title: A Prospective, One-arm and Open Clinical Study to Assess Safety and Efficacy of Nicotinamide Adenine Dinucleotide in the Treatment of Primary Immune Thrombocytopenia
To evaluate the safety and efficacy of nicotinamide adenine dinucleotide in the treatment of immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including Anti-CD20 Antibody and/or TPO-RA.
Read the detailed description
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Therefore, there is still a lack of effective treatment for adult ITP, especially for recurrent and refractory ITP patients, which is one of the problems that have attracted more attention and need to be solved urgently.
The main pathogenesis of ITP is the loss of platelet autoantigen immune tolerance, which leads to abnormal activation of humoral and cellular immunity. It is characterized by antibody mediated platelet destruction and insufficient platelet production by megakaryocytes. The residual long-term autoreactive plasma cells may be a source of therapeutic resistance to autoimmune cytopenia. Antiplatelet specific plasma cells have been detected in the spleen of patients with rituximab refractory ITP. Therefore, the strategy of simply eliminating B cells may not work, because LLPC will continue to produce pathogenic antibodies. However, targeting LLPC becomes a new strategy to treat autoimmune diseases.
CM313, a kind of anti-CD38 antibody, is a new type of monoclonal antibody targeting CD38. It targets plasma cells and has carried out some clinical studies in multiple myeloma, with good therapeutic effects. We treated patients with refractory/relapsed ITP using CM313 and achieved good therapeutic outcomes, which may provide a new strategy for treating ITP. Current research shows that CD38, as a metabolic enzyme, can hydrolyze nicotinamide adenine dinucleotide (NAD+), leading to a decrease in intracellular NAD+. Additionally, during aging and inflammation, CD38 is highly expressed on macrophages, and the downregulation of NAD+ levels. Based on the current mechanistic studies of CD38 monoclonal antibody treatment for ITP, we found that macrophages in ITP patients exhibit a shift towards M1 polarization. We hypothesize that CD38 antibodies may reduce platelet destruction by macrophages by upregulating NAD+ levels, which in turn inhibits M1 polarization. Therefore, increasing NAD+ levels may have therapeutic potential for ITP. Since nicotinamide adenine dinucleotide (NAD+) has been used in clinical practice as an adjunctive treatment for leukopenia, coronary artery disease as well as myocarditis, and nicotinamide mononucleotide (NMN) has been explored in clinical trials, we will further investigate the therapeutic efficacy and safety of these compounds in ITP patients.
Therefore, the investigators designed this clinical trial to evaluate the safety and efficacy of Nicotinamide Adenine Dinucleotide (Coenzyme I for Injection, NAD+) and nicotinamide mononucleotide (NMN) in the treatment of primary immune thrombocytopenia in patients who are steroid-refractory or steroid-dependent, and fail to respond to at least one previous second-line therapy, including rituximab and/ or TPO agonist.
- Conditions
- Immune Thrombocytopenia, Treatment
- Being tested
- nicotinamide adenine dinucleotid/nicotinamide mononucleotide
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nicotinamide adenine dinucleotid/nicotinamide mononucleotideDrug
Also called Coenzyme I for Injection（NAD+）/NMN
Drug: intravenous nicotinamide adenine dinucleotide (Coenzyme I for Injection) administration This study adopts a prospective, single arm, open design method. Twenty subjects were enrolled in the study and were treated with nicotinamide adenine dinucleotide (100mg/d) for 1 week.
Drug: oral nicotinamide mononucleotide administration This study adopts a prospective, single arm, open design method. Twenty subjects were enrolled in the study and were treated with nicotinamide mononucleotide 450mg BID for 2 weeks.
The first stage is the main research stage (d0-w2), which is the core treatment period. The aim is to observe the safety and efficacy during treatment.
The second stage (w2-w8) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy after treatment.
| Study group | Role | What they receive |
|---|---|---|
| Experimental: | Experimental | nicotinamide adenine dinucleotide/nicotinamide mononucleotide Receives: Drug: nicotinamide adenine dinucleotid/nicotinamide mononucleotide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of overall efficacy response after nicotinamide adenine dinucleotide/NMN treatment within 2 weeks
Proportion of subjects with a platelet count ≥ 50 × 10^9/L (two consecutive measurements at least 1 day apart) within 2 weeks after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
Measured: [Time Frame: 2 weeks]
incidence of treatment-emergent Adverse Events and TRAE
Incidence, severity, and relationship of treatment emergent adverse events after nicotinamide adenine dinucleotide/NMN treatment
Measured: [Time Frame: 8 weeks]
Secondary outcomes (8)
Evaluation of the efficacy at each visit period
Proportion of subjects with a platelet count ≥ 50 × 10^9/L at week 1, week 2, week 3, week 4, week 6，week 8 in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
Measured: [Time Frame: 8 weeks]
Duration from treatment initiation to platelet count ≥30×10^9/L (at least two times of baseline platelet count) and ≥50×10^9/L
Duration from treatment initiation to platelet count ≥30×10^9/L (at least two times of baseline platelet count) and ≥50×10^9/L without having received any platelet elevating therapy or having had dose increment of TPO-RA and/or corticosteroids
Measured: [Time Frame: 8 weeks]
Platelet count at each visit time point
Platelet count values at each visit time point including day0, week 1, week 2, week 3, week 4, week 5, week 6, week 7, week 8.
Measured: [Time Frame: 8 weeks]
Emergency treatment
The proportion of subjects who received emergency treatment within 2 weeks and during the entire study period.
Measured: [Time Frame: 8 weeks]
Cumulative weeks of platelet ≥30×10^9/L (at least two times of baseline platelet count) and platelet ≥50×10^9/L
Cumulative weeks of platelet ≥30×10^9/L (at least two times of baseline platelet count) and platelet ≥50×10^9/L without having received any platelet elevating therapy or having had dose increment of TPO-RA and/or corticosteroids
Measured: [Time Frame: 8 weeks]
The change in bleeding score before treatment and 2 weeks after treatment assessed using the world health organization (WHO) bleeding scale
The change in bleeding score before treatment and 2 weeks after treatment according to the reported World Health Organization's Bleeding Scale. The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Measured: [Time Frame: 2 weeks]
Evaluation of the efficacy during the first 4 weeks
Proportion of subjects achieving platelet counts ≥ 50×10^9/L at least once in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the first 4 weeks
Measured: 4 weeks
The response rate within 4 weeks
Proportion of subjects whose platelet counts ≥ 30×10^9/L and at least two times of baseline platelet count in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids within 4 weeks（two consecutive measurements at least 7 days apart）.
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2025
- Study start15 Jan 2025
- Primary completion1 Dec 2025 (estimated)
- Study completion1 Mar 2026 (estimated)
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 Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Chinese Academy of Medical Science and Blood Disease Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06776510
- Sponsor's protocol ID
- IIT2024089
- Status last verified
- Jul 2025
- First submitted
- 7 Jan 2025
- Last updated
- 5 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Researchers qualified can request the dataset, including de-identified individual subject data. Data may be requested from PI from 12 months 36 months after study completion.
Publications
Li H, Xu Y, Chen Y, Ji L, Xu Y, Zheng W, Sun T, Fu R, Pei X, Liu X, Xue F, Liu W, Wang W, Chi Y, Yang R, Wei J, Zhang L. Low-dose oral nicotinamide mononucleotide for immune thrombocytopenia: a phase 1/2 trial. Nat Med. 2026 Jun;32(6):2026-2036. doi: 10.1038/s41591-026-04366-x. Epub 2026 Apr 29.
PubMed 42056497
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, One-arm and Open Clinical Study to Assess Safety and Efficacy of Nicotinamide Adenine Dinucleotide in the Treatment of Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT06776510
- Acronym / sponsor ID
- IIT2024089
- Conditions
- Immune Thrombocytopenia, Treatment
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 15 Jan 2025 (actual)
- Primary completion
- 1 Dec 2025 (estimated)
- Study completion
- 1 Mar 2026 (estimated)
- First posted
- 15 Jan 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.
To evaluate the safety and efficacy of nicotinamide adenine dinucleotide in the treatment of immune thrombocytopenia in patients who have not responded adequately or relapsed after first-line treatment and at least one second-line therapy including Anti-CD20 Antibody and/or TPO-RA.
Detailed description
Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Therefore, there is still a lack of effective treatment for adult ITP, especially for recurrent and refractory ITP patients, which is one of the problems that have attracted more attention and need to be solved urgently.
The main pathogenesis of ITP is the loss of platelet autoantigen immune tolerance, which leads to abnormal activation of humoral and cellular immunity. It is characterized by antibody mediated platelet destruction and insufficient platelet production by megakaryocytes. The residual long-term autoreactive plasma cells may be a source of therapeutic resistance to autoimmune cytopenia. Antiplatelet specific plasma cells have been detected in the spleen of patients with rituximab refractory ITP. Therefore, the strategy of simply eliminating B cells may not work, because LLPC will continue to produce pathogenic antibodies. However, targeting LLPC becomes a new strategy to treat autoimmune diseases.
CM313, a kind of anti-CD38 antibody, is a new type of monoclonal antibody targeting CD38. It targets plasma cells and has carried out some clinical studies in multiple myeloma, with good therapeutic effects. We treated patients with refractory/relapsed ITP using CM313 and achieved good therapeutic outcomes, which may provide a new strategy for treating ITP. Current research shows that CD38, as a metabolic enzyme, can hydrolyze nicotinamide adenine dinucleotide (NAD+), leading to a decrease in intracellular NAD+. Additionally, during aging and inflammation, CD38 is highly expressed on macrophages, and the downregulation of NAD+ levels. Based on the current mechanistic studies of CD38 monoclonal antibody treatment for ITP, we found that macrophages in ITP patients exhibit a shift towards M1 polarization. We hypothesize that CD38 antibodies may reduce platelet destruction by macrophages by upregulating NAD+ levels, which in turn inhibits M1 polarization. Therefore, increasing NAD+ levels may have therapeutic potential for ITP. Since nicotinamide adenine dinucleotide (NAD+) has been used in clinical practice as an adjunctive treatment for leukopenia, coronary artery disease as well as myocarditis, and nicotinamide mononucleotide (NMN) has been explored in clinical trials, we will further investigate the therapeutic efficacy and safety of these compounds in ITP patients.
Therefore, the investigators designed this clinical trial to evaluate the safety and efficacy of Nicotinamide Adenine Dinucleotide (Coenzyme I for Injection, NAD+) and nicotinamide mononucleotide (NMN) in the treatment of primary immune thrombocytopenia in patients who are steroid-refractory or steroid-dependent, and fail to respond to at least one previous second-line therapy, including rituximab and/ or TPO agonist.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental: | Experimental | Drug: nicotinamide adenine dinucleotid/nicotinamide mononucleotidenicotinamide adenine dinucleotide/nicotinamide mononucleotide |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age 18 and above, male or female;
- Conform to the diagnostic criteria of immune Thrombocytopenia (ITP) ≥3 months;
- Failure to achieve response or relapse after corticosteroid therapy, and failure to achieve response or relapse after previous second-line treatments such as TPO/TPORAs therapy, or are unable to afford the cost of the treatment;
- The platelet count of <30 X 10^9/L measured within 2 days prior to inclusion (During the screening visit and/or before receiving the study drug, platelet counts must be less than 30×10^9/L on at least two consecutive occasions, with a minimum interval of 1 day between the two tests.);
- ECOG physical state score ≤ 2 points;
Key exclusion criteria (19 listed)
- Those who are allergic to Nicotinamide adenine dinucleotide or excipients, or who have previously received CD38 monoclonal antibody treatment with no efficacy.
- Those with autoimmune hemolytic anemia, or various types of secondary and genetic thrombocytopenia, such as leukemia, lymphoma, multiple myeloma, aplastic anemia, myelodysplastic syndrome, Evans syndrome, common variable immunodeficiency, systemic lupus erythematosus, cirrhosis, antiphospholipid antibody syndrome, pseudo-thrombocytopenia, drug-induced thrombocytopenia (e.g., quinine, heparin, antimicrobial drugs, anticonvulsants, etc.).
- A history of any thrombosis or embolism events within 12 months prior to the first dose, or the presence of extensive and severe bleeding, such as hemoptysis, upper gastrointestinal bleeding, intracranial hemorrhage, sepsis, or other irregular bleeding.
- Participation in any other clinical trial involving investigational drugs (including vaccine studies) or exposure to other investigational drugs within 4 weeks or 5 half-lives (whichever is longer) prior to the first dose.
- Use of anticoagulants or any drugs with antiplatelet effects (e.g., aspirin) within 2 weeks prior to the first dose.
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 |
|---|---|---|
| nicotinamide adenine dinucleotid/nicotinamide mononucleotideAlso: Coenzyme I for Injection（NAD+）/NMNDrug: intravenous nicotinamide adenine dinucleotide (Coenzyme I for Injection) administration This study adopts a prospective, single arm, open design method. Twenty subjects were enrolled in the study and were treated with nicotinamide adenine dinucleotide (100mg/d) for 1 week. Drug: oral nicotinamide mononucleotide administration This study adopts a prospective, single arm, open design method. Twenty subjects were enrolled in the study and were treated with nicotinamide mononucleotide 450mg BID for 2 weeks. The first stage is the main research stage (d0-w2), which is the core treatment period. The aim is to observe the safety and efficacy during treatment. The second stage (w2-w8) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy after treatment. | Drug | Experimental: |
Primary outcome
Evaluation of overall efficacy response after nicotinamide adenine dinucleotide/NMN treatment within 2 weeks
Time frame: [Time Frame: 2 weeks]
Proportion of subjects with a platelet count ≥ 50 × 10^9/L (two consecutive measurements at least 1 day apart) within 2 weeks after initial administration in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period
incidence of treatment-emergent Adverse Events and TRAE
Time frame: [Time Frame: 8 weeks]
Incidence, severity, and relationship of treatment emergent adverse events after nicotinamide adenine dinucleotide/NMN treatment
8 secondary outcomes
Evaluation of the efficacy at each visit period
Time frame: [Time Frame: 8 weeks]
Duration from treatment initiation to platelet count ≥30×10^9/L (at least two times of baseline platelet count) and ≥50×10^9/L
Time frame: [Time Frame: 8 weeks]
Platelet count at each visit time point
Time frame: [Time Frame: 8 weeks]
Emergency treatment
Time frame: [Time Frame: 8 weeks]
Cumulative weeks of platelet ≥30×10^9/L (at least two times of baseline platelet count) and platelet ≥50×10^9/L
Time frame: [Time Frame: 8 weeks]
The change in bleeding score before treatment and 2 weeks after treatment assessed using the world health organization (WHO) bleeding scale
Time frame: [Time Frame: 2 weeks]
Evaluation of the efficacy during the first 4 weeks
Time frame: 4 weeks
The response rate within 4 weeks
Time frame: 4 weeks
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 5 Aug 2025.
- Derived from this study: PubMed 42056497Li H, Xu Y, Chen Y, Ji L, Xu Y, Zheng W, Sun T, Fu R, Pei X, Liu X, Xue F, Liu W, Wang W, Chi Y, Yang R, Wei J, Zhang L. Low-dose oral nicotinamide mononucleotide for immune thrombocytopenia: a phase 1/2 trial. Nat Med. 2026 Jun;32(6):2026-2036. doi: 10.1038/s41591-026-04366-x. Epub 2026 Apr 29.
- Individual participant dataSharing planned: Yes. Researchers qualified can request the dataset, including de-identified individual subject data. Data may be requested from PI from 12 months 36 months after study completion.
Related trials for Immune Thrombocytopenia
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
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.
Lei Zhang, MD principal investigator
Chinese Academy of Medical Science and Blood Disease Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing nicotinamide adenine dinucleotid/nicotinamide mononucleotide in people with Immune Thrombocytopenia and Treatment, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
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 8 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: looking for participants now, as of the sponsor's last update on 5 Aug 2025. 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 China. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Yunfei Chen, MD (+8618502220788, chenyunfei@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of overall efficacy response after nicotinamide adenine dinucleotide/NMN treatment within 2 weeks (time frame: [Time Frame: 2 weeks]). 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?
On the ClinicalTrials.gov record for NCT06776510, 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 5 Aug 2025; AzTrials last refreshed its copy on 8 Nov 2026.