Skip to content

Trial · Interventional

RecruitingPhase 1/2Registry data

A Clinical Study of NAD in the Treatment of Immune Thrombocytopenia

Overview

Status
Recruiting
Phase
Phase 1/2
Enrollment
20 participants (estimated)
Sponsor
Institute of Hematology & Blood Diseases Hospital, China · Other
Design
Allocation not applicable · Open label (no masking)
Countries
1 country

China

Start
15 Jan 2025
Primary completion
1 Dec 2025 (estimated)
Completion
1 Mar 2026 (estimated)
First posted
15 Jan 2025
Last update posted
5 Aug 2025
Conditions
Immune Thrombocytopenia · Treatment

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
202520262027
  1. Ph 1/2

    A Clinical Study of NAD in the Treatment of Immune Thrombocytopenia

    NCT06776510

    15 Jan 2025 to est. 1 Mar 2026

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • nicotinamide adenine dinucleotid/nicotinamide mononucleotideDrug

Arms1

  • Experimental

    Experimental:

    nicotinamide adenine dinucleotide/nicotinamide mononucleotide

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. Evaluation of overall efficacy response after nicotinamide adenine dinucleotide/NMN treatment within 2 weeks

    Time frame · [Time Frame: 2 weeks]

    Registry description

    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

  2. incidence of treatment-emergent Adverse Events and TRAE

    Time frame · [Time Frame: 8 weeks]

    Registry description

    Incidence, severity, and relationship of treatment emergent adverse events after nicotinamide adenine dinucleotide/NMN treatment

Secondary outcomes(8)
  1. Evaluation of the efficacy at each visit period

    Time frame · [Time Frame: 8 weeks]

    Registry description

    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

  2. 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]

    Registry description

    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

  3. Platelet count at each visit time point

    Time frame · [Time Frame: 8 weeks]

    Registry description

    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.

  4. Emergency treatment

    Time frame · [Time Frame: 8 weeks]

    Registry description

    The proportion of subjects who received emergency treatment within 2 weeks and during the entire study period.

  5. 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]

    Registry description

    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

  6. 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]

    Registry description

    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.

  7. Evaluation of the efficacy during the first 4 weeks

    Time frame · 4 weeks

    Registry description

    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

  8. The response rate within 4 weeks

    Time frame · 4 weeks

    Registry description

    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).

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Clinical trial · 2026

13 results

Low-dose oral nicotinamide mononucleotide for immune thrombocytopenia: a phase 1/2 trial.

Li et al. 2026Nature medicinecited by 3

PMID 42056497DOI 10.1038/s41591-026-04366-xPUB-42056497

Full text at the publisher13 results

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * 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 Exclusion Criteria: * 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). * 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.

PepBank is a research reference. Talk to a doctor before you use any peptide.