A Clinical Study of NAD in the Treatment of Immune Thrombocytopenia

NCT06776510 | Recruiting Phase 1 DMC

• 1 other identifier: IIT2024089
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Immune Thrombocytopenia; Treatment

Outcome Measures

Primary Outcomes (2)

• 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

  [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

  [Time Frame: 8 weeks]

Secondary Outcomes (8)

• Evaluation of the efficacy at each visit period

  [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: 8 weeks]

• Platelet count at each visit time point

  [Time Frame: 8 weeks]

• Emergency treatment

  [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: 8 weeks]

Study Arms (1)

Experimental:

EXPERIMENTAL

nicotinamide adenine dinucleotide/nicotinamide mononucleotide

Drug: nicotinamide adenine dinucleotid/nicotinamide mononucleotide

Interventions

nicotinamide adenine dinucleotid/nicotinamide mononucleotide DRUG

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.

Also known as: Coenzyme I for Injection（NAD+）/NMN

Experimental:

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Nicotinamide Mononucleotide; NAD

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Ribonucleotides; Nucleotides; Nucleic Acids, Nucleotides, and Nucleosides; Adenine Nucleotides; Purine Nucleotides; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Coenzymes; Enzymes and Coenzymes

Study Officials

• Lei Zhang, MD

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yunfei Chen, MD

CONTACT

+8618502220788; chenyunfei@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2025
• First Posted: January 15, 2025
• Study Start: January 15, 2025
• Primary Completion: December 1, 2025
• Study Completion: March 1, 2026
• Last Updated: August 5, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: 12 months to 36 months after study completion
• Access Criteria: Upon request to PI

Locations

CN (1)