Low-dose oral nicotinamide mononucleotide for immune thrombocytopenia: a phase 1/2 trial.
Huiyuan Li, Yuan Xu, Yunfei Chen...
https://pubmed.ncbi.nlm.nih.gov/42056497Actively Recruiting
Phase 1
Phase 2
Age: 18Years +
All Genders
ID06776510
A Prospective, Open-Label Study to Assess Safety and Efficacy of Nicotinamide Adenine Dinucleotide and Nicotinamide Mononucleotide in Primary Immune Thrombocytopenia
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-08-05
20
Participants Needed
1
Research Sites
12 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Immune thrombocytopenia (ITP) is an autoimmune disease that causes a low platelet count leading to bleeding in the skin and mucous membranes. This trial studies the safety and effectiveness of nicotinamide adenine dinucleotide (NAD+) and nicotinamide mononucleotide (NMN) for treating ITP in patients who have not responded well or have relapsed after first-line and at least one second-line therapy such as Anti-CD20 antibody or TPO receptor agonists. The study addresses the need for better treatments for adults with recurrent or difficult-to-treat ITP, a condition that severely impacts quality of life. Participants receive intravenous NAD+ (100 mg daily for 1 week) and oral NMN (450 mg twice daily for 2 weeks) in an open, single-arm design. The study has two stages: the first 2 weeks focus on treatment safety and efficacy, followed by a 6-week observation period to monitor ongoing safety and effects after stopping the treatment. During the trial, participants will have regular blood tests to measure platelet counts and bleeding scores, along with tracking any side effects for up to 8 weeks. Researchers will evaluate how well the treatment increases platelet levels and reduces bleeding symptoms. The study also monitors adverse events and treatment tolerability. Overall participation lasts about 8 weeks, including treatment and follow-up assessments.
CONDITIONS
Brief Title
A Clinical Study of NAD in the Treatment of Immune Thrombocytopenia
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Age 18 years or older, male or female
- Diagnosed with immune thrombocytopenia (ITP) for at least 3 months
- Have failed to respond or relapsed after corticosteroid therapy and at least one second-line treatment such as TPO receptor agonists or rituximab
- Platelet count less than 30 x 10^9/L on at least two occasions within 2 days prior to inclusion
- ECOG physical state score 2 or less
- On stable dose of one maintenance therapy (corticosteroids or TPO receptor agonists) for at least 4 weeks prior to first study dose
- Negative pregnancy test for women of childbearing potential and use of effective contraception for both sexes during study and for several months after
- Signed informed consent form
You will not qualify if you...
- Allergy to nicotinamide adenine dinucleotide or excipients, or prior ineffective CD38 monoclonal antibody treatment
- Presence of autoimmune hemolytic anemia or secondary/genetic thrombocytopenias such as leukemia, lymphoma, multiple myeloma, aplastic anemia, myelodysplastic syndrome, Evans syndrome, or systemic lupus erythematosus
- History of thrombosis or embolism within 12 months, or severe bleeding events
- Participation in other investigational drug trials within 4 weeks prior to enrollment
- Use of anticoagulants or antiplatelet drugs within 2 weeks prior to enrollment
- Emergency ITP treatment within 2 weeks prior to enrollment
- Use of certain immunosuppressive drugs within 3-4 months prior to enrollment
- Splenectomy within 6 months prior to enrollment
- Recent or planned live vaccinations during study
- History of stem cell or organ transplantation
- Clinically significant diseases posing safety risks
- Malignant tumors within past 5 years (except certain cured skin and cervical cancers)
- Abnormal liver or kidney function at screening
- Positive HIV, syphilis, hepatitis B or C infections
- Pregnant or breastfeeding women, or men with partners planning pregnancy
- Mental disorders impairing consent or participation
- Unresolved prior treatment toxicities
- Other conditions judged unsuitable by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - 3 weeks
Participants receive intravenous nicotinamide adenine dinucleotide daily for 1 week followed by oral nicotinamide mononucleotide twice daily for 2 weeks to evaluate safety and efficacy.
Weekly visits for 3 weeks
Follow-up
Duration - 6 weeks
Participants are monitored for safety and continued efficacy after treatment withdrawal.
Visits approximately every 2 weeks for 6 weeks
Trial Site Locations
Total: 1 location
1
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
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Research Team
Y
Yunfei Chen, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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