A Clinical Study of NAD in the Treatment of Immune Thrombocytopenia
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorInstitute of Hematology & Blood Diseases Hospital, China
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.);
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
nicotinamide adenine dinucleotid/nicotinamide mononucleotide
DrugDrug: 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.
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: Experimental:Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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
[Time Frame: 2 weeks]2
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
[Time Frame: 8 weeks]Secondary outcomes
1
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
Time frame
[Time Frame: 8 weeks]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
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
Time frame
[Time Frame: 8 weeks]3
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.
Time frame
[Time Frame: 8 weeks]4
Emergency treatment
The proportion of subjects who received emergency treatment within 2 weeks and during the entire study period.
Time frame
[Time Frame: 8 weeks]Sponsors and contacts
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Source ClinicalTrials.gov