Anti-CD38 Antibody Treating Pediatric Primary Immune Thrombocytopenia (ITP)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age6-17
SponsorInstitute of Hematology & Blood Diseases Hospital, China
About this trial
To evaluate the safety and efficacy of Anti-CD38 Antibody in the treatment of pediatric primary 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, or those in whom no other second-line treatment options are suitable.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age 6 years and above, male or female
Conform to the diagnostic criteria of immune Thrombocytopenia (ITP)
Diagnosis of ITP ≥3 months, and with a platelet count of <30 X 10^9/L measured within 2 days prior to inclusion
Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy including rituximab or TPORAs.
Disqualifiers
Allergy to daratumumab or its excipients, or prior treatment with daratumumab that was refractory or achieved a response lasting <6 months；
Uncontrollable primary diseases of important organs, such as malignant tumors, liver failure, heart failure, renal failure and other diseases;
HIV positive;
Accompanied by uncontrollable active infection, including hepatitis B, hepatitis C, cytomegalovirus, EB virus and syphilis positive;
Trial design
Design model
Single group
Treatments tested in this trial
Anti-CD38 antibody Injection
Drugintravenous Anti-CD38 antibody administration This study adopts a prospective, single arm, open design method. Twenty subjects were enrolled in the study and were treated with Anti-CD38 antibody (16mg/kg/w) for 8 weeks. The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of 16mg/kg Anti-CD38 antibody once a week for 8 weeks to observe the safety and efficacy during treatment. The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of Anti-CD38 antibody after treatment. The third stage (w 25 to y 5): Extension visit phase, primarily to observe the long-term safety and sustained efficacy following CD38 monoclonal antibody therapy.
Treatment groups
60 Participants
are divided into 1 treatment groupGroup A: Experimental: Intervention (Anti-CD38 antibody)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Evaluation of overall efficacy response after Anti-CD38 antibody treatment within 8 weeks
Proportion of subjects with a platelet count ≥ 50 × 10\^9/L within 8 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
8 weeks2
Safety of Anti-CD38 antibody treatment
Incidence, severity, and relationship of treatment emergent adverse events after Anti-CD38 antibody treatment
Time frame
5 yearsSecondary outcomes
1
Other efficacy evaluation
Including: 1. Proportion of subjects with a platelet count ≥ 50 × 10\^9/L at week 2, week 4, week 6，week 8，week 10, week 12 and other visit throughout the study period in absence of rescue therapy, and without having had dose increment of TPO-RA or corticosteroids during the study period; 2. 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 12 weeks and other visit throughout the study period; 3. 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 8 weeks and other visit throughout the study period（two consecutive measurements at least 7 days apart）.
Time frame
5 years2
Duration from treatment initiation to platelet count ≥30×10^9/L and ≥50×10^9/L
Duration from treatment initiation to platelet count ≥30×10\^9/L 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
12 weeks3
Cumulative weeks of platelet ≥30×10^9/L and platelet ≥50×10^9/L
Cumulative weeks of platelet ≥30×10\^9/L and platelet ≥50×10\^9/L without having received any platelet elevating therapy or having had dose increment of TPO-RA and/or corticosteroids
Time frame
5 years4
Reduction of concomitant drug
Percentage of patients with reduced doses of corticosteroids and/or other concomitant immunosuppressive drugs at baseline by 24 weeks of Anti-CD38 antibody treatment
Time frame
5 yearsOther outcomes
1
Prognostic model establishment using multi-omics data such as transcriptome, proteome, metabolome, microbiome, et al.
The prognosis related factors will be selected from multi-omics data such as transcriptome, proteome, metabolome, microbiome, et al. And then selected prognosis related factors will be used to establish prognosis prediction model
Time frame
5 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
Beijing Children's Hospital
Collaborator
Tianjin Children's Hospital
Collaborator
Source ClinicalTrials.gov