Anti-CD38 Antibody Treating Pediatric Primary Immune Thrombocytopenia (ITP)

NCT06168851 | Recruiting Phase 2 DMC

• 1 other identifier: IIT2023072
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Immune Thrombocytopenia; Treatment

Outcome Measures

Primary Outcomes (2)

• 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

  8 weeks

• Safety of Anti-CD38 antibody treatment

  Incidence, severity, and relationship of treatment emergent adverse events after Anti-CD38 antibody treatment

  5 years

Secondary Outcomes (9)

• Other efficacy evaluation

  5 years

• Duration from treatment initiation to platelet count ≥30×10^9/L and ≥50×10^9/L

  12 weeks

• Cumulative weeks of platelet ≥30×10^9/L and platelet ≥50×10^9/L

  5 years

• Reduction of concomitant drug

  5 years

• Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale

  5 years

Other Outcomes (1)

• Prognostic model establishment using multi-omics data such as transcriptome, proteome, metabolome, microbiome, et al.

  5 years

Study Arms (1)

Experimental: Intervention (Anti-CD38 antibody)

EXPERIMENTAL

60 enrolled subjects : once a week x 8 doses

Drug: Anti-CD38 antibody Injection

Interventions

Anti-CD38 antibody Injection DRUG

intravenous 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.

Experimental: Intervention (Anti-CD38 antibody)

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age 6 years and above, male or female
• Conform to the diagnostic criteria of immune Thrombocytopenia (ITP)
• Failure to achieve response or relapse after corticosteroid therapy, and at least one second-line therapy including rituximab or TPORAs.
• The previous emergency treatment of ITP (e.g. methylprednisolone, platelet transfusion, IVIG transfusion) must be completed at least 2 weeks before the first administration
• Signed and dated written informed consent
• With normal hepatic and renal functions
• ECOG physical state score ≤ 2 points
• Cardiac function of the New York Society of Cardiac Function ≤ 2
• Patients receiving maintenance treatment (including corticosteroids (less than or equal to 0.5mg/kg prednisone), TPO receptor agonists, etc.) must have a stable dose at least 4 weeks before the first administration, and azathioprine, danazol, cyclosporin A, tacrolimus, sirolimus, etc. must be stopped at least 4 weeks before the first administration; The end of rituximab treatment was\>3 months；More than 6 months after splenectomy.
• March 26,2024 After approval by the Ethics Committee，age of subjects has been modified to 6 years and above upon enrollment. Approval Number: IIT2023072-EC-2.

You may not qualify if:

• 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;
• Accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.;
• At present, there are heart diseases, arrhythmias that need treatment or hypertension that researchers judge is poorly controlled;
• Patients with thrombotic diseases such as pulmonary embolism, thrombosis and atherosclerosis;
• Those who have received allogeneic stem cell transplantation or organ transplantation in the past;
• Patients with mental disorders who cannot normally obtain informed consent and conduct trials and follow-up;
• Patients whose toxic symptoms caused by pre-trial treatment have not disappeared;
• Other serious diseases that may limit the subject's participation in this test (such as diabetes; Severe cardiac insufficiency; Myocardial obstruction or unstable arrhythmia or unstable angina pectoris in recent 6 months; Gastric ulcer, etc.);
• Patients with septicemia or other irregular severe bleeding;
• Patients taking antiplatelet drugs at the same time;
• Pregnant women, suspected pregnancies (positive pregnancy test for human chorionic gonadotropin in urine at screening) and lactating patients.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• Beijing Children's Hospital: collaborator
• Tianjin Children's Hospital: collaborator

Study Sites (1)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

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

Study Officials

• Lei Zhang, MD

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ting Sun, MD

CONTACT

+8615822339131; sunting@ihcams.ac.cn

Yunfei Chen, MD

CONTACT

+8618502220788; chenyunfei@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 5, 2023
• First Posted: December 13, 2023
• Study Start: December 28, 2023
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2030
• Last Updated: November 28, 2025

Record last verified: 2024-12

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)