Dose-escalation and dose-optimization of CID-103 for adults with chronic immune thrombocytopenia

A Dose-escalation and Safety Study of CID-103 Followed by a Randomized, Open-label, Parallel-arm Multi-dose Study Evaluating the Efficacy and Tolerability of CID-103 in Adults With Chronic Immune Thrombocytopenia

This trial will test CID-103, an anti-CD38 antibody given by IV, to see if it raises platelet counts in adults 18–65 with chronic ITP who have not responded adequately to prior treatments.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 75 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: CASI Pharmaceuticals, Inc. (Industry-sponsored)
• Drugs / interventions: rituximab
• Locations: 6 sites (Tangshan, Hebei and 5 other locations)
• Trial ID: NCT07017725 on ClinicalTrials.gov

What this trial studies

CID-103 is an experimental anti-CD38 monoclonal antibody given by intravenous infusion; the program starts with a Phase 1 dose-escalation (Part A) to define safety and tolerability and a Phase 2 dose-optimal stage (Part B) comparing up to three dose regimens. Eligible adults with chronic ITP and low platelet counts will be enrolled at sites in China. Key outcomes include adverse events, tolerability, and platelet count responses over time to identify an optimal dose and schedule for future studies. The protocol includes regular safety labs, scheduled IV administrations, and follow-up visits to monitor efficacy and side effects.

Who should consider this trial

Good fit: Adults 18–65 with chronic ITP (≥3 months), a mean platelet count ≤35 × 10^9/L on screening, a prior response to ITP therapy (excluding TPO-RAs), and at least two prior lines of systemic treatment are the intended participants.

Not a fit: Patients outside the 18–65 age range, those with platelet counts persistently above 35 × 10^9/L, or those who have not had at least two prior systemic treatment lines (or who only received TPO-RAs) are unlikely to be eligible or to benefit from this protocol.

Why it matters

Potential benefit: If successful, CID-103 could raise platelet counts, reduce bleeding risk, and lessen the need for other ITP therapies.

How similar studies have performed: Other anti-CD38 agents such as daratumumab have shown promise in case series for refractory autoimmune cytopenias including ITP, but CID-103 itself is novel and has not been proven in large trials.

Eligibility criteria

Inclusion Criteria:

1. Male or female individuals aged 18 to 65 years at time of signing of ICF. Disease-related.
2. Diagnosed with ITP that has persisted for ≥ 3 months, diagnosed in accordance with The American Society of Hematology 2019 Guidelines for Immune Thrombocytopenia or the Updated International Consensus Report on the Investigation and Management of Primary Immune Thrombocytopenia (as locally applicable).
3. Diagnosis of ITP supported by a prior response to an ITP treatment (other than a thrombopoietin receptor agonists \[TPO-RA\]) that achieved a platelet count of ≥ 30 x 10\^9/L and a doubling of baseline measurement.
4. Has received at least two lines of SOC systemic treatment (i.e., corticosteroids and one other agent).
5. Has a mean platelet count ≤ 35 x 10\^9/L on at least two measurements at least one week apart during screening.
6. If receiving standard background treatment for ITP, treatment should be stable in dose and frequency for at least four weeks prior to first dose of CID-103.
7. Adequate organ function.
8. Contraception: Female participants must either be non-pregnant or not breastfeeding and must have a negative pregnancy test. Male and female participants must meet the contraceptive requirements.

Exclusion Criteria:

1. Prior treatment with any anti-CD38 agent, or has been treated with anti-Bruton's tyrosine kinase (BTK), neonatal Fc receptor (FcRn) antagonist or complement inhibitor within three months prior to first dose of CID-103.
2. Use of IV immunoglobulin, subcutaneous immunoglobulin or anti-D immunoglobulin treatment within four weeks of screening.
3. Treatment with rituximab or splenectomy within the three months prior to first dose of CID-103.
4. Use of anticoagulants or any drug with antiplatelet effect (such as aspirin) within three weeks before screening.
5. Receiving other concurrent investigational therapies or have received investigational therapies within four weeks of the first dose of CID-103 or five half-lives (if shorter).
6. Active hemolytic anemia.
7. Diagnosed with severe chronic obstructive pulmonary disease (COPD), Global Initiative for Chronic Obstructive Lung Disease (GOLD Stage 3 or 4) or asthma.
8. Has been diagnosed with myelodysplastic syndrome or other active malignancy.
9. Known / clinically significant amyloidosis.
10. Has a history of any thrombotic or embolic event within six months before screening.
11. A history or evidence of cardiovascular risk including left ventricular ejection fraction \< 50%, clinically significant uncontrolled ventricular arrhythmia, acute coronary syndrome history, coronary angioplasty or stenting within six months, current ≥ Class III congestive heart failure (NYHA guidelines), and treatment refractory hypertension.
12. Clinically significant medical history or ongoing chronic illness.
13. Known active infection with hepatitis B (HBV) (surface antigen) or infection with hepatitis C (HCV) in absence of sustained virologic response.
14. History of known or suspected immunosuppression.
15. Known active infection with human immunodeficiency virus (HIV) and CD4+ T cell count \< 350/μL.
16. Karnofsky Performance Status ≤ 70.

Where this trial is running

Tangshan, Hebei and 5 other locations

• North China University of Science and Technology Affiliated Hospital — Tangshan, Hebei, China (Recruiting)
• Henan Cancer Hospital — Zhengzhou, Henan, China (Not_yet_recruiting)
• The First Affiliated Hospital of Nanchang University — Nanchang, Jiangxi, China (Not_yet_recruiting)
• Qilu Hospital of Shandong University — Jinan, Shandong, China (Not_yet_recruiting)
• Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences — Tianjin, Tianjin Municipality, China (Recruiting)
• The Second Affiliated Hospital of Kunming Medical University — Kunming, Yunnan, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Aaron Yang

Conditions Chronic Immune Thrombocytopenia; Purpura; Thrombocytopenic; Idiopathic