Aplastic AnemiaPhase 1NCT05832216
Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients with Aplastic Anemia
A Phase 1 study testing Daratumumab in people with Aplastic Anemia and Platelet Transfusion Refractoriness, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
This is a phase 1, prospective, single-arm, open-label study. The aim of this study is to evaluate the transfusion responses of platelet increment by using Daratumumab among aplastic anemia patients with platelet transfusion refractoriness. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Aplastic Anemia, Platelet Transfusion Refractoriness
- What is being tested?
- Daratumumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 17 Apr 2023to 1 Dec 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of aplastic anemia (AA)
- Dependent on platelet transfusions, characterized by either PLT<10×10^9/L, or PLT<20×10^9/L with bleeding events.
- Diagnosed with platelet transfusion refractoriness, characterized by Corrected count increment (CCI) <7500/ul at 60 min, or CCI <4500/ul at 24 hrs.
- Male or female age ≥ 12 years
- ECOG performance status ≤2
- Willing and able to comply with the requirements for this study and written informed consent.
Who cannot join
- The inherited bone marrow failure syndromes
- The presence of hemolytic PNH clone
- Combination of either radiotherapy or chemotherapy for solid tumors in recent 3 years, excluding the local tumor diagnosed 1 year ago and cured by surgical resection.
- Cytopenia caused by other diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc.
- Uncontrolled infection
- HIV, HCV or HBV active infection
Show all 24Show fewer
- The presence of any of the following bleeding events:
- Gastrointestinal bleeding
- Respiratory tract hemorrhage
- Central nervous system bleeding
- Abnormal liver function: ALT or AST > 2 ULN, or TBil > 2 ULN after treatment.
- Abnormal kidney function: Creatinine clearance < 30 ml/min
- Heart failure (NYHA class III or IV)
- Poorly controlled diabetes, characterized by fasting blood glucose > 8.8mmol/L or post-meal blood glucose > 11.1mmol/L after therapy with insulin or oral hypoglycemic agents
- History of congestive heart failure, unstable angina pectoris, myocardial infarction, arterial or venous thrombosis
- Pregnant or breast-feeding patients
- Had a history of any psychiatric diseases, cerebrovascular disease or cognitive sequelae of head injury.
- Participation in another clinical trial within 4 weeks before the start of this trial
- Have an allergy to Daratumumab or any other part of this medicine.
- Previously treated with Daratumumab
- Previously treated with ATG/ALG within 4 months before the start of this trial
- Previously treated with the anti-CD20 monoclonal antibody within 2 months before the start of this trial
- Currently treated with TPO-RA except for a minimum of 4 weeks for washout before the start of this trial
- Patients considered to be ineligible for the study by the investigator for reasons other than above
Trial locations and contacts
1 site in 1 country.
About this study
This is a phase 1, prospective, single-arm, open-label study. The aim of this study is to evaluate the transfusion responses of platelet increment by using Daratumumab among aplastic anemia patients with platelet transfusion refractoriness.
- Being tested
- Daratumumab
- Keywords
- Platelet transfusion refractoriness, Daratumumab
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DaratumumabDrug
Daratumumab is a monoclonal globulin targeting the CD38 molecule of immune cells, which can eliminate antigen-presenting cells such as dendritic cells and macrophages, and indirectly reduce the production of autoantibodies. In addition, Daratumumab reduces autoantibody levels by targeting plasma cells, B lymphocytes and T lymphocytes.
| Study group | Role | What they receive |
|---|---|---|
| Daratumumab | Experimental | Not described Receives: Drug: Daratumumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients with platelet transfusion responsiveness
To evaluate the safety and efficacy of Daratumumab to increase the platelet increment, defined as Corrected Count Increment (CCI) ≥7500/μL at 60 min or CCI≥4500/μL at 24 hrs post transfusion, or platelet transfusion independence, i.e. PLT>10×10^9/L without any bleeding events.
Measured: 12 weeks
Secondary outcomes (1)
Time to the platelet increment
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2023
- Study start17 Apr 2023
- Primary completion12 Oct 2024
- Study completion1 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05832216
- Sponsor's protocol ID
- IIT2023015
- Status last verified
- Jan 2025
- First submitted
- 3 Apr 2023
- Last updated
- 3 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Gao Z, Pan H, Zhang L, Li W, Li R, Zhao J, Luo Y, Lian Y, Yu X, Kuang Z, Nie N, Li J, Huang J, Zhao X, Li Y, Fang L, Ge M, Zheng Y, Shi J. Targeting CD38 with daratumumab for platelet transfusion refractoriness in aplastic anemia. Blood. 2025 Jun 26;145(26):3189-3193. doi: 10.1182/blood.2025029006.
PubMed 40331918
Study at a glance
From the registry record, last updated by the sponsor on 3 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Daratumumab to Overcome Platelet Transfusion Refractoriness in Patients with Aplastic Anemia
- ClinicalTrials.gov ID
- NCT05832216
- Acronym / sponsor ID
- IIT2023015
- Keywords
- Platelet transfusion refractoriness, Daratumumab
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 17 Apr 2023 (actual)
- Primary completion
- 12 Oct 2024 (actual)
- Study completion
- 1 Dec 2024 (actual)
- First posted
- 27 Apr 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 1, prospective, single-arm, open-label study. The aim of this study is to evaluate the transfusion responses of platelet increment by using Daratumumab among aplastic anemia patients with platelet transfusion refractoriness.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Daratumumab | Experimental | Drug: Daratumumab |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of aplastic anemia (AA)
- Dependent on platelet transfusions, characterized by either PLT<10×10^9/L, or PLT<20×10^9/L with bleeding events.
- Diagnosed with platelet transfusion refractoriness, characterized by Corrected count increment (CCI) <7500/ul at 60 min, or CCI <4500/ul at 24 hrs.
- Male or female age ≥ 12 years
- ECOG performance status ≤2
Key exclusion criteria (24 listed)
- The inherited bone marrow failure syndromes
- The presence of hemolytic PNH clone
- Combination of either radiotherapy or chemotherapy for solid tumors in recent 3 years, excluding the local tumor diagnosed 1 year ago and cured by surgical resection.
- Cytopenia caused by other diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc.
- Uncontrolled infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DaratumumabDaratumumab is a monoclonal globulin targeting the CD38 molecule of immune cells, which can eliminate antigen-presenting cells such as dendritic cells and macrophages, and indirectly reduce the production of autoantibodies. In addition, Daratumumab reduces autoantibody levels by targeting plasma cells, B lymphocytes and T lymphocytes. | Drug | Daratumumab |
Primary outcome
Percentage of patients with platelet transfusion responsiveness
Time frame: 12 weeks
To evaluate the safety and efficacy of Daratumumab to increase the platelet increment, defined as Corrected Count Increment (CCI) ≥7500/μL at 60 min or CCI≥4500/μL at 24 hrs post transfusion, or platelet transfusion independence, i.e. PLT>10×10^9/L without any bleeding events.
1 secondary outcome
Time to the platelet increment
Time frame: 12 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jan 2025.
- Derived from this study: PubMed 40331918Gao Z, Pan H, Zhang L, Li W, Li R, Zhao J, Luo Y, Lian Y, Yu X, Kuang Z, Nie N, Li J, Huang J, Zhao X, Li Y, Fang L, Ge M, Zheng Y, Shi J. Targeting CD38 with daratumumab for platelet transfusion refractoriness in aplastic anemia. Blood. 2025 Jun 26;145(26):3189-3193. doi: 10.1182/blood.2025029006.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Daratumumab in people with Aplastic Anemia and Platelet Transfusion Refractoriness, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of patients with platelet transfusion responsiveness (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05832216, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jan 2025.