Platelet RefractorinessEarly Phase 1NCT05284032
A Pilot Trial Using Isatuximab to Overcome Platelet Transfusion Refractoriness in Human Leukocyte Antigen Allo-Immunized Patients (SuppCare 001)
A Early Phase 1 study testing isatuximab 10 mg/kg in people with Platelet Refractoriness and Hematologic Malignancy, run by Firas El Chaer, MD at 1 site in United States.
Trial summary
Some of the treatments for cancer can cause platelets (the part of the blood that helps with clotting) to decrease. If they are too low, then clinicians may recommend a transfusion (getting platelets from another person added to someone else's body). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Platelet Refractoriness, Hematologic Malignancy
- What is being tested?
- isatuximab 10 mg/kg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Nov 2022to 11 Jun 2024
- Phase
- Early 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, age ≥ 18 years
- Diagnosis of immune mediated platelet transfusion refractoriness secondary to class I anti-HLA antibodies according to institutional practice, including calculated percent panel-reactive antibodies (%PRA) > 80%
- Adequate Organ Function:
- serum creatinine <= 1.5 x upper limit of normal
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- bilirubin <= 1.5 x upper limit of normal (exceptions for Gilbert's disease)
- AST and ALT <= 2.5 x upper limit of normal
- Alkaline phosphatase <= 2.5 x upper limit of normal
- For females and males of reproductive potential: agreement to use adequate contraception (see section 5.3)
- Agreement to adhere to Lifestyle Considerations (see Section 5.3) throughout study duration
Who cannot join
- Immune-mediated platelet refractoriness other than anti-HLA antibody-mediated
- Non-immune-mediated platelet refractoriness (e.g. splenomegaly or disseminated intravascular coagulation)
- Diagnosis of thrombocytopenia induced by other drugs, such as vancomycin, heparin, or amphotericin
- Diagnosis of thrombotic thrombocytopenic purpura or idiopathic immune thrombocytopenia
- Active bleeding
- Greater than Grade 2 active graft versus host disease (GVHD) following allogeneic HSCT
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- Bi-directional ABO mismatched allogeneic stem cell transplantation
- Prior administration of daratumumab, isatuximab or any other anti-CD38 antibodies
- Known uncontrolled HIV disease and/or active Hepatitis A, B, or C infection
- Active systemic infection and severe infections requiring treatment with a parenteral administration of antimicrobials.
- Controlled systemic infections on antimicrobial therapy that are stable at the time of screening are not an exclusion criterion.
- Hypersensitivity or history of intolerance to steroids, mannitol, pregelatinized starch, sodium stearyl fumarate, histidine (as base and hydrochloride salt), arginine hydrochloride, poloxamer 188, sucrose or any of the other components of study intervention that are not amenable to premedication with steroids and H2 blockers or would prohibit further treatment with these agents.
- Received any investigational drug within 14 days or 5 half-lives of the investigational drug prior to initiation of study intervention, whichever is longer. In case of very aggressive disease (i.e acute leukemia) delay could be shortened after agreement between sponsor and investigator, in absence of residual toxicities from previous therapy
- Pregnancy or lactation
- Any clinically significant, uncontrolled medical conditions that, in the Investigator's opinion, would expose the patient to excessive risk or may interfere with compliance or interpretation of the study results.
- Current receipt of, or expectation to require anti-CD20 therapy, proteasome inhibitors, intravenous immune globulin ("IVIG"), and plasma exchange therapy during the study
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Virginia
Charlottesville, Virginia, 22903
About this study
Official title: A Pilot Trial Using Isatuximab to Overcome Platelet Transfusion Refractoriness in Human Leukocyte Antigen Allo-Immunized Patients
Some of the treatments for cancer can cause platelets (the part of the blood that helps with clotting) to decrease. If they are too low, then clinicians may recommend a transfusion (getting platelets from another person added to someone else's body). This usually works to increase the person's platelets to a healthy level, but sometimes it doesn't work. This is called platelet refractoriness. This study is trying to find out whether isatuximab (the study drug) may help people with a certain type of platelet refractoriness by removing some cells in order to make platelet transfusions more effective.
Read the detailed description
Participants in this study will receive 4 weekly infusions of the study drug, isatuximab, by intravenous infusion. The dose of isatuximab infusions may be larger or smaller and take a longer or shorter time to infuse depending on your weight and time required will decrease from the first to second infusion and from the second to third and fourth infusion. Participants will be observed for 2 hours after each infusion. Participants will continue to receive platelet transfusions according to standard clinical care and will be followed for about 120 days after their last dose of isatuximab.
- Conditions
- Platelet Refractoriness, Hematologic Malignancy
- Being tested
- isatuximab 10 mg/kg
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
isatuximab 10 mg/kgDrug
Given by intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| Isatuximab (Sarclisa) | Experimental | 4 weekly doses of isatuximab Receives: Drug: isatuximab 10 mg/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent panel-reactive antibodies (PRAs)- change over time/with study treatment
A weighted percent of class I HLA targets to which the patient has made antibodies
Measured: Through about 120 days following last study drug infusion
Secondary outcomes (3)
Mean fluorescence intensity (MFI) - change over time/ with study treatment
MFI of each class I anti-HLA antibody contributing to the %PRA
Measured: Through about 120 days following last study drug infusion
Quality of life - changes over time/with study treatment according to the Functional Assessment of Cancer Therapy - Leukemia (FACT-Leu)
Each question is scored on a 5-point scale (0 - 4), with a mixture of questions scored with low numbers indicating better quality of life and others indicating worse quality
Measured: Through about 120 days following last study drug infusion
Adverse events
Frequency, severity (by CTCAE v5), and duration of Grade 3 or higher adverse events considered related to the study intervention
Measured: Through about 30 days following last study drug infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2022
- Study start29 Nov 2022
- Primary completion11 Jun 2024
- Study completion11 Jun 2024
Why it stopped: Slow accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Platelet RefractorinessStudies of the same condition, open and under way.
- 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
- Early Phase 1
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Firas El Chaer, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Firas El Chaer, MD, University of Virginia
- Investigators
- Firas El Chaer, MD, Principal investigator, UVA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05284032
- Sponsor's protocol ID
- HSR210463
- Status last verified
- Jul 2024
- First submitted
- 8 Mar 2022
- Last updated
- 31 Jul 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Trial Using Isatuximab to Overcome Platelet Transfusion Refractoriness in Human Leukocyte Antigen Allo-Immunized Patients
- ClinicalTrials.gov ID
- NCT05284032
- Acronym / sponsor ID
- HSR210463
- Conditions
- Platelet Refractoriness, Hematologic Malignancy
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow accrual
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Firas El Chaer, MDUniversity, hospital or other
- Study start
- 29 Nov 2022 (actual)
- Primary completion
- 11 Jun 2024 (actual)
- Study completion
- 11 Jun 2024 (actual)
- First posted
- 17 Mar 2022 (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.
Some of the treatments for cancer can cause platelets (the part of the blood that helps with clotting) to decrease. If they are too low, then clinicians may recommend a transfusion (getting platelets from another person added to someone else's body). This usually works to increase the person's platelets to a healthy level, but sometimes it doesn't work. This is called platelet refractoriness. This study is trying to find out whether isatuximab (the study drug) may help people with a certain type of platelet refractoriness by removing some cells in order to make platelet transfusions more effective.
Detailed description
Participants in this study will receive 4 weekly infusions of the study drug, isatuximab, by intravenous infusion. The dose of isatuximab infusions may be larger or smaller and take a longer or shorter time to infuse depending on your weight and time required will decrease from the first to second infusion and from the second to third and fourth infusion. Participants will be observed for 2 hours after each infusion. Participants will continue to receive platelet transfusions according to standard clinical care and will be followed for about 120 days after their last dose of isatuximab.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Isatuximab (Sarclisa) | Experimental | Drug: isatuximab 10 mg/kg4 weekly doses of isatuximab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, age ≥ 18 years
- Diagnosis of immune mediated platelet transfusion refractoriness secondary to class I anti-HLA antibodies according to institutional practice, including calculated percent panel-reactive antibodies (%PRA) > 80%
- Adequate Organ Function:
Key exclusion criteria (16 listed)
- Immune-mediated platelet refractoriness other than anti-HLA antibody-mediated
- Non-immune-mediated platelet refractoriness (e.g. splenomegaly or disseminated intravascular coagulation)
- Diagnosis of thrombocytopenia induced by other drugs, such as vancomycin, heparin, or amphotericin
- Diagnosis of thrombotic thrombocytopenic purpura or idiopathic immune thrombocytopenia
- Active bleeding
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 |
|---|---|---|
| isatuximab 10 mg/kgGiven by intravenous infusion | Drug | Isatuximab (Sarclisa) |
Primary outcome
Percent panel-reactive antibodies (PRAs)- change over time/with study treatment
Time frame: Through about 120 days following last study drug infusion
A weighted percent of class I HLA targets to which the patient has made antibodies
3 secondary outcomes
Mean fluorescence intensity (MFI) - change over time/ with study treatment
Time frame: Through about 120 days following last study drug infusion
Quality of life - changes over time/with study treatment according to the Functional Assessment of Cancer Therapy - Leukemia (FACT-Leu)
Time frame: Through about 120 days following last study drug infusion
Adverse events
Time frame: Through about 30 days following last study drug infusion
Results and safety
The study was stopped early (reason given: Slow accrual). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2024.
- Individual participant dataSharing planned: No
Related trials for Platelet Refractoriness
RecruitingPhase 1
RN1201 Injection in the Treatment of Antibody-Mediated Diseases
Refractory Immune-mediated Platelet Transfusion Refractoriness, Relapsed or Refractory Immune Thrombocytopenia
Not yet recruitingPhase 3
Acalabrutinib Maleate and Bortezomib for Patients With HLA Antibodies
Hematological Malignancies, Platelet Transfusion Refractoriness (PTR)
RecruitingPhase 1/2
Study of CM313(SC) Injection in Subjects With Platelet Transfusion Refractoriness
Platelet Transfusion Refractoriness
RecruitingPhase 2
Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness
Severe Aplastic Anemia, Platelet Transfusion Refractoriness
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.
Firas El Chaer, MD principal investigator
UVA
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing isatuximab 10 mg/kg in people with Platelet Refractoriness and Hematologic Malignancy, run by Firas El Chaer, MD at 1 site in United States.
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 11 inclusion and 16 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: stopped early, as of the sponsor's last update on 31 Jul 2024. 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 United States. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 was stopped early (reason given: Slow accrual). Results are not available in the source record.
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?
Percent panel-reactive antibodies (PRAs)- change over time/with study treatment (time frame: Through about 120 days following last study drug infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Jul 2024.