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New treatment option for Sickling Disorder Due to Hemoglobin S
Official title 131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)
ClinicalTrials.gov ID: NCT07015684
What this study is testing
What is 131I-apamistmab?
131I-apamistmab is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for sickling disorder due to hemoglobin s.
Also referred to as Iodine (131I) apamistamab.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to find the smallest amount of the 131 I-apamistamab needed for preparing patients with severe sickle cell disease (SCD) for a bone marrow transplant. This is the first time 131 I-apamistamab is being used for advanced Sickle Cell Disease (SCD) in the setting of allogeneic stem cell transplant.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 50
You may be able to join if
- Availability of an HLA-matched sibling donor
- Patients with sickle cell anemia (Hb SS, Sβ0 thalassemia or severe SC) who are 12 - 50 years of age inclusive AND who have 1 or more of the following:
- Clinically significant neurologic event (stroke) or any neurological deficit lasting at least 24 hours. Stroke will be defined as a clinically...
- History of two or more episodes of ACS in the 2-year period preceding enrollment despite supportive care measures (i.e. asthma therapy and/or...
- History of three or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive...
You likely can't join if
- Pulmonary dysfunction defined as DLCO (corrected for hemoglobin and alveolar volume) \< 40% of predicted OR baseline oxygen saturation of \<85% or...
- Severe cardiac dysfunction defined as ejection fraction \<35%.
- Impaired renal function defined as GFR \<40.
- Hepatic dysfunction defined as bridging (portal to portal) fibrosis or cirrhosis of the liver OR transaminases \>5x ULN for age.
- Clinical stroke within 6 months of anticipated transplant
- Karnofsky performance score \< 50%
See the full eligibility criteria
- Availability of an HLA-matched sibling donor
- Patients with sickle cell anemia (Hb SS, Sβ0 thalassemia or severe SC) who are 12 - 50 years of age inclusive AND who have 1 or more of the following:
- Clinically significant neurologic event (stroke) or any neurological deficit lasting at least 24 hours. Stroke will be defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral...
- History of two or more episodes of ACS in the 2-year period preceding enrollment despite supportive care measures (i.e. asthma therapy and/or hydroxyurea).
- History of three or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with...
- Administration of regular RBC transfusion therapy, defined as receiving 8 or more transfusions per year for 1 year or more to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and ACS)
- An echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity \> or equal to 2.7 m/sec or pulmonary hypertension diagnosed by right heart catherization.
- Sickle hepatopathy defined as EITHER ferritin \>1000mcg/L OR direct bilirubin \>0.4mg/dl but \<5xULN AND platelet count \<250,000/uL at baseline
- Adequate organ functions as defined as:
- ECOG performance status of 2 or better
- Cardiac function: LVEF of 40% or greater
- Pulmonary Function: Pulse oximetry with a baseline oxygen saturation of 85% or greater and corrected DLCO of 40% or greater
- Hepatic Function: Serum conjugated (direct) bilirubin less than 5x upper limit of normal for age as per local laboratory, ALT and AST less than 5 x upper limit of normal as per local laboratory. Patients whose...
- Absence of liver cirrhosis, bridging fibrosis and active hepatitis as documented by liver biopsy for patients with evidence of iron overload by serum ferritin or MRI. The histological grading and scale described by...
- Donor should be evaluated for eligibility to donate by an independent physician not directly caring for the patient on study protocol.
- Donor is willing to sign informed consent allowing the use of the PBSC product for the HSCT of the recipient.
- Donor cannot be pregnant or lactating and must agree to contraception until after the donation procedure is complete.
- Testing negative for HIV and viral hepatitis
- Free of Hb S (defined as Hb S less than 50%) and other hemoglobinopathies that are symptomatic or of clinical significance.
- Targeted minimum stem cell dose of 5.0 x 10e6 CD34 cells/Kg of recipient weight
- Fulfills standard criteria for eligibility as a donor for HSCT. Note: HSCT can be deleterious for the developing fetus and pregnant mother due to the conditioning regimen, GVHD prophylaxis and treatment. Agents used in...
- Pulmonary dysfunction defined as DLCO (corrected for hemoglobin and alveolar volume) \< 40% of predicted OR baseline oxygen saturation of \<85% or PaO2 \<70.
- Severe cardiac dysfunction defined as ejection fraction \<35%.
- Impaired renal function defined as GFR \<40.
- Hepatic dysfunction defined as bridging (portal to portal) fibrosis or cirrhosis of the liver OR transaminases \>5x ULN for age.
- Clinical stroke within 6 months of anticipated transplant
- Karnofsky performance score \< 50%
- HIV infection
- Uncontrolled viral, bacterial, fungal, or protozoal infection at the time of study enrollment.
- Have circulating HAMA noted on initial screening.
- Have received prior radiation to maximally tolerated levels to any critical normal organs
- Patients with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate HSCT.
- Patients' unable to understand the nature and risks inherent in the HSCT process.
- History of non-compliance severe enough in the estimation of the treating team to preclude the patient from undergoing unrelated donor transplantation.
- Patient is pregnant or lactating.
- Inability to provide adequate transfusion support or increased risk immunohematological complications due presence of anti-RBC antibody against stem cell donor.
- Patients with any history of radiation therapy.
The study team makes the final eligibility decision.
Where it's taking place
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New York, New York, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.