Actively Recruiting
131I-apamistamab-based Conditioning for Stem Cell Transplant in Advanced Sickle Cell Disease Open-label, Single-Center Phase 1 Study to Find Minimum Effective Dose
Led by Columbia University · Updated on 2025-06-11
24
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
C
Columbia University
Lead Sponsor
A
Actinium Pharmaceuticals
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying the smallest effective dose of 131I-apamistamab to prepare patients with advanced sickle cell disease (SCD) for a bone marrow transplant. This is the first time 131I-apamistamab, an investigational drug not yet approved by the FDA, is being used as part of the conditioning regimen before an allogeneic stem cell transplant. The goal is to see if eliminating total body irradiation, which can cause long-term side effects, is possible while still allowing successful transplantation. Participants will receive 131I-apamistamab as an intravenous infusion about ten days before receiving donor stem cells. The dose will be either 100 mCi or 150 mCi based on the dose level. This drug replaces the usual conditioning treatments like chemotherapy, total body irradiation, and Campath antibody. Other treatments involved include sirolimus and Campath, given to support the transplant process. During the study, participants will undergo various assessments including blood tests to monitor graft success, immune recovery, hormone levels, and transplant-related complications up to seven years after transplant. Imaging with planar gamma camera will evaluate drug absorption. Follow-up includes checking for graft failure 42 days post-transplant and monitoring long-term safety. Total participation lasts several years to observe transplant outcomes and side effects.
CONDITIONS
Brief Title
131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Availability of an HLA-matched sibling donor
- Age between 12 and 50 years inclusive
- Diagnosis of sickle cell anemia (Hb SS, Sb20 thalassemia, or severe SC) with one or more of the following: clinically significant stroke or neurological deficit lasting at least 24 hours; two or more acute chest syndrome episodes in past 2 years despite care; three or more severe pain crises per year in past 2 years despite care; regular red blood cell transfusions (8 or more per year for at least 1 year); echocardiographic evidence of tricuspid valve regurgitant jet velocity �32.7 m/sec or pulmonary hypertension; sickle hepatopathy with ferritin >1000 mcg/L or direct bilirubin >0.4 mg/dl but <5x upper limit normal and platelet count <250,000/uL
- Adequate organ function defined by ECOG performance status 2 or better; left ventricular ejection fraction of 40% or greater; oxygen saturation of 85% or higher and corrected DLCO of 40% or greater; liver function tests within limits; absence of liver cirrhosis, bridging fibrosis, and active hepatitis confirmed by biopsy if iron overload present
You will not qualify if you...
- Pulmonary dysfunction with corrected DLCO less than 40% or oxygen saturation below 85% or PaO2 under 70
- Severe cardiac dysfunction with ejection fraction below 35%
- Impaired kidney function with GFR under 40
- Liver dysfunction including bridging fibrosis, cirrhosis, or transaminases over 5 times upper limit
- Clinical stroke within 6 months before transplant
- Karnofsky performance score under 50%
- HIV infection
- Uncontrolled viral, bacterial, fungal, or protozoal infection at enrollment
- Presence of circulating human anti-mouse antibodies (HAMA)
- Prior radiation at maximum tolerated levels to critical organs
- Serious chronic toxicity affecting transplant tolerance
- Inability to understand risks and nature of transplant
- History of severe non-compliance
- Pregnancy or lactation
- Inability to provide adequate transfusion support or risk of immunohematological complications
- Any history of radiation therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 10 days
Participants receive a nonmyeloablative conditioning regimen including a dose of 131I-apamistamab given by intravenous infusion about 10 days before stem cell transplant. This replaces the typical conditioning regimen to reduce long-term side effects.
1 infusion visit (in-person)
Duration - 1 day
Participants receive donor stem cell infusion following conditioning to treat advanced sickle cell disease.
1 infusion visit (in-person)
Duration - Up to 12 months post-transplant
Participants take immunosuppressant medication (Sirolimus and Campath) orally and via IV, with dosing adjusted over time to prevent transplant rejection and complications. Participants are closely monitored for graft success, transplant-related toxicities, and immune recovery.
Frequent visits initially, then periodic visits for up to 12 months
Duration - Up to 7 years
Participants are monitored for long-term outcomes including graft failure, survival, immune reconstitution, hormone levels, and transplant-related complications for up to 7 years after transplant.
Periodic visits over several years
Trial Site Locations
Total: 1 location
1
Columbia University Irving Medical Center
New York, New York, United States, 10032
Actively Recruiting
Research Team
C
Central Nurse Navigator, RN
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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