What is 131I-apamistamab studying for sickle cell disease?

131I-apamistamab is being studied as part of a new conditioning treatment before stem cell transplant for advanced sickle cell disease. Researchers want to find the smallest effective dose to prepare patients safely and possibly reduce side effects compared to standard conditioning methods.

Who may be eligible for this sickle cell disease clinical trial?

People ages 12 to 50 with sickle cell anemia and an available matching sibling donor may be eligible. They should have specific disease complications like stroke, frequent pain crises, or require regular transfusions. Please review eligibility criteria for more details.

Is there any cost to participate in this sickle cell disease clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. Personal travel expenses may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 1

Age: 12Years - 50Years

All Genders

ID07015684

Phase 1 Study to Find the Minimum Effective Dose of 131I-apamistamab for Stem Cell Transplant Conditioning in Severe Sickle Cell Disease Patients Aged 12 to 50

Led by Columbia University · Updated on 2026-06-10

Participants Needed

Research Sites

104 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are investigating the minimum effective dose of 131I-apamistamab as part of a conditioning regimen for patients with advanced sickle cell disease SCD undergoing hematopoietic stem cell transplantation. This Phase 1, single-center study focuses on patients aged 12 to 50 years with sickle cell anemia and an available HLA-matched sibling donor. The study aims to replace the typical conditioning regimen, which includes chemotherapy, total body irradiation, and Campath antibody, with 131I-apamistamab to potentially reduce long-term side effects such as secondary cancers, infertility, cataracts, and lung toxicity. Participants will receive 131I-apamistamab as an intravenous infusion approximately ten days before receiving donor stem cell infusion. The dose of 131I-apamistamab will be personalized and prepared in advance, with target dose levels of either 100 mCi or 150 mCi. Additional treatments may include oral sirolimus to suppress immune response and intravenous Campath antibody. Other procedures such as total body irradiation and red blood cell exchange transfusions may also be part of the overall treatment process, following institutional practices. During the study, participants will undergo various assessments including blood tests, hormone level measurements, and imaging scans to monitor drug absorption and treatment effects. Researchers will evaluate outcomes such as graft failure rate 42 days after transplant, event-free survival, engraftment times for neutrophils and platelets, incidence of graft-versus-host disease, transplant-related toxicities and infections, donor cell presence, and immune recovery. Hormonal evaluations related to fertility will be measured at study start and one year post-transplant. The study includes long-term follow-up of up to seven years to assess these outcomes and safety.

CONDITIONS

Brief Title

131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)

Who Can Participate

Age: 12Years - 50Years

All Genders

Eligibility Criteria

You may qualify if you...

Availability of an HLA-matched sibling donor
Diagnosis of sickle cell anemia (Hb SS, Sβ0 thalassemia, or severe SC) aged 12 to 50 years
At least one of the following: clinically significant stroke or neurological deficit lasting 24 hours or more; 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 transfusion therapy (8+ transfusions per year for 1+ year); echocardiographic tricuspid valve regurgitant jet velocity ≥2.7 m/sec or pulmonary hypertension; sickle hepatopathy with specific lab criteria
Adequate organ function including ECOG performance status 2 or better; cardiac ejection fraction ≥40%; oxygen saturation ≥85% and corrected DLCO ≥40%; liver function within limits; no liver cirrhosis or active hepatitis confirmed by biopsy when indicated

You will not qualify if you...

Pulmonary dysfunction with DLCO <40% or oxygen saturation <85% or PaO2 <70
Severe cardiac dysfunction with ejection fraction <35%
Impaired kidney function with GFR <40
Liver fibrosis or cirrhosis or liver enzymes >5x upper limit
Stroke within 6 months prior to transplant
Karnofsky performance score <50%
HIV infection
Uncontrolled infections
Circulating human anti-mouse antibodies (HAMA) at screening
Prior radiation exceeding tolerance to critical organs
Serious chronic toxicity impairing transplant tolerance
Inability to understand transplant risks
History of severe non-compliance
Pregnancy or breastfeeding
Inability to provide transfusion support or presence of anti-RBC antibodies against donor
Any history of radiation therapy

Research Team

Central Nurse Navigator, RN

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here