Actively Recruiting

Phase 1
Age: 12Years - 50Years
All Genders
NCT07015684

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

Led by Columbia University · Updated on 2025-06-11

24

Participants Needed

1

Research Sites

358 weeks

Total Duration

On this page

Sponsors

C

Columbia University

Lead Sponsor

A

Actinium Pharmaceuticals

Collaborating Sponsor

AI-Summary

What this Trial Is About

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. 131 I-apamistamab is an investigational product. This means that 131 I-apamistamab has not been approved by the Food and Drug Administration (FDA) for medical use in patients. The study treatment that is given before the transplant is called the conditioning regimen. In this study, the investigators are adding a drug called 131 I-apamistamab instead of the conditioning regimen typically given before a stem cell transplant.

CONDITIONS

Official 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

Eligible

You may qualify if you...

  • Availability of an HLA-matched sibling donor
  • Patients aged 12 to 50 years with sickle cell anemia (Hb SS, Sβ0 thalassemia, or severe SC) who have one or more of the following: clinically significant stroke or neurological deficit lasting at least 24 hours, history of two or more acute chest syndrome episodes in past 2 years despite care, history of three or more severe vaso-occlusive pain crises per year in past 2 years despite care, regular red blood cell transfusion therapy of 8 or more transfusions per year for at least 1 year, echocardiographic tricuspid valve regurgitant jet velocity ≥2.7 m/sec or pulmonary hypertension, or sickle hepatopathy with ferritin >1000 mcg/L or direct bilirubin >0.4 mg/dl but <5x upper limit of normal and platelet count <250,000/µL at baseline
  • Adequate organ function including ECOG performance status of 2 or better, cardiac ejection fraction (LVEF) ≥40%, pulmonary oxygen saturation ≥85% and corrected DLCO ≥40%, hepatic function with bilirubin and transaminases below 5x upper limit of normal, absence of liver cirrhosis, bridging fibrosis, and active hepatitis as confirmed by liver biopsy if iron overload present
Not Eligible

You will not qualify if you...

  • Pulmonary dysfunction with DLCO <40% predicted or baseline oxygen saturation <85% or PaO2 <70
  • Severe cardiac dysfunction with ejection fraction <35%
  • Impaired kidney function with GFR <40
  • Liver dysfunction with bridging fibrosis or cirrhosis or transaminases >5x upper limit of normal
  • Clinical stroke within 6 months before transplant
  • Karnofsky performance score less than 50%
  • HIV infection
  • Uncontrolled infections at enrollment
  • Presence of circulating human anti-mouse antibodies (HAMA)
  • Prior radiation to maximum tolerated levels to critical organs
  • Serious chronic toxicity that limits ability to tolerate transplant
  • Inability to understand transplant risks and process
  • History of severe non-compliance affecting transplant eligibility
  • Pregnancy or lactation
  • Inability to provide adequate transfusion support or presence of anti-red blood cell antibodies against donor
  • Any history of radiation therapy

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 1 location

1

Columbia University Irving Medical Center

New York, New York, United States, 10032

Actively Recruiting

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Research Team

C

Central Nurse Navigator, RN

CONTACT

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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