131I-apamistamab for Sickle Cell Disease

Overseen By Central Nurse Navigator, RN

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Columbia University

Disqualifiers: Pulmonary dysfunction, Severe cardiac dysfunction, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 2

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the use of 131I-apamistamab, an experimental drug, to prepare patients with severe sickle cell disease for a bone marrow transplant. The goal is to determine the smallest effective dose for this preparation step, potentially making transplants safer and more accessible. This trial targets individuals with severe sickle cell complications, such as frequent pain crises, strokes, or lung issues, despite other treatments. Participants must have a matched sibling donor to join the trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that 131I-apamistamab is likely to be safe for humans?

Research is currently exploring 131I-apamistamab as a potential new treatment for sickle cell disease. The FDA has not yet approved this treatment, so it remains experimental. Initial studies in other conditions, such as a type of blood cancer, have shown promising results, with patients experiencing successful outcomes after receiving the treatment.

Since this is an early-stage study for sickle cell disease, information on how well people with this condition tolerate the treatment is limited. The main goal at this stage is to determine the safest dose to use before a bone marrow transplant. Like any new treatment, side effects may occur, but detailed safety information for sickle cell patients is not yet available. By joining this trial, participants would help researchers learn more about the safety and effectiveness of this treatment. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Sickle Cell Disease?

Most treatments for sickle cell disease focus on managing symptoms and preventing complications, often through blood transfusions or medications like hydroxyurea. But 131I-apamistamab works differently, targeting the disease at its root. This treatment uses a radio-labeled monoclonal antibody to deliver targeted radiation directly to the bone marrow. By doing so, it prepares the body for a stem cell transplant, potentially offering a more effective and long-lasting solution to sickle cell disease. Researchers are excited about this approach because it could significantly improve outcomes for patients who otherwise rely on less targeted therapies.

What evidence suggests that 131I-apamistamab might be an effective treatment for severe sickle cell disease?

Research has shown that 131I-apamistamab, the treatment under study in this trial, is a promising therapy that uses targeted radiation to attack specific cells. In studies with similar treatments, such as Iomab-B, all patients successfully underwent a stem cell transplant. One study found that 131I-apamistamab outperformed standard care for older patients with acute myeloid leukemia (AML). This trial aims to use 131I-apamistamab to help patients with severe sickle cell disease (SCD) prepare for bone marrow transplants. By targeting specific cells, this approach may prove more effective and safer than traditional methods. ^{1 2 3 4 6}

Who Is on the Research Team?

Markus Y Mapara, MD

Principal Investigator

Columbia University

Are You a Good Fit for This Trial?

This trial is for patients with severe sickle cell disease who are candidates for a bone marrow transplant. The study aims to determine the minimal effective dose of a new drug, 131I-apamistamab, as part of the pre-transplant conditioning regimen.

Inclusion Criteria

My organs are functioning well.

- 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.

I have had a stroke or lasting neurological issues confirmed by an MRI.

Exclusion Criteria

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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning

Participants receive 131I-apamistamab as part of the conditioning regimen before stem cell transplant

10 days

1 visit (in-person)

Transplantation

Participants undergo hematopoietic stem cell transplantation

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after transplantation

12 months

Multiple visits (in-person and virtual)

What Are the Treatments Tested in This Trial?

Interventions

• 131I-apamistamab

Trial Overview The trial tests a novel approach using 131I-apamistamab in place of traditional conditioning treatments before stem cell transplantation. It also involves other interventions like Campath, exchange transfusion, sirolimus, and total body irradiation to prepare patients with SCD.

How Is the Trial Designed?

Group I: 131I-apamistmab-based nonmyeloablative conditioning Experimental Treatment 6 Interventions

Ten days before participants receive donor stem cell infusion, they will receive a dose of 131 I-apamistamab as an intravenous (IV) infusion into a vein in the arm. The 131 I-apamistamab product will be formulated in advance of the anticipated infusion date, for infusion on a specific date, based on target dose (100 mCi or 150 mCi, based on dose level).

Who Is Running the Clinical Trial?

Columbia University

Lead Sponsor

Trials

1,529

Recruited

2,832,000+

Actinium Pharmaceuticals

Industry Sponsor

Trials

8

Recruited

290+

Citations

1. withpower.com withpower.com/trial/phase-1-disease-3-2025-fd726

131I-apamistamab for Sickle Cell Disease 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 ...

2. trialx.com trialx.com/5-breakthrough-studies-shaping-the-future-of-sickle-cell-disease-care/

Clinical Trial Awareness: 5 Breakthrough Studies Shaping ... This early-phase study is evaluating the safety and effectiveness of 131I-apamistamab, a targeted radioactive antibody therapy, as part of the ...

3. astctjournal.org astctjournal.org/article/S1083-8791(19)31504-6/fulltext

Targeted Conditioning with Anti-CD45 Iodine ( 131 I) ... 100% (31/31) of all patients in the Iomab-B arm who received treatment with Iomab-B were able to undergo HCT and successfully engrafted.

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39298738/

Randomized Phase III SIERRA Trial of 131 I-Apamistamab ... The 131 I-apamistamab-led regimen was associated with a higher dCR rate than conventional care in older patients with RR AML.

5. cancer.columbia.edu cancer.columbia.edu/cancer-types-care/clinical-trials/find-clinical-trial/open-label-single-center-phase-1-study-estimate-minimum-effective-dose-med-131i-apamistamab-non-myeloablative-conditioning-patients-severe-sickle-cell-disease

Open-label, Single-Center, Phase 1 Study to Estimate the ... The purpose of this study is to research the minimum effective dose (MED) of 131 I- apamistamab conditioning for hematopoietic stem cell transplantation for ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07157514

Study Details | NCT07157514 | Radioimmunotherapy ... It has two parts. In Phase 2, we are testing three radiation dose levels of 131I-apamistamab combined with fludarabine and low-dose whole-body ...