20 Participants Needed

Stem Cell Transplant Prep Regimen for Immune Deficiency Disorders

Recruiting at 1 trial location
Age: < 65
Sex: Any
Trial Phase: Phase 2
Sponsor: Washington University School of Medicine
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Do I need to stop 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.

What data supports the effectiveness of the drug Alemtuzumab in the stem cell transplant prep regimen for immune deficiency disorders?

Alemtuzumab is effective in reducing the risk of graft-versus-host disease (GVHD) in pediatric stem cell transplants, as shown by a study where it was part of the pretransplant conditioning regimen, resulting in no chronic GVHD and a high survival rate. Additionally, Alemtuzumab has been used successfully in treating chronic lymphocytic leukemia, indicating its potential effectiveness in targeting immune cells.12345

Is there safety data for the use of thiotepa in stem cell transplant preparation?

Thiotepa, used in high doses for stem cell transplants, can cause skin reactions like redness, blistering, and changes in skin color, affecting nearly 80% of patients in a study of children.678910

What makes this stem cell transplant prep regimen unique for immune deficiency disorders?

This treatment regimen is unique because it uses a combination of drugs, including alemtuzumab, which helps reduce the risk of graft-versus-host disease (GVHD) by depleting T-cells (a type of immune cell), potentially leading to fewer complications compared to traditional regimens. Additionally, the use of reduced-intensity conditioning with drugs like fludarabine and melphalan aims to minimize toxicity while maintaining effectiveness.311121314

What is the purpose of this trial?

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

Eligibility Criteria

This trial is for people under 28 with certain immune disorders like DiGeorge Syndrome and Wiskott-Aldrich Syndrome. They must have normal organ function, a performance status of at least 40%, and a matched donor for cell transplant. People with HIV, uncontrolled infections, or who are pregnant/breastfeeding can't join.

Inclusion Criteria

Your blood creatinine levels are within a certain range.
Your liver enzymes are not more than 5 times the normal upper limit.
Your heart is pumping normally, with a strong enough force.
See 5 more

Exclusion Criteria

Pregnant or breastfeeding
I have not had an uncontrolled viral infection in the week before starting alemtuzumab.
I haven't had severe fungal or bacterial infections in the last month.
See 1 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Preparative Regimen

Participants undergo a reduced intensity immunosuppressive preparative regimen to establish engraftment of donor hematopoietic cells

4-6 weeks

Hematopoietic Stem Cell Transplantation

Participants receive hematopoietic stem cell transplantation to optimize donor cell engraftment

1 week

Follow-up

Participants are monitored for safety and effectiveness after transplantation

12 months

Treatment Details

Interventions

  • Alemtuzumab
  • Fludarabine
  • Melphalan
  • Thiotepa
Trial Overview The study tests whether a mix of drugs (alemtuzumab, fludarabine, thiotepa, melphalan) before a cell transplant can help the body accept new immune cells with manageable side effects in patients with immune deficiencies.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: PreparativeExperimental Treatment1 Intervention

Alemtuzumab is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Campath for:
  • Chronic lymphocytic leukemia
  • Multiple sclerosis
🇪🇺
Approved in European Union as Lemtrada for:
  • Multiple sclerosis
🇪🇺
Approved in European Union as Campath for:
  • Chronic lymphocytic leukemia

Find a Clinic Near You

Who Is Running the Clinical Trial?

Washington University School of Medicine

Lead Sponsor

Trials
2,027
Recruited
2,353,000+

Findings from Research

CAMPATH-1H (alemtuzumab) is effective in depleting lymphocytes and is licensed for treating chronic lymphocyte leukemia, showcasing its efficacy in managing blood cancers.
Short-term use of alemtuzumab has shown long-term benefits in various autoimmune conditions, suggesting it may help in reducing the need for ongoing immunosuppressive drugs in transplantation and autoimmune diseases.
CAMPATH: from concept to clinic.Waldmann, H., Hale, G.[2018]
Alemtuzumab (Campath 1H), a monoclonal antibody targeting CD52 on B and T cells, is increasingly used as a conditioning agent for bone marrow transplantation, but it can have serious side effects.
In a case study of a 37-year-old woman, acute renal failure and disseminated intravascular coagulation (DIC) occurred after receiving Campath, leading to the abortion of her transplant and ongoing dialysis, highlighting the need for caution and further investigation into its safety profile.
Acute renal failure and disseminated intravascular coagulation following an idiosyncratic reaction to Alemtuzumab (Campath 1H) or fludarabine.Osborne, WL., Lennard, AL.[2017]
In a study of 17 pediatric patients undergoing matched unrelated hematopoietic stem cell transplantation (HSCT) with Campath-1H, the incidence of grade I-II acute graft versus host disease (GVHD) was only 29.4%, and no cases of chronic GVHD were observed, suggesting effective reduction of GVHD risk.
Despite a 35.3% relapse rate of primary disease, the overall survival rates were high at 100% at 100 days and 94% at one year, indicating that Campath-1H does not significantly increase the risk of life-threatening infections or relapse compared to conventional regimens.
Pretransplant conditioning with Campath-1H (alemtuzumab) in pediatric matched unrelated hematopoietic stem cell transplants: an institutional experience.Nageswara Rao, AA., Kumar, R., Altaf, S., et al.[2017]

References

CAMPATH: from concept to clinic. [2018]
Acute renal failure and disseminated intravascular coagulation following an idiosyncratic reaction to Alemtuzumab (Campath 1H) or fludarabine. [2017]
Pretransplant conditioning with Campath-1H (alemtuzumab) in pediatric matched unrelated hematopoietic stem cell transplants: an institutional experience. [2017]
Population Pharmacokinetics of Alemtuzumab (Campath) in Pediatric Hematopoietic Cell Transplantation: Towards Individualized Dosing to Improve Outcome. [2023]
Alemtuzumab in previously treated chronic lymphocytic leukemia patients who also had received fludarabine. [2017]
Cutaneous effects of thiotepa in pediatric patients receiving high-dose chemotherapy with autologous stem cell transplantation. [2013]
Drug eruption caused by azathioprine: value of using the drug-induced lymphocytes stimulation test for diagnosis. [2019]
Azathioprine-induced severe myelosuppression accompanied by massive hair loss and painful oral ulcer in an autoimmune hepatitis patient with NUDT15 minor variant: A case report. [2021]
Bone marrow inhibition induced by azathioprine in a patient without mutation in the thiopurine S-methyltransferase pathogenic site: A case report. [2021]
10.United Statespubmed.ncbi.nlm.nih.gov
Off-label use of azathioprine in dermatology: a systematic review. [2022]
A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. [2005]
12.United Statespubmed.ncbi.nlm.nih.gov
A study of a reduced-intensity conditioning regimen followed by allogeneic stem cell transplantation for patients with hematologic malignancies using Campath-1H as part of a graft-versus-host disease strategy. [2017]
13.United Statespubmed.ncbi.nlm.nih.gov
A novel GVHD-prophylaxis with low-dose alemtuzumab in allogeneic sibling or unrelated donor hematopoetic cell transplantation: the feasibility of deescalation. [2017]
14.United Statespubmed.ncbi.nlm.nih.gov
Alemtuzumab with fludarabine and cyclophosphamide reduces chronic graft-versus-host disease after allogeneic stem cell transplantation for acquired aplastic anemia. [2021]
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