254 Participants Needed

Bone Marrow Transplant for Immune Deficiency

Recruiting at 2 trial locations
DD
AH
Overseen ByAmy H Chai
Age: Any Age
Sex: Any
Trial Phase: Phase 2
Sponsor: National Cancer Institute (NCI)
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
Approved in 3 JurisdictionsThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, since chemotherapy is involved, it's possible that some medications might need to be adjusted. Please discuss this with the trial team for specific guidance.

What data supports the effectiveness of the treatment Allo BMT for immune deficiency?

Allogeneic bone marrow transplantation (Allo BMT) is considered an effective treatment for various immune deficiency disorders, as it is the only cure for many primary immune deficiency disorders and inherited bone marrow failure syndromes. Studies have shown that it is also effective for severe combined immunodeficiency (SCID), with a high survival rate in patients receiving HLA-identical transplants.12345

Is allogeneic bone marrow transplantation generally safe for humans?

Allogeneic bone marrow transplantation (BMT) can be risky, with potential complications like graft failure, graft-versus-host disease (GVHD), and infections. Long-term effects may include lung problems, cataracts, and other health issues. While it can be life-saving for certain conditions, it is associated with significant risks and is usually done in specialized centers.16789

How is the treatment Allo BMT different from other treatments for immune deficiency?

Allo BMT is unique because it involves transplanting healthy bone marrow from a donor to replace the patient's faulty immune system, offering a potential cure for many immune deficiencies. Unlike other treatments that may only manage symptoms, this approach aims to restore normal immune function by using donor stem cells.210111213

What is the purpose of this trial?

Background:Allogeneic blood or marrow transplant is when stem cells are taken from one person s blood or bone marrow and given to another person. Researchers think this may help people with immune system problems.Objective:To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with primary immunodeficiencies.Eligibility:Donors: Healthy people ages 4 or olderRecipients: People ages 4-75 with a primary immunodeficiency that may be treated with allogeneic blood or marrow transplantDesign:Participants will be screened with medical history, physical exam, and blood tests.Participants will have urine tests, EKG, and chest x-ray.Donors will have:Bone marrow harvest: With anesthesia, marrow is taken by a needle in the hipbone.ORBlood collection: They will have several drug injections over 5-7 days. Blood is taken by IV in one arm, circulates through a machine to remove stem cells, and returned by IV in the other arm.Possible vein assessment or pre-anesthesia evaluationRecipients will have:Lung test, heart tests, radiology scans, CT scans, and dental examPossible tissue biopsies or lumbar punctureBone marrow and a small piece of bone removed by needle in the hipbone.Chemotherapy 1-2 weeks before transplant dayDonor stem cell donation through a catheter put into a vein in the chest or neckSeveral-week hospital stay. They will take medications and may need blood transfusions and additional procedures.After discharge, recipients will:Remain near the clinic for about 3 months. They will have weekly visits and may require hospital readmission.Have multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years.

Research Team

DD

Dimana Dimitrova, M.D.

Principal Investigator

National Cancer Institute (NCI)

Eligibility Criteria

This trial is for people aged 4-75 with primary immunodeficiencies that could be treated by a blood or marrow transplant. Donors must be healthy and at least 4 years old. Participants need to have certain immune system issues, like severe infections or autoimmune diseases, and good organ function. Pregnant women can't join, and participants must agree to use contraception for one year post-transplant.

Inclusion Criteria

I am between the ages of 4 and 75.
I have a donor who is a close match to my tissue type.
Ability to understand and sign a written informed consent document
See 5 more

Exclusion Criteria

I have had a condition where my lymphocytes grow abnormally.
Lack of adequate central venous access potential
I haven't had any cancer except for skin cancer in the last 5 years.
See 5 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks
1 visit (in-person)

Pre-transplant Conditioning

Recipients undergo chemotherapy 1-2 weeks before transplant day

1-2 weeks
Inpatient stay

Transplantation

Donor stem cell donation and transplantation procedure

1 day
Inpatient procedure

Post-transplant Hospitalization

Several-week hospital stay for monitoring and treatment post-transplant

Several weeks
Inpatient stay

Initial Follow-up

Participants remain near the clinic for about 3 months with weekly visits

3 months
Weekly visits (in-person)

Long-term Follow-up

Multiple follow-up visits to the clinic in the first 6 months, and less frequently for at least 5 years

5 years
Regular visits (in-person)

Treatment Details

Interventions

  • Allo BMT
Trial Overview The trial tests if blood or bone marrow transplants from donors are safe and effective in treating various primary immunodeficiencies. It involves screening, pre-transplant assessments, chemotherapy before the transplant day, hospital stay for several weeks with follow-up visits up to five years.
Participant Groups
6Treatment groups
Experimental Treatment
Active Control
Group I: 6/ RIC-SHORT ArmExperimental Treatment3 Interventions
Reduced Intensity Conditioning with shortened duration and dose-reduced PTCy
Group II: 4/ RIC-MMF ArmExperimental Treatment3 Interventions
Reduced Intensity Conditioning with MMF duration de-escalation design
Group III: 3/ MAC Arm-Closed with amendment L (07/05/2019)Experimental Treatment3 Interventions
Myeloablative Conditioning Arm
Group IV: 2/ RIC Arm - Closed with Amendment L (07/05/2019)Experimental Treatment3 Interventions
Reduced Intensity Conditioning Arm
Group V: 1/ IOC Arm-Closed with amendment L (07/05/2019)Experimental Treatment3 Interventions
Immunosuppression Only Conditioning Arm
Group VI: 5/ Donor ArmActive Control1 Intervention
Donor

Allo BMT is already approved in European Union, United States, Canada for the following indications:

๐Ÿ‡ช๐Ÿ‡บ
Approved in European Union as Allogeneic Blood or Marrow Transplantation for:
  • Primary Immunodeficiencies
  • Severe Combined Immunodeficiency
  • Wiskott-Aldrich Syndrome
  • X-linked Lymphoproliferative Syndrome
๐Ÿ‡บ๐Ÿ‡ธ
Approved in United States as Allogeneic Blood or Marrow Transplantation for:
  • Primary Immunodeficiencies
  • Severe Combined Immunodeficiency
  • Wiskott-Aldrich Syndrome
  • X-linked Lymphoproliferative Syndrome
  • Chronic Granulomatous Disease
๐Ÿ‡จ๐Ÿ‡ฆ
Approved in Canada as Allogeneic Blood or Marrow Transplantation for:
  • Primary Immunodeficiencies
  • Severe Combined Immunodeficiency
  • Wiskott-Aldrich Syndrome
  • X-linked Lymphoproliferative Syndrome

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials
14,080
Recruited
41,180,000+

Findings from Research

Allogeneic bone marrow transplantation (BMT) is a critical treatment for various hematologic diseases, particularly severe aplastic anemia and certain leukemias, with the best outcomes seen in younger patients and those in remission.
While BMT can offer a chance for cure, it carries significant risks, including graft failure, graft-versus-host disease, and long-term complications, which may limit its use to specialized centers.
Allogeneic bone marrow transplantation in the treatment of hematologic diseases.Yee, GC., McGuire, TR.[2021]
In a study of 25 patients undergoing allogeneic bone marrow transplantation (alloBMT) with reduced intensity conditioning and post-transplantation cyclophosphamide, the 2-year overall survival rate was an impressive 92%, indicating high efficacy of this treatment approach.
The modified protocol resulted in low rates of graft-versus-host disease (GVHD), with only 17% experiencing grade II acute GVHD and 14% chronic GVHD, suggesting that the removal of low-dose TBI and the use of alternative donor sources can enhance safety and reduce complications.
Reduced Intensity Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Pediatric Inherited Immune Deficiencies and Bone Marrow Failure Syndromes.Klein, OR., Bapty, S., Lederman, HM., et al.[2022]
Allogeneic bone marrow transplants are an effective treatment for various hematologic diseases, including leukemia and aplastic anemia, with over 33,000 procedures performed between 1955 and 1990, particularly increasing in the late 1980s.
Recent advancements have allowed for successful transplants using unrelated or partially matched donors, although complications like graft failure and graft versus host disease remain significant challenges that can be predicted through risk factor assessment.
Current status of allogeneic bone marrow transplantation.Sobocinski, KA., Horowitz, MM., Rimm, AA., et al.[2019]

References

Allogeneic bone marrow transplantation in the treatment of hematologic diseases. [2021]
Reduced Intensity Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Pediatric Inherited Immune Deficiencies and Bone Marrow Failure Syndromes. [2022]
Current status of allogeneic bone marrow transplantation. [2019]
Influence of severe combined immunodeficiency phenotype on the outcome of HLA non-identical, T-cell-depleted bone marrow transplantation: a retrospective European survey from the European group for bone marrow transplantation and the european society for immunodeficiency. [2019]
European experience of bone-marrow transplantation for severe combined immunodeficiency. [2019]
HLA-matched sibling transplantation with G-CSF mobilized PBSCs and BM decreases GVHD in adult patients with severe aplastic anemia. [2021]
Allogeneic HSCT for autoimmune diseases: conventional conditioning regimens. [2007]
Allogeneic hematopoietic stem cell transplantation for severe autoimmune diseases. [2009]
Long-term treatment burden following allogeneic blood and marrow transplantation in NSW, Australia: a cross-sectional survey. [2022]
10.United Statespubmed.ncbi.nlm.nih.gov
T-cell receptor αβ+ and CD19+ cell-depleted haploidentical and mismatched hematopoietic stem cell transplantation in primary immune deficiency. [2022]
11.United Statespubmed.ncbi.nlm.nih.gov
Allogeneic bone marrow transplantation. [2019]
12.United Statespubmed.ncbi.nlm.nih.gov
Bone marrow transplantation for severe combined immune deficiency. [2022]
Decision analysis of allogeneic bone marrow transplantation versus immunosuppressive therapy for young adult patients with aplastic anemia. [2023]
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