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Reduced Intensity BMT + Cyclophosphamide for Primary Immunodeficiency & Bone Marrow Failure
Study Summary
This trial will study how well a reduced intensity conditioning hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) work in patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- I have a diagnosed inherited bone marrow failure disorder.I have been diagnosed with a specific immune deficiency condition.- Your blood test shows a bad reaction to other people's white blood cells.
- You have had a transplant of someone else's stem cells.
- You have an uncontrolled infection caused by bacteria, viruses, or fungi.
- You have a specific type of anemia or genetic condition affecting your blood.
- You have tested positive for HIV.
- You have active Hepatitis B or C.
- You are pregnant or breastfeeding.
- You have an ongoing or recent history of cancer.I have been diagnosed with an immune dysregulation syndrome like IPEX or HLH.
- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- Approved for 20 Other Conditions - This treatment demonstrated efficacy for 20 other conditions.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
- Group 1: PID/IDS
- Group 2: IBMFS
Frequently Asked Questions
To what purpose is IBMFS most often put?
"The following conditions can all be treated by IBMFS: dermatitis, atopic, multiple sclerosis, and leukemia, myelocytic, acute."
Are there similar treatments to IBMFS that have been explored in other research?
"At this moment, there are 1109 clinical trials studying IBMFS with 198 of them at Phase 3. Most of the research concerning IBMFS is happening in Philadelphia, but there are a total of 30680 locations running these trials."
How does IBMFS impact human health?
"Even though there is some data supporting its safety, the lack of efficacy data knocks down IBMFS's score to a 2."
How many people are sign ups for this research project?
"That is correct. The trial, which was originally posted on February 12th 2020 according to clinicaltrials.gov, is still looking for patients. Recently, on October 20th 2022, the study updated its information seeking 27 patients from 1 site."
Who is the target demographic for this clinical trial?
"The eligibility criteria for this clinical study includes patients that have pancytopenia and are between the ages of 4 months to 40 years old. A total of 27 participants will be enrolled in the trial."
Is this research being conducted with middle-aged adults?
"The required age range for prospective participants of this clinical trial is between 4 months to 40 years old."
Are new patients able to join this clinical study?
"That is accurate. According to information found on clinicaltrials.gov, this particular trial began recruiting patients on February 12th 2020 and is still looking for 27 individuals from 1 centre."
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