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Alkylating agents
Stem Cell Transplant Conditioning for SCID (CSIDE Trial)
Phase 2
Recruiting
Research Sponsored by Michael Pulsipher, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
- Absence of maternally derived T cells
Omenn syndrome • Generalized skin rash
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights
CSIDE Trial Summary
This trial is studying a new way to do stem cell transplants in babies with certain types of severe combined immunodeficiency (SCID).
Who is the study for?
This trial is for babies aged 0-2 years with Severe Combined Immunodeficiency (SCID), including typical SCID, leaky SCID, or Omenn syndrome. They should have specific immune cell counts and gene mutations related to SCID. Babies must not have serious infections at enrollment and need a suitable unrelated or half-matched related donor for stem cell transplant.Check my eligibility
What is being tested?
The study tests whether low or medium doses of busulfan chemotherapy can help infants with SCID develop immunity after a stem cell transplant from an unrelated or half-matched donor. The transplanted cells undergo special processing to reduce the risk of graft-versus-host disease.See study design
What are the potential side effects?
Busulfan may cause side effects like nausea, vomiting, diarrhea, mouth sores, hair loss, liver problems, lung issues (difficulty breathing), seizures in high doses and increased risk of future cancers. TCRαβ+/CD19+ depletion's side effects are less known but could include infection risks.
CSIDE Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My body does not have any T cells from my mother.
Select...
I have Omenn syndrome with a widespread skin rash.
Select...
My kidney function is good, based on tests.
Select...
I have an enlarged liver.
Select...
My donor for a stem cell transplant is a 9/10 or 10/10 match.
Select...
My immune system markers are lower than normal.
Select...
My body has T cells from my mother.
Select...
My condition involves a specific immune system gene defect.
Select...
I have a genetic defect related to my immune system.
Select...
My spleen is enlarged.
Select...
I have swollen lymph nodes.
Select...
My infant has been diagnosed with SCID, including typical, leaky, or Omenn syndrome.
Select...
My condition involves a known genetic mutation linked to SCID.
Select...
I am between 0 and 2 years old.
Select...
I do not have a sibling who is a genetic match for a donation.
CSIDE Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Vaccine specific antibody response
Secondary outcome measures
Acute graft-versus-host disease (aGVHD)
Busulfan Pharmacokinetics
Chronic graft-versus-host disease (cGVHD)
+5 moreSide effects data
From 2020 Phase 3 trial • 256 Patients • NCT0147144495%
Nausea
95%
Mucositis
82%
Elevated transminitis
79%
Infections
55%
Skin GvHD
50%
ATG induced fevers
45%
Fluid overload
32%
Diarrhea
32%
Neutropenic fevers
29%
Hypertension
27%
Upper GI GvHD
22%
Renal insufficiency
19%
Liver GvHD
18%
Chronic ocular GvHD
15%
ATG induced skin rash
15%
BK virus associated hemorrhagic cystitis
15%
Chronic oral GvHD
14%
GI GvHD
12%
Pneumonitis
11%
Rash
8%
Headaches
8%
Palmar-plantar erythrodysesthesia
7%
Chronic lung GvGHD
3%
ABO incompatibility
3%
Dysrhythmia
3%
Viral Infections
2%
Cardiomyopathy
2%
Poor graft function
2%
Fungal Infections
2%
Allergic reaction to ATG
2%
Ascites
2%
Hemorrhagic cystits
2%
Diffused alveolar hemorrhage
2%
Thrombocytopenia
2%
Skin GVHD
2%
Bacterial Infections
1%
PRES
1%
Hemochromatosis
1%
Transcient secondary graft failure
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm A (Flu+Bu)
Arm B (Flu+Clo+Bu)
CSIDE Trial Design
2Treatment groups
Experimental Treatment
Group I: Medium Dose BusulfanExperimental Treatment2 Interventions
Busulfan based preparative regimen targeted at area-under-the-curve (cAUC) exposure of 55-65 mg*h/L.
Randomization between the two dose levels will be done separately in each genotype stratum (RAG1/RAG2 and IL2RG/JAK3), using permuted blocks.
Group II: Low Dose BusulfanExperimental Treatment2 Interventions
Busulfan based preparative regimen targeted at area-under-the-curve (cAUC) exposure of 25-35 mg*h/L .
Randomization between the two dose levels will be done separately in each genotype stratum (RAG1/RAG2 and IL2RG/JAK3), using permuted blocks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Busulfan
2008
Completed Phase 3
~1120
Find a Location
Who is running the clinical trial?
Michael Pulsipher, MDLead Sponsor
4 Previous Clinical Trials
180 Total Patients Enrolled
Sung-Yun Pai, MDStudy ChairNational Institutes of Health (NIH)
1 Previous Clinical Trials
10 Total Patients Enrolled
1 Trials studying Severe Combined Immunodeficiency
10 Patients Enrolled for Severe Combined Immunodeficiency
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I had a fungal infection confirmed by a test but have completed at least 14 days of treatment.My condition is diagnosed as typical SCID.My body does not have any T cells from my mother.My kidney function is good, based on tests.I don't need extra oxygen and my oxygen level is above 92% on room air.My kidney function is within the required range.My heart condition is stable.My immune cells show specific activation markers.I have an enlarged liver.I do not have any serious or life-threatening infections.My skin infection has fully healed after finishing at least 10 days of antibiotics.I will receive a stem cell transplant from a partially matched family member.My immune response to common antigens is low, indicating possible Omenn Syndrome.My immune response to candida and tetanus is very low or absent.My immune cells show specific markers indicating a certain type of activity or condition.My organs are functioning well.I have Omenn syndrome with a widespread skin rash.My donor for a stem cell transplant is a 9/10 or 10/10 match.My immune system markers are lower than normal.My body has T cells from my mother.My condition involves a specific immune system gene defect.My liver is functioning properly.I have a genetic defect related to my immune system.My symptoms have fully improved, and if possible, this is shown in my scans.My spleen is enlarged.I have swollen lymph nodes.I had a bacterial infection confirmed by a culture, but it's now cleared after completing antibiotics.My pneumonia symptoms are gone and I've finished my antibiotics.I have fully recovered from Pneumocystis, including finishing my treatment.I have been diagnosed with a RAG1/RAG2 condition for 12 weeks.My infant has been diagnosed with SCID, including typical, leaky, or Omenn syndrome.My condition involves a known genetic mutation linked to SCID.I am between 0 and 2 years old.I have not received IL2RG/JAK3 treatments in the last 8 weeks.My age fits the requirement for starting busulfan treatment.I do not have a sibling who is a genetic match for a donation.My condition is diagnosed as leaky SCID.
Research Study Groups:
This trial has the following groups:- Group 1: Low Dose Busulfan
- Group 2: Medium Dose Busulfan
Timeline:
This trial has the following timeline:- 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.
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Could I be a test subject for this research?
"Eligible patients for this research must have the diagnosis of scid and be between 0 and 2 years old. The aim is to enroll a total of 64 individuals."
Answered by AI
What is the scientific research history of Busulfan?
"There are one hundred and four clinical trials currently underway concerning the efficacy of Busulfan. Thirteen of those live trials are in their third phase. The majority of these trials are based out of Philadelphia, although there are one thousand and seventy-two locations running some form of trial for this medication."
Answered by AI
Are geriatric patients being included in this research?
"Based on the age parameters set by the researchers conducting this study, 0 is the minimum age for enrollment while 2 years old is the maximum age."
Answered by AI
How many men and women are participating in this experiment?
"Indeed, the listing on clinicaltrials.gov shows that this trial is open for enrollment. This particular study was originally posted on October 22nd, 2018 and was last updated on March 2nd, 2022. They are looking to enroll a total of 64 patients at 41 different locations."
Answered by AI
How does the toxicity profile of Busulfan compare to other treatments?
"Although there is some evidence pointing to its safety, Busulfan's lack of efficacy data leaves it with a score of 2."
Answered by AI
What disease does Busulfan usually target?
"Busulfan is commonly used to treat chronic myelogenous leukemia and can also be useful in ameliorating conditions such as thrombocythemia, essential, polycythemia vera (pv), and for conditioning regimens prior to allogeneic stem cell transplantation therapy."
Answered by AI
Are there any patients you are still looking for to complete this research project?
"That is accurate. The information available on clinicaltrials.gov suggests that this trial is still recruiting patients. This study was originally posted on October 22, 2018 and the most recent update was March 2, 2022. A total of 64 individuals are being sought for this trial across 41 locations."
Answered by AI
What is the total number of hospitals participating in this clinical trial?
"Out of the 41 sites that are enrolling patients for this trial, there may be one near you. To minimize travel difficulties, it is best to select a location close to where you live. Some notable locations include Washington, Gainesville and New Orleans."
Answered by AI
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