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Alkylating agents
Stem Cell Transplant Conditioning for SCID (CSIDE Trial)
Saint Louis, MO
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
Must not have
Presence of any serious life-threatening or opportunistic infection at time of enrollment and prior to the initiation of the preparative regimen. Serious infections as defined below that occur after enrollment must be reported immediately to the Study Coordinating Center, and enrollment will be put on hold until the infection resolves. Ideally enrolled subjects will not have had any infection. If patients have experienced infections, these must have resolved by the following definitions:
iii. Pneumonia, organism not identified by bronchoalveolar lavage: Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). If possible, radiographic resolution should also be demonstrated.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights
No Placebo-Only Group
Summary
This trial is studying a new way to do stem cell transplants in babies with certain types of severe combined immunodeficiency (SCID).
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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.
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.
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I have Omenn syndrome with a widespread skin rash.
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My kidney function is good, based on tests.
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I have an enlarged liver.
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My donor for a stem cell transplant is a 9/10 or 10/10 match.
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My immune system markers are lower than normal.
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My body has T cells from my mother.
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My condition involves a specific immune system gene defect.
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I have a genetic defect related to my immune system.
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My spleen is enlarged.
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I have swollen lymph nodes.
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My infant has been diagnosed with SCID, including typical, leaky, or Omenn syndrome.
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My condition involves a known genetic mutation linked to SCID.
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I am between 0 and 2 years old.
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I do not have a sibling who is a genetic match for a donation.
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Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I do not have any serious or life-threatening infections.
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My pneumonia symptoms are gone and I've finished my antibiotics.
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Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Vaccine specific antibody response
Secondary study objectives
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%
BK virus associated hemorrhagic cystitis
15%
ATG induced skin rash
15%
Chronic oral GvHD
14%
GI GvHD
12%
Pneumonitis
11%
Rash
8%
Headaches
8%
Palmar-plantar erythrodysesthesia
7%
Chronic lung GvGHD
3%
Viral Infections
3%
Dysrhythmia
3%
ABO incompatibility
2%
Bacterial Infections
2%
Fungal Infections
2%
Skin GVHD
2%
Poor graft function
2%
Allergic reaction to ATG
2%
Cardiomyopathy
2%
Ascites
2%
Hemorrhagic cystits
2%
Diffused alveolar hemorrhage
2%
Thrombocytopenia
1%
Hemochromatosis
1%
PRES
1%
Transcient secondary graft failure
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm A (Flu+Bu)
Arm B (Flu+Clo+Bu)
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
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 4
~1710
Find a Location
Closest Location:Nationwide Children's Hospital· Columbus, OH· 3 miles
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
12 Total Patients Enrolled
1 Trials studying Severe Combined Immunodeficiency
12 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.