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Alkylating agents

Stem Cell Transplant for SCID

Phase 1 & 2
Waitlist Available
Led By Elizabeth M Kang, M.D.
Research Sponsored by National Institute of Allergy and Infectious Diseases (NIAID)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Auto- or allo-immunity: Objective physical findings including but are not limited to alopecia, severe rashes, uveitis, joint pain with redness or swelling or limitation of movement that is not a result of infection, lupus-like lesions, and granulomas.
Chronic pulmonary disease as defined by bronchiectasis by x-ray computerized tomography, pulmonary function test (PFT) evidence for restrictive or obstructive disease that is less than or equal to 60% of predicted for age, or pulse oximetry less than or equal to 94% in room air (if patient is too young to comply with performance of PFTs).
Timeline
Screening 3 weeks
Treatment Varies
Follow Up one year post transplant
Awards & highlights

Study Summary

This trial is for people with SCID who are getting a stem cell transplant. It will test whether or not low doses of drugs with or without radiation before transplant work just as well.

Who is the study for?
This trial is for people aged 3-40 with SCID who have a stem cell donor. They must be HIV negative, not pregnant or breastfeeding, and able to stay near NIH for 3 months post-transplant. Participants should agree to use contraception and have no major organ failure or severe psychiatric conditions.Check my eligibility
What is being tested?
The study tests if low-dose drugs alone or combined with radiation are effective in preparing SCID patients for stem cell transplants. It involves hospital admission, various health assessments, chemotherapy (with some receiving radiation), followed by the transplant procedure.See study design
What are the potential side effects?
Possible side effects include reactions to medication like busulfan and sirolimus such as nausea, liver issues, mouth sores; complications from total body irradiation like fatigue and skin changes; risks associated with anti-thymocyte globulin including allergic reactions; plus general transplant-related risks.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have physical signs of auto-immunity, like severe rashes or joint pain without infection.
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I have a chronic lung condition confirmed by tests.
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My growth in height is in the lowest 3% for my age.
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I have had watery stools 3+ times a day for over 3 months not due to infection.
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I have had 3 significant infections in the last 2 years.
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I am between 3 and 40 years old.
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I have a suitable donor for my transplant.
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I have a confirmed genetic diagnosis of SCID due to gamma c or JAK3 deficiency.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~one year post transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and one year post transplant for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Engraftment rate of > 80%
Engraftment with no grade 3 GvHD
Secondary outcome measures
Donor B cell engraftment
Donor T cell engraftment

Trial Design

2Treatment groups
Active Control
Group I: Group 1Active Control5 Interventions
Patients will be treated with Total Body Irradiation (TBI)
Group II: Group 2Active Control4 Interventions
Patients will not be treated with Total Body Irradiation (TBI)

Find a Location

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,268 Previous Clinical Trials
5,481,102 Total Patients Enrolled
Elizabeth M Kang, M.D.Principal InvestigatorNational Institute of Allergy and Infectious Diseases (NIAID)
11 Previous Clinical Trials
364 Total Patients Enrolled

Media Library

Busulfan (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT04370795 — Phase 1 & 2
Severe Combined Immune Deficiency Research Study Groups: Group 1, Group 2
Severe Combined Immune Deficiency Clinical Trial 2023: Busulfan Highlights & Side Effects. Trial Name: NCT04370795 — Phase 1 & 2
Busulfan (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04370795 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the eligibility criteria for prospective participants of this research endeavor?

"This research team is searching to enroll 30 individuals between 3 and 40 years old who suffer from severe combined immunodeficiencies (SCID). Participants must also meet the following conditions: i. Three distinct or chronic active infections in the two preceding years; ii. Treatment with systemic antibiotics, hospitalization due to infection, or isolation of a bacterial/fungal/viral pathogen; iii. Pulmonary function tests (PFT) no higher than 60% of predicted for age OR pulse oximetry readings not surpassing 94%; iv. Endoscopic evidence of enteropathy OR other markers such as malabsorption, abnormal D"

Answered by AI

How many individuals are taking part in the clinical trial?

"This trial has met its recruitment quota and is no longer taking partcipants. It was originally posted on December 4th 2022, with the last edit on November 28th 2022. For those searching for other studies, 86 clinical trials are recruiting patients diagnosed with severe combined immunodeficiencies (SCID) while 352 experiments have open spots in Group 1."

Answered by AI

What conditions has Group 1 proven to be efficacious in treating?

"Group 1 is mainly employed to treat organ transplantation, but can also aid in managing certain blood-related conditions such as acute myelocytic leukemia, chronic myelogenous leukemia and essential thrombocythemia. Allogeneic hematopoietic stem cell transplants are another example of a condition that Group 1 could be used for."

Answered by AI

Are elderly individuals being considered for this research undertaking?

"This clinical trial can only enroll patients aged 3 to 40. For those under 18 or above 65, there are 231 and 282 respective trials for them to consider."

Answered by AI

Are there any opportunities for participation in this experiment currently open?

"Confirmed. As observed on clinicaltrials.gov, this trial is inactive at the moment; it was initially published in December 4th 2022 and its last update occurred a few weeks ago. Although there are no current openings for participants, other trials of similar therapeutics have opened up to 438 applicants already."

Answered by AI

Could you provide insight into other investigations that have been conducted concerning Group 1?

"As of now, there are a total of 352 open studies focusing on the Group 1 treatment plan. 61 trials have reached Phase 3 and most take place in San Antonio, Texas but they can also be found at 6300 different sites across the country."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus
2024. "Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04370795.
~20 spots leftby Sep 2026
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