Severe Immunodeficiency DiseasesPhase 2SIDSNCT00613561
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases
A Phase 2 study testing Fludarabine, Busulfan, and Anti-Thymocyte Globulin in people with Severe Immunodeficiency Diseases, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
The hypothesis of this study is that children with severe primary immunodeficiencies will benefit from early stem cell transplantation utilizing a reduced intensity conditioning regimen. This regimen is associated with a low risk of complications and will lead to correction of the underlying immunological defects. Read more about this study
- Who is it for?
- Ages Up to 11 years
- Conditions the study is for
- Severe Immunodeficiency Diseases
- What is being tested?
- Fludarabine, Busulfan, and Anti-Thymocyte Globulin
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2007to Dec 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Must show one of the following diseases:
- SCIDS
- Hyper-IgM
- Wiskott-Aldrich Syndrome
- Chediak-Higashi and Griscelli Syndromes
- X-Linked Lymphoproliferative Diseases
Show all 16Show fewer
- IPEX Syndrome
- NEMO Syndrome
- other severe immunodeficiency diseases not stated above at the discretion of the Principal Investigator
- Informed Consent
- Adequate Renal Function
- Adequate Liver Function
- Adequate Cardiac Function
- Adequate Pulmonary Function
- Adequate Performance Statue
- Adequate Venous Access
Who cannot join
- Patient/Family has not signed informed consent
- Patient does not have a clear diagnosis of a severe immunodeficiency disease
- A suitable donor for the patient cannot be found
- Patient is HIV positive
- Patient has active Hepatitis B
- Patient is pregnant
- Patient is considered unsuitable for transplant at the discretion of the Principal Investigators or the medical director
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Children's Memorial Hospital
Chicago, Illinois, 60614
About this study
The hypothesis of this study is that children with severe primary immunodeficiencies will benefit from early stem cell transplantation utilizing a reduced intensity conditioning regimen. This regimen is associated with a low risk of complications and will lead to correction of the underlying immunological defects.
- Conditions
- Severe Immunodeficiency Diseases
- Being tested
- Fludarabine, Busulfan, and Anti-Thymocyte Globulin
- Keywords
- Patients with the Severe Immunodeficiency diseases, Severe Immunodeficiency diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fludarabine, Busulfan, and Anti-Thymocyte GlobulinDrug
Also called Anti-Thymocyte Globulin is also refered to as ATG.
Patients on this study will receive 5 days of Fludarabine, 1 day of Busulfan Test Dose, 2 Days of Busulfan Regimen Dose, and 4 days of Anti-Thymocyte Globulin
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the efficacy of an optimized reduced intensity conditioning regimen using Fludarabine, Busulfan, and Anti-Thymocyte Globulin in preparing severe immunodeficiency patients for allogeneic hematopoietic progenitor cell transplantation
Measured: 5 years
Secondary outcomes (2)
To prospectively follow the natural course of severe immunodeficiency diseases after transplantation
Measured: 5 years
To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2008 (estimated)
- Study startDec 2007
- Primary completionDec 2012 (estimated)
- Study completionDec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Immunodeficiency DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Investigators
- Morris Kletzel, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00613561
- Sponsor's protocol ID
- SCT 0707
- Other IDs
- IRB # 2007-13271
- Acronym
- SIDS
- Status last verified
- May 2009
- First submitted
- 31 Jan 2008
- Last updated
- 27 May 2009
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases
- ClinicalTrials.gov ID
- NCT00613561
- Acronym / sponsor ID
- SIDS / SCT 0707
- Conditions
- Severe Immunodeficiency Diseases
- Keywords
- Patients with the Severe Immunodeficiency diseases, Severe Immunodeficiency diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Study start
- Dec 2007
- Primary completion
- Dec 2012 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 13 Feb 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The hypothesis of this study is that children with severe primary immunodeficiencies will benefit from early stem cell transplantation utilizing a reduced intensity conditioning regimen. This regimen is associated with a low risk of complications and will lead to correction of the underlying immunological defects.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (16 listed)
- Must show one of the following diseases:
- Informed Consent
- Adequate Renal Function
- Adequate Liver Function
- Adequate Cardiac Function
Key exclusion criteria (7 listed)
- Patient/Family has not signed informed consent
- Patient does not have a clear diagnosis of a severe immunodeficiency disease
- A suitable donor for the patient cannot be found
- Patient is HIV positive
- Patient has active Hepatitis B
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fludarabine, Busulfan, and Anti-Thymocyte GlobulinAlso: Anti-Thymocyte Globulin is also refered to as ATG.Patients on this study will receive 5 days of Fludarabine, 1 day of Busulfan Test Dose, 2 Days of Busulfan Regimen Dose, and 4 days of Anti-Thymocyte Globulin | Drug | – |
Primary outcome
To evaluate the efficacy of an optimized reduced intensity conditioning regimen using Fludarabine, Busulfan, and Anti-Thymocyte Globulin in preparing severe immunodeficiency patients for allogeneic hematopoietic progenitor cell transplantation
Time frame: 5 years
2 secondary outcomes
To prospectively follow the natural course of severe immunodeficiency diseases after transplantation
Time frame: 5 years
To measure the outcomes after stem cell transplantation using the reduced intensity transplant regimen.
Time frame: 5 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2009.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Morris Kletzel, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fludarabine, Busulfan, and Anti-Thymocyte Globulin in people with Severe Immunodeficiency Diseases, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 27 May 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Morris Kletzel, MD (773-880-4562, mkletzel@childrensmemorial.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the efficacy of an optimized reduced intensity conditioning regimen using Fludarabine, Busulfan, and Anti-Thymocyte Globulin in preparing severe immunodeficiency patients for allogeneic hematopoietic progenitor cell transplantation (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 May 2009.