I Cell DiseaseNCT00005900
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
An observational study of I Cell Disease and Fucosidosis, run by Fairview University Medical Center at 1 site in United States.
Trial summary
OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory cytokines and for the production of nitric oxide by alveolar macrophages to identify possible… Read more about this study
- Who is it for?
- Ages 0 years and older
- Conditions the study is for
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 1999to not given
- Phase
- Observational
- 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 0 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
- Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01
Trial locations and contacts
1 site in 1 country.
United States1 site
Fairview University Medical Center
Minneapolis, Minnesota, 55455
About this study
OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory cytokines and for the production of nitric oxide by alveolar macrophages to identify possible risk factors for pulmonary complications.
II. Investigate the underlying mechanism for the development of significant pulmonary complications in these patients during HSCT.
III. Evaluate bronchoalveolar lavage fluid and serum obtained from these same patients at the time a pulmonary complication develops post-HSCT, or at 60 days post-HSCT if there has been no pulmonary complications.
Read the detailed description
PROTOCOL OUTLINE:
Patients undergo bronchoscopy with bronchoalveolar lavage (BAL) prior to allogeneic hematopoietic stem cell transplantation (HSCT). ELISA assays for cytokines are performed. Patients are followed post-HSCT for the development of transplant related pulmonary complications. A repeat bronchoscopy with BAL is performed at the time pulmonary complications develop or at day 60 post-HSCT if no complications develop. Cytokine assays are repeated.
- Conditions
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease
- Keywords
- Gaucher's disease, I cell disease, Niemann-Pick disease, Wolman disease, adrenoleukodystrophy, disease-related problem/condition, fucosidosis, genetic diseases and dysmorphic syndromes, globoid cell leukodystrophy, inborn errors of metabolism, mannosidosis, metachromatic leukodystrophy, mucopolysaccharidosis, mucopolysaccharidosis I, mucopolysaccharidosis VI, oncologic disorders, pulmonary complications, rare disease, sphingolipidoses
- Medical subject headings
- Mucolipidoses, Fucosidosis, Leukodystrophy, Globoid Cell, Adrenoleukodystrophy, Mannosidase Deficiency Diseases, Niemann-Pick Diseases, Mucopolysaccharidosis I, Mucopolysaccharidosis VI
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2000 (estimated)
- Study startAug 1999
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for I Cell DiseaseStudies 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
- Observational
- Enrolment
- 10 participants
Sponsor and investigators
- Lead sponsor
- Fairview University Medical Center
- Sponsor type
- University, hospital or other
- Investigators
- K. Scott Baker, Study chair, Fairview University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00005900
- Sponsor's protocol ID
- 199/15111
- Other IDs
- UMN-MT-1999-18, UMN-MT-9818
- Status last verified
- Oct 2003
- First submitted
- 2 Jun 2000
- Last updated
- 24 Jun 2005
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT00005900
- Acronym / sponsor ID
- 199/15111
- Conditions
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease
- Keywords
- Gaucher's disease, I cell disease, Niemann-Pick disease, Wolman disease, adrenoleukodystrophy, disease-related problem/condition, fucosidosis, genetic diseases and dysmorphic syndromes
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 10 participants
- Lead sponsor
- Fairview University Medical CenterUniversity, hospital or other
- Study start
- Aug 1999
- First posted
- 5 Jun 2000 (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.
OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory cytokines and for the production of nitric oxide by alveolar macrophages to identify possible risk factors for pulmonary complications.
II. Investigate the underlying mechanism for the development of significant pulmonary complications in these patients during HSCT.
III. Evaluate bronchoalveolar lavage fluid and serum obtained from these same patients at the time a pulmonary complication develops post-HSCT, or at 60 days post-HSCT if there has been no pulmonary complications.
Detailed description
PROTOCOL OUTLINE:
Patients undergo bronchoscopy with bronchoalveolar lavage (BAL) prior to allogeneic hematopoietic stem cell transplantation (HSCT). ELISA assays for cytokines are performed. Patients are followed post-HSCT for the development of transplant related pulmonary complications. A repeat bronchoscopy with BAL is performed at the time pulmonary complications develop or at day 60 post-HSCT if no complications develop. Cytokine assays are repeated.
Study design
Who may qualify
- Ages
- 0 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2005.
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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.
K. Scott Baker study chair
Fairview University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of I Cell Disease and Fucosidosis, run by Fairview University Medical Center 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?
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 24 Jun 2005. 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
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 24 Jun 2005.