Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Related Clinical Trial
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy and Metachromatic Leukodystrophy SMART-ALD – A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD) Quality of Life in Women With X-linked Adrenoleukodystrophy A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) Plasma Exchange With Albumin in AMN Patients Stem Cell Transplant for Inborn Errors of Metabolism Human Placental-Derived Stem Cell Transplantation Precision Exercise in Children With Malignant Hemopathies Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy MD1003-AMN MD1003 in Adrenomyeloneuropathy A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD) A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy Clinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese Children Early Diagnosis Of Childhood Cerebral ALD Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children HSCT for High Risk Inherited Inborn Errors Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD) UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) A Phase III Trial of Lorenzo’s Oil in Adrenomyeloneuropathy Lentiviral Gene Therapy for X-ALD MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD) Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) Newborn Screening for Adrenoleukodystrophy A Study to Prospectively Assess Disease Progression in Male Children With X-ALD Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy Expanded Access for Lorenzo’s Oil (GTO/GTE) in Adrenoleukodystrophy Minnesota Adrenoleukodystrophy Registry Study (MARS) and Biobank A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Brief Title

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation


Brief 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 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 Type

Observational




Condition

I Cell Disease



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

10

Start Date

August 1999



Eligibility Criteria

        -  Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem
             cell transplantation on protocol UMN-MT-1995-01
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

K. Scott Baker, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00005900

Organization ID

199/15111

Secondary IDs

UMN-MT-1999-18


Study Sponsor

Fairview University Medical Center


Study Sponsor

K. Scott Baker, Study Chair, Fairview University Medical Center


Verification Date

October 2003