| 1 |
NCT02064933 |
Recruiting |
Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990 |
|
|
Observational |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- Primary Immune Deficiency Treatment Consortium
- Rare Diseases Clinical Research Network
|
NIH / Other |
- Observational Model: Cohort
- Time Perspective: Other
|
- Longitudinal Analysis: Overall Survival From Time of HCT/Gene Therapy
- Cross-Sectional Analysis: Proportion of Participants Achieving Full T Cell Reconstitution
- Cross-Sectional Analysis: Proportion of Participants Achieving Full B Cell Reconstitution
- (and 28 more...)
|
541 |
Male |
Child, Adult, Senior |
NCT02064933 |
DAIT RDCRN PIDTC-6904 |
|
June 2014 |
August 2019 |
August 2019 |
February 17, 2014 |
October 17, 2017 |
|
- University of Alabama at Birmingham
Birmingham, Alabama, United States - Phoenix Children's Hospital
Phoenix, Arizona, United States - Children's Hospital Los Angeles
Los Angeles, California, United States - (and 41 more...)
|
| 2 |
NCT03399461 |
Recruiting |
Targeted Literature Review and Subject Interviews in Wiskott-Aldrich Syndrome (WAS) |
|
- Other: Patient Interview guide
- Other: Caregiver interview guide
- Other: Sociodemographic questionnaire
- (and 2 more...)
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- Number of subject reported perspectives on WAS
- Number of caregiver reported perspectives on WAS
- Number of subject reported preventative measures taken to avoid infection and bleeding
- (and 12 more...)
|
1 |
Male |
12 Years to 30 Years (Child, Adult) |
NCT03399461 |
208034 |
|
January 24, 2018 |
July 31, 2018 |
July 31, 2018 |
January 16, 2018 |
April 11, 2018 |
|
- GSK Investigational Site
Collegeville, Pennsylvania, United States
|
| 3 |
NCT01515462 |
Active, not recruiting |
Gene Therapy for Wiskott-Aldrich Syndrome |
|
- Genetic: Autologous CD34 positive cells transduced with WAS encoding lentiviral vector.
|
Interventional |
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Conditioning regimen-related safety
- Safety of lentivirus gene transfer into HSC
- Sustained engraftment of genetically corrected haematopoietic stem cells in peripheral blood and/or in bone marrow
- (and 12 more...)
|
8 |
Male |
Child, Adult, Senior |
NCT01515462 |
201228
2009-017346-32 |
TIGET-WAS |
April 20, 2010 |
September 11, 2018 |
September 11, 2023 |
January 24, 2012 |
January 24, 2018 |
|
- GSK Investigational Site
Milan, Italy
|
| 4 |
NCT03198195 |
Enrolling by invitation |
Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome |
|
- Procedure: cyclophosphamide
|
Observational |
|
- Capital Research Institute of Pediatrics
|
Other |
- Observational Model: Other
- Time Perspective: Prospective
|
|
5 |
All |
5 Months to 10 Years (Child) |
NCT03198195 |
CIP-2015-08 |
|
March 10, 2015 |
March 10, 2017 |
July 10, 2020 |
June 26, 2017 |
June 27, 2017 |
|
|
| 5 |
NCT00885833 |
Completed |
Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) |
|
- Drug: Fludarabine, Busulfan, Thymoglobulin
|
Interventional |
Phase 1
Phase 2 |
- The Korean Society of Pediatric Hematology Oncology
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for HSCT in WAS.
- To evaluate the incidence and severity of toxicity and treatment related mortality.
- To evaluate overall and event free survival rate.
- (and 2 more...)
|
5 |
All |
1 Year to 25 Years (Child, Adult) |
NCT00885833 |
KSPHO-S0701 |
|
February 2007 |
March 2012 |
March 2012 |
April 22, 2009 |
July 14, 2014 |
|
- Seoul National University Hospital
Seoul, Chongno-gu, Korea, Republic of
|
| 6 |
NCT01347242 |
Unknown † |
Gene Therapy for Wiskott-Aldrich Syndrome (WAS) |
|
- Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
|
Interventional |
Phase 1
Phase 2 |
- Genethon
- Great Ormond Street Hospital for Children NHS Foundation Trust
- Institute of Child Health
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Improvement in the eczema status
- Reduction in the frequency and severity of infection episodes
- Reduction in the frequency and severity of bruising and bleeding episodes
- (and 9 more...)
|
5 |
Male |
Child, Adult, Senior |
NCT01347242 |
GTG002.07 |
|
March 2011 |
December 2017 |
December 2017 |
May 4, 2011 |
April 12, 2016 |
|
- Great Ormond Street Hospital
London, United Kingdom - Royal Free Hospital
London, United Kingdom
|
| 7 |
NCT01410825 |
Active, not recruiting |
Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome |
|
- Biological: Retrovirus-mediated gene transfer
|
Interventional |
Phase 1
Phase 2 |
- David Williams
- Boston Children’s Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety of infusion of transduced cells
- Engraftment of genetically corrected T cells
|
5 |
Male |
3 Months to 35 Years (Child, Adult) |
NCT01410825 |
CHB-P00000148 |
|
July 2011 |
January 2018 |
July 2031 |
August 5, 2011 |
January 2, 2018 |
|
- Children's Hospital Boston
Boston, Massachusetts, United States
|
| 8 |
NCT00160355 |
Completed |
Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome |
|
- Procedure: Hematopoietic stem cell transplantation
- Device: Miltenyi CliniMACS selection system
- Drug: Fludarabine, Melphalan, Thiotepa
|
Interventional |
Phase 1 |
- St. Jude Children's Research Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To determine safety in regards to engraftment and toxicity within 100 days post-haploidentical T- and B-cell depleted hematopoietic stem cell transplantation for patients with Wiskott-Aldrich syndrome who received a reduced intensity conditioning
|
4 |
Male |
up to 18 Years (Child, Adult) |
NCT00160355 |
WASHAP |
|
May 2005 |
July 2008 |
February 2009 |
September 12, 2005 |
April 26, 2017 |
|
- St. Jude Children's Research Hospital
Memphis, Tennessee, United States
|
| 9 |
NCT01347346 |
Unknown † |
Gene Therapy for WAS |
|
- Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
|
Interventional |
Phase 1
Phase 2 |
- Genethon
- Hôpital Necker-Enfants Malades
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Improvement in the eczema status
- Reduction in the frequency and severity of infection episodes
- Reduction in the frequency and severity of bruising and bleeding episodes
- (and 9 more...)
|
5 |
Male |
Child, Adult, Senior |
NCT01347346 |
GTG003.08
2009-011152-22 |
|
May 2011 |
December 2017 |
December 2017 |
May 4, 2011 |
April 12, 2016 |
|
- Hôpital Necker-Enfants Malades
Paris, France
|
| 10 |
NCT02333760 |
Recruiting |
Gene Therapy for WAS Follow-up |
|
- Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector
|
Interventional |
Phase 1
Phase 2 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
|
- Incidence and type of SAEs
- Lentiviral integration sites
- Vector copy numbers
- (and 8 more...)
|
10 |
Male |
Child, Adult, Senior |
NCT02333760 |
GNT-WAS-03 |
WAS FUP |
September 2014 |
December 2019 |
December 2019 |
January 7, 2015 |
August 17, 2016 |
|
- Hopital Necker - Enfants Malades
Paris, France - UCL Institute of Child Health
London, United Kingdom
|
| 11 |
NCT00774358 |
Completed |
Interleukin-2 Treatment for Wiskott-Aldrich Syndrome |
- Wiskott-Aldrich Syndrome (WAS)
- X-linked Thrombocytopenia
|
|
Interventional |
Phase 1 |
- Soma Jyonouchi
- Texas Children's Hospital
- Children's Hospital of Philadelphia
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety and Tolerability
- Evaluate effects on cytoskeletal dynamics
- Requirement for treatment dose antibiotics
- (and 4 more...)
|
9 |
All |
24 Months and older (Child, Adult, Senior) |
NCT00774358 |
2007-6-5354
1R01FD004091-01A1 |
WAS |
October 2008 |
September 2016 |
September 2016 |
October 17, 2008 |
August 16, 2017 |
|
- Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States - Hospital of the University of Pennsylvania
Philadelphia, Pennsylvania, United States - The Texas Children's Hospital
Houston, Texas, United States
|
| 12 |
NCT00909363 |
Active, not recruiting |
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients |
- Wiskott-Aldrich Syndrome
- Thrombocytopenia
- Bleeding
|
- Drug: Promacta (eltrombopag)
- Drug: Eltrombopag/promacta
|
Interventional |
Phase 2 |
- Weill Medical College of Cornell University
- Novartis Pharmaceuticals
|
Other / Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To increase platelet counts of WAS and XLT patients to above 30,000/ul.
- To describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.
|
19 |
Male |
3 Months to 80 Years (Child, Adult, Senior) |
NCT00909363 |
0801009600 |
WAS |
June 2009 |
May 2019 |
June 2020 |
May 28, 2009 |
February 2, 2017 |
|
- Weill Cornell Medical College
New York, New York, United States
|
| 13 |
NCT03019809 |
Recruiting |
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients |
- Wiskott-Aldrich Syndrome
- Hematopoietic Stem Cell Transplantation
- Graft Failure
|
- Biological: G-CSF for Conditioning before HSCT.
- Biological: Plerixafor for Conditioning before HSCT.
|
Interventional |
Phase 2 |
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Event free survival (EFS)
- Overall survival (OS)
- Percentage of patients with full/mixed donor chimerism
- (and 6 more...)
|
30 |
All |
1 Month to 19 Years (Child, Adult) |
NCT03019809 |
WAS_PG 2016 |
|
June 2016 |
June 2018 |
|
January 13, 2017 |
January 13, 2017 |
|
- Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
Moscow, Russian Federation
|
| 14 |
NCT01953016 |
Recruiting |
Participation in a Research Registry for Immune Disorders |
- Primary Immunodeficiencies
- GATA2
- CGD (Chronic Granulomatous Disease)
- (and 2 more...)
|
|
Observational |
|
- National Human Genome Research Institute (NHGRI)
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
- We will add NHGRI patients enrolled in 00-HG-0209 into a national registry of individuals with primary immune deficiency disorders to discover basic outcome data, ethnic and racial characteristics, kinds of complications etc. of these immune def...
|
1000 |
All |
up to 99 Years (Child, Adult, Senior) |
NCT01953016 |
130199
13-HG-0199 |
|
September 25, 2013 |
May 1, 2020 |
May 1, 2020 |
September 30, 2013 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 15 |
NCT00004341 |
Unknown † |
Study of Genetic and Molecular Defects in Primary Immunodeficiency Disorders |
- X-Linked Agammaglobulinemia
- X-Linked Hyper IgM Syndrome
- Wiskott-Aldrich Syndrome
- Leukocyte Adhesion Deficiency Syndrome
|
|
Observational |
|
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- University of Washington
- Office of Rare Diseases (ORD)
|
NIH / Other |
- Primary Purpose: Screening
|
|
|
All |
Child, Adult, Senior |
NCT00004341 |
199/11900
UW-533 |
|
July 1995 |
|
|
October 19, 1999 |
June 24, 2005 |
|
- University of Washington School of Medicine
Seattle, Washington, United States
|
| 16 |
NCT01289847 |
Completed
Has Results |
A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency |
- Primary Immune Deficiency Disorders
- Common Variable Immunodeficiency
- X-linked Agammaglobulinemia
- (and 2 more...)
|
|
Interventional |
Phase 4 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Adverse Events
- Therapeutic Efficacy
|
25 |
All |
2 Years to 16 Years (Child) |
NCT01289847 |
GMX04
IND 12569 |
|
March 2011 |
April 2014 |
April 2014 |
February 4, 2011 |
December 23, 2014 |
December 23, 2014 |
- Department of Medicine, University of California
Irvine, California, United States - Children's Hospital Los Angeles
Los Angeles, California, United States - IMMUNOe International Reseach Centers
Centennial, Colorado, United States - (and 7 more...)
|
| 17 |
NCT00220766 |
Completed |
Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients |
- Immunologic Deficiency Syndrome
- Agammaglobulinemia
- Severe Combined Immunodeficiency
- (and 2 more...)
|
- Drug: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified
- Drug: Dextrose, 5% in Water
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Single (Participant)
- Primary Purpose: Treatment
|
- Infusion related adverse events
- All adverse events
|
100 |
All |
18 Years to 75 Years (Adult, Senior) |
NCT00220766 |
100348 |
|
August 2002 |
August 2002 |
March 2004 |
September 22, 2005 |
September 25, 2009 |
|
- Departments of Medicine and Microbiology
Birmingham, Alabama, United States - National Jewish Medical and Researach Center
Denver, Colorado, United States - International Center for Interdisciplinary Studies of Immunology
Washington, District of Columbia, United States - (and 10 more...)
|
| 18 |
NCT00278954 |
Completed
Has Results |
Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases. |
- Primary Immunodeficiency
- Common Variable Hypogammaglobulinemia
- X-linked Hypogammaglobulinemia
- (and 3 more...)
|
- Biological: Gammaplex (Intravenous immunoglobulin)
|
Interventional |
Phase 3 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Serious, Acute, Bacterial Infections (SABIs) Per Subject Per Year in Subjects With Primary Immunodeficiency Disease.
- The Pharmacokinetic (PK) Half-Life of Immunoglobulin G (IgG)
- The Pharmacokinetic (PK) Clearance of Immunoglobulin G (IgG)
- (and 2 more...)
|
50 |
All |
3 Years and older (Child, Adult, Senior) |
NCT00278954 |
GMX01 |
|
January 2006 |
November 2007 |
November 2007 |
January 19, 2006 |
January 24, 2013 |
January 28, 2010 |
- Allergy Associates of the Palm Beaches
North Palm Beach, Florida, United States - Family Allergy and Asthma Center PC
Atlanta, Georgia, United States - Rush University Medical Centre, University Consultants in Allergy & Immunology
Chicago, Illinois, United States - (and 4 more...)
|
| 19 |
NCT01319851 |
Terminated
Has Results |
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation |
- Thalassemia
- Sickle Cell Disease
- Glanzmann Thrombasthenia
- (and 10 more...)
|
|
Interventional |
Not Applicable |
- Emory University
- Children's Healthcare of Atlanta
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Feasibility of Alefacept Pre-conditioning, Measured by Number of Subjects With Full Donor Engraftment
- Number of Participants That Expressed Grade 2 or 3 Regimen-Related Toxicity
- Number of Participants That Expressed Successful Neutrophil Engraftment
- (and 4 more...)
|
3 |
All |
up to 21 Years (Child, Adult) |
NCT01319851 |
IRB00039680
BMT Alefacept |
|
September 2010 |
September 2013 |
September 2013 |
March 22, 2011 |
July 27, 2017 |
March 9, 2015 |
- Children's Healthcare of Atlanta
Atlanta, Georgia, United States
|
| 20 |
NCT00730314 |
Completed |
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells |
- Sickle Cell Disease
- Thalassemia
- Anemia
- (and 9 more...)
|
- Procedure: Hematopoietic stem cell transplantation
|
Interventional |
Phase 1
Phase 2 |
- Children's Hospital Los Angeles
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- toxicities
- adverse events
- engraftment
- (and 2 more...)
|
25 |
All |
up to 21 Years (Child, Adult) |
NCT00730314 |
CCI #07-00119
CHLA-#07-00119 |
|
August 2008 |
August 2015 |
August 2015 |
August 8, 2008 |
June 23, 2016 |
|
- Children Hospital Los Angeles
Los Angeles, California, United States
|
| 21 |
NCT00006054 |
Terminated |
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies |
- Immunologic Deficiency Syndromes
- Chediak-Higashi Syndrome
- Common Variable Immunodeficiency
- (and 11 more...)
|
- Drug: anti-thymocyte globulin
- Drug: busulfan
- Drug: cyclophosphamide
- (and 6 more...)
|
Interventional |
Not Applicable |
- Fairview University Medical Center
- Office of Rare Diseases (ORD)
|
Other |
- Primary Purpose: Treatment
|
|
|
All |
up to 35 Years (Child, Adult) |
NCT00006054 |
199/15104
UMN-MT-1995-26
UMN-MT-9526 |
|
March 2000 |
December 2002 |
December 2002 |
July 6, 2000 |
October 15, 2009 |
|
- Fairview University Medical Center
Minneapolis, Minnesota, United States
|
| 22 |
NCT03333486 |
Recruiting |
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer |
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Acute Leukemia in Remission
- Acute Lymphoblastic Leukemia
- (and 34 more...)
|
- Drug: Cyclophosphamide
- Drug: Fludarabine Phosphate
- Other: Laboratory Biomarker Analysis
- (and 2 more...)
|
Interventional |
Phase 2 |
- Roswell Park Cancer Institute
- National Cancer Institute (NCI)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Relapse rate
- Engraftment rate
- Incidence of acute graft versus host disease (GVHD)
- (and 4 more...)
|
58 |
All |
1 Year to 75 Years (Child, Adult, Senior) |
NCT03333486 |
I 40916
NCI-2017-01949
P30CA016056 |
|
December 7, 2017 |
September 6, 2021 |
September 6, 2022 |
November 7, 2017 |
December 11, 2017 |
|
- Roswell Park Cancer Institute
Buffalo, New York, United States
|
| 23 |
NCT01917708 |
Recruiting |
BMT Abatacept for Non-Malignant Diseases |
- Hurler Syndrome
- Fanconi Anemia
- Glanzmann Thrombasthenia
- (and 12 more...)
|
|
Interventional |
Phase 1 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
- Tolerability of abatacept
- Proportion of Participants experiencing Immunological effects
- Proportion of Participants experiencing Regimen-related toxicity
- (and 4 more...)
|
20 |
All |
up to 21 Years (Child, Adult) |
NCT01917708 |
IRB00069836 |
|
January 2014 |
September 2018 |
January 2020 |
August 7, 2013 |
March 30, 2018 |
|
- Children's Healthcare of Atlanta
Atlanta, Georgia, United States
|
| 24 |
NCT01652092 |
Recruiting |
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies |
- SCID
- Omenn's Syndrome
- Reticular Dysgenesis
- (and 11 more...)
|
- Drug: Alemtuzumab 0.3 mg
- Drug: Cyclophosphamide
- Drug: Busulfan
- (and 6 more...)
|
Interventional |
Not Applicable |
- Masonic Cancer Center, University of Minnesota
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Neutrophil Engraftment
- Incidence of Graft Failure
- Incidence of Chimerism
- (and 5 more...)
|
30 |
All |
up to 50 Years (Child, Adult) |
NCT01652092 |
2012OC055
MT2012-10C |
|
September 4, 2012 |
December 2018 |
December 2020 |
July 27, 2012 |
December 5, 2017 |
|
- Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
|
| 25 |
NCT01821781 |
Recruiting |
Immune Disorder HSCT Protocol |
- Immune Deficiency Disorders
- Severe Combined Immunodeficiency
- Chronic Granulomatous Disease
- (and 11 more...)
|
- Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
|
Interventional |
Phase 2 |
- Washington University School of Medicine
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with donor engraftment
- Major Transplant Related Toxicities
- Time to neutrophil recovery
- (and 7 more...)
|
20 |
All |
up to 21 Years (Child, Adult) |
NCT01821781 |
201301135 |
|
March 2013 |
March 2019 |
March 2021 |
April 1, 2013 |
November 21, 2017 |
|
- Washington University
Saint Louis, Missouri, United States - Methodist Heathcare
San Antonio, Texas, United States
|
| 26 |
NCT02512679 |
Terminated
Has Results |
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells |
- Stem Cell Transplantation
- Bone Marrow Transplantation
- Peripheral Blood Stem Cell Transplantation
- (and 10 more...)
|
- Drug: Cyclophosphamide Dose Level 1
- Drug: Cyclophosphamide Dose Level 2
- Drug: Cyclophosphamide Dose Level 3
- Drug: Cyclophosphamide Dose Level 4
|
Interventional |
Phase 2 |
- Children's Hospital Los Angeles
- Lucile Packard Children's Hospital
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants With Neutrophil Engraftment (=/>500 Cells/uL) and Platelet Engraftment (>20K Cell/uL) at 30 Days
- Number of Participants With Disease Recurrence at 1 Year Post-transplant
- Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant
- (and 2 more...)
|
20 |
All |
3 Months and older (Child, Adult, Senior) |
NCT02512679 |
CCI-06-00177 |
|
February 2007 |
September 2013 |
February 2014 |
July 31, 2015 |
February 27, 2017 |
June 17, 2016 |
|
| 27 |
NCT01852370 |
Enrolling by invitation |
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases |
- Severe Combined Immunodeficiency (SCID)
- Immunodeficiency With Predominant T-cell Defect, Unspecified
- Severe Chronic Neutropenia
- (and 6 more...)
|
- Biological: CD3/CD19 negative allogeneic hematopoietic stem cells
|
Interventional |
Phase 1
Phase 2 |
- Paul Szabolcs
- University of Pittsburgh
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety: Death
- Safety: Engraftment syndrome
- Safety: Engraftment failure
- (and 19 more...)
|
16 |
All |
5 Years to 45 Years (Child, Adult) |
NCT01852370 |
PRO16010311 |
BOLT+BMT |
June 20, 2013 |
December 2019 |
December 2021 |
May 13, 2013 |
February 8, 2018 |
|
- Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
|
| 28 |
NCT01529827 |
Active, not recruiting
Has Results |
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies |
- Accelerated Phase Chronic Myelogenous Leukemia
- Adult Acute Lymphoblastic Leukemia in Remission
- Adult Acute Myeloid Leukemia in Remission
- (and 75 more...)
|
- Drug: fludarabine phosphate
- Drug: melphalan
- Radiation: total-body irradiation
- (and 6 more...)
|
Interventional |
Phase 2 |
- Roswell Park Cancer Institute
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Transplant Related Mortality (TRM)
- Clinical Response
- Progression Free Survival (PFS) at One Year
- Median Time to Neutrophil Engraftment
|
94 |
All |
3 Years to 75 Years (Child, Adult, Senior) |
NCT01529827 |
I 177110
NCI-2011-03563 |
|
February 28, 2012 |
May 28, 2015 |
May 1, 2018 |
February 9, 2012 |
September 12, 2017 |
July 2, 2017 |
- Roswell Park Cancer Institute
Buffalo, New York, United States
|
| 29 |
NCT00634569 |
Completed
Has Results |
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects |
- Primary Immune Deficiency Disease
|
- Biological: Flebogamma 5% DIF
|
Interventional |
Phase 4 |
- Instituto Grifols, S.A.
- Grifols Biologicals Inc.
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Serious Bacterial Infections.
- Days of School/Usual Activities Missed Per Year
- Days of Hospitalization Per Year
- (and 5 more...)
|
24 |
All |
2 Years to 16 Years (Child) |
NCT00634569 |
IG-0705 |
|
May 2008 |
March 2011 |
May 2011 |
March 13, 2008 |
February 2, 2017 |
December 8, 2016 |
- University of South Florida
St. Petersburg, Florida, United States - Family Allergy & Asthma Center, PC
Atlanta, Georgia, United States - Rush University Medical Center
Chicago, Illinois, United States - (and 4 more...)
|
| 30 |
NCT02092558 |
Completed |
Evaluation of Immunological Disorders of T Lymphocytes and Endocrinological Disorders as Pathogen Factors in Patients With Metaplasia of Urinary Bladder |
- The Follow-up Duration Was 1-8 Years.
- The Main Reasons Behind Visiting the Hospital Were Recurrent Urinary Tract Infection,
- Urinary Urgencies, Pollakiuria, Difficulty in Initiating Micturition, Pain in Hypogastrium,
- (and 2 more...)
|
- Drug: Second-generation cephalosporin, nitrofurantoin, 1% aminoglycoside solution
- Drug: cephalosporin with chemotherapeutics
|
Observational |
|
- Children's Hospital, Dziekanów Leśny, Poland
|
Other |
- Observational Model: Case Control
- Time Perspective: Retrospective
|
- Symptoms of stratified squamous metaplasia of urinary bladder and treatment's ways
|
2 |
All |
5 Years to 17 Years (Child) |
NCT02092558 |
KB/677/09
Bioethics Commitee of Regional |
|
January 2005 |
January 2009 |
December 2013 |
March 20, 2014 |
March 20, 2014 |
|
- Children's Hospital, Marii Konopnickiej Street 65
Dziekanów Lesny, Poland
|
| 31 |
NCT00006319 |
Enrolling by invitation |
Molecular and Clinical Studies of Primary Immunodeficiency Diseases |
- Immunologic Deficiency Syndrome
|
|
Observational |
|
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
266 |
All |
Child, Adult, Senior |
NCT00006319 |
000209
00-HG-0209 |
|
September 25, 2000 |
|
|
October 2, 2000 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
| 32 |
NCT01962415 |
Recruiting |
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT |
- Primary Immunodeficiency (PID)
- Congenital Bone Marrow Failure Syndromes
- Inherited Metabolic Disorders (IMD)
- (and 2 more...)
|
- Drug: Hydroxyurea
- Drug: Alemtuzumab
- Drug: Fludarabine
- (and 2 more...)
|
Interventional |
Phase 2 |
- Paul Szabolcs
- University of Pittsburgh
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Post-transplant treatment-related mortality (TRM)
- Neurodevelopmental milestones
- Immune Reconstitution
- (and 9 more...)
|
30 |
All |
2 Months to 35 Years (Child, Adult) |
NCT01962415 |
PRO13100018 |
HSCT+RIC |
February 4, 2014 |
November 2019 |
November 2020 |
October 14, 2013 |
April 5, 2018 |
|
- Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
|
| 33 |
NCT00919503 |
Recruiting |
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders |
|
- Procedure: Allogeneic Bone Marrow Transplantation
- Biological: Anti-Thymocyte Globulin
- Drug: Cyclosporine
- (and 9 more...)
|
Interventional |
Phase 2 |
- Fred Hutchinson Cancer Research Center
- medac GmbH
- National Cancer Institute (NCI)
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / Industry / NIH |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Preliminary efficacy
- Disease response following hematopoietic cell transplantation
- Donor chimerism
- (and 6 more...)
|
120 |
All |
up to 49 Years (Child, Adult) |
NCT00919503 |
2256.00
NCI-2010-01277
P30CA015704 |
|
July 31, 2009 |
February 1, 2019 |
|
June 12, 2009 |
April 5, 2018 |
|
- Children's Hospital Colorado
Aurora, Colorado, United States - Oregon Health and Science University
Portland, Oregon, United States - Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, United States - (and 3 more...)
|
| 34 |
NCT00086476 |
Completed |
Study of Megakaryocytes From Patients With Abnormal Platelet Vesicles |
- Blood Coagulation Disorders
|
|
Observational |
|
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
3 |
All |
2 Years to 80 Years (Child, Adult, Senior) |
NCT00086476 |
040226
04-HG-0226 |
|
June 29, 2004 |
|
June 13, 2011 |
July 2, 2004 |
July 2, 2017 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
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