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Wiskott-Aldrich SyndromeNCT02064933

Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990

An observational study of Wiskott-Aldrich Syndrome, run by National Institute of Allergy and Infectious Diseases (NIAID) at 43 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems both with the immune system and with easy bruising and bleeding. The immune abnormalities cause patients with WAS to be very susceptible to infections. Read more about this study

Who is it for?
Any age, men only
Conditions the study is for
Wiskott-Aldrich Syndrome
How many people
305 participants
Where
43 sitesin 2 countries
Study dates
2 Feb 2014to 1 May 2019
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 Any age
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: Male WAS participants treated at consortium centers Since 1990

Who may be able to join

  • WAS participants will be defined as males who have:
  • thrombocytopenia (< 100K) AND EITHER molecular diagnosis of WAS OR reduced WASP expression; OR
  • thrombocytopenia (< 100K) AND positive family history consistent with WAS diagnosis; OR
  • chronic thrombocytopenia (< 100K for minimum of 3 months) AND low mean platelet volume (MPV below normal range for age) AND EITHER recurrent and/or severe infections requiring treatment and/or eczema OR lack of antibody response to polysaccharide antigens or low IgM.
  • Longitudinal Analysis (Retrospective and Prospective)
  • Stratum A. Participants with WAS who have or will Receive HCT
Show all 10Show fewer
  • Participants with WAS who have received an HCT since January 1, 1990
  • Stratum B. Participants with WAS who have or will Receive Gene Transfer
  • Participants in which the intention is to treat with gene transfer with autologous modified cells
  • Cross-Sectional Analysis (Strata A and B) 1. Participants with WAS who are surviving and at least 2 years after the most recent HCT or gene therapy.

Who cannot join

  • As this is a natural history study, for both the Longitudinal Analysis and the Cross-Sectional Analysis we will not exclude any patients due to race or age who fit the inclusion criteria.

Trial locations and contacts

43 sites in 2 countries.

43 sites in 2 countries

United States38 sites

  • Department of Pediatrics, C. S. Mott Children's Hospital, University of Michigan

    Ann Arbor, Michigan, 48109

  • Aflac Cancer and Blood Disorders Center, Emory/Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322

  • Department of Pediatrics, University of Alabama at Birmingham

    Birmingham, Alabama, 35233

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60614

  • Cincinnati Children's Hospital Medical Center, University of Cincinnati

    Cincinnati, Ohio, 45229-3039

  • Rainbow Babies and Children's Hospital

    Cleveland, Ohio, 44106

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • Pediatrics, University of Texas Southwestern Medical Center

    Dallas, Texas, 75390-9263

  • Children's Hospital Denver, University of Colorado

    Denver, Colorado, 80220

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Institute for Pediatric Cancer and Blood Disorders, Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Baylor College of Medicine Section of Immunology, Allergy, and Retrovirology, Texas Children's Hospital

    Houston, Texas, 77030-2399

  • Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Keck School of Medicine, University of Southern California

    Los Angeles, California, 90027

  • Department of Pediatrics, David Geffen School of Medicine at University of California, Los Angeles,

    Los Angeles, California, 90095-1752

  • American Family Children's Hospital, University of Wisconsin

    Madison, Wisconsin, 53705-2275

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105

  • Children's Hospital of Wisconsin-Milwaukee

    Milwaukee, Wisconsin, 53226

  • Division of Pediatric Blood and Marrow Transplantation, University of Minnesota

    Minneapolis, Minnesota, 55455

  • Center for Cancer and Blood Disorders, Children's Hospital/Louisiana State University

    New Orleans, Louisiana, 70118

  • Department of Pediatrics, Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Lucile Salter Packard Children's Hospital at Stanford

    Palo Alto, California, 94304

  • Children's Hospital of Philadelphia, University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

  • University of Pittsburgh Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

  • Division of Pediatric Hematology/Oncology, Oregon Health and Science University

    Portland, Oregon, 97239

  • Mayo Clinic Children's Center

    Rochester, Minnesota, 55905

  • Department of Pediatrics, Golisano Children's Hospital, University of Rochester

    Rochester, New York, 14642

  • Primary Children's Hospital, University of Utah

    Salt Lake City, Utah, 84113

  • Texas Transplant Institute, Methodist Children's Hospital

    San Antonio, Texas, 78229

  • University of California, San Francisco Benioff Children's Hospital

    San Francisco, California, 94143-1278

  • Fred Hutchinson Cancer Research Center and University of Washington-Seattle Children's Hospital

    Seattle, Washington, 98109

  • Cardinal Glennon Children's Hospital, Saint Louis University

    St Louis, Missouri, 63104

  • Saint Louis Children's Hospital, Washington University

    St Louis, Missouri, 63110

  • Blood and Marrow Transplant Program, Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701

  • Maria Fareri Children's Hospital, New York Medical College

    Valhalla, New York, 10595

  • Children's National Hospital-George Washington University School of Medicine and Health Sciences

    Washington D.C., District of Columbia, 20010-2970

  • Nemours Alfred I. duPont Hospital for Children

    Wilmington, Delaware, 19803

Other trials in United States

Canada5 sites

  • Alberta Children's Hospital

    Calgary, Alberta, T3B 6A8

  • CHU Sainte-Justine, Department of Pediatrics, University of Montreal

    Montreal, Quebec, H3T 1C5

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1XB

  • Children's & Women's Health Centre of British Columbia

    Vancouver, British Columbia, V6H 3V4

  • Cancer Care Manitoba, University of Manitoba

    Winnipeg, Manitoba, R3E 0V9

Other trials in Canada

About this study

Official title: Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)

Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems both with the immune system and with easy bruising and bleeding. The immune abnormalities cause patients with WAS to be very susceptible to infections. Depending on the specific type of primary immune deficiency diseases, there are effective treatments, including antibiotics, cellular therapy and gene therapy, but studies of large numbers of patients are needed to determine the full range of causes, natural history, or the best methods of treatment for long term success.

This multicenter study combines retrospective, prospective and cross-sectional analyses of the transplant experiences for patients with WAS who have already received HCT since 1990, or who will undergo Hematopoietic cell transplant (HCT) during the study period. The retrospective and prospective portions of the study will address the impact of a number of pre and post-transplant factors on post-transplant disease correction and ultimate benefit from HCT and the cross-sectional portion of the study will assess the benefit of HCT 2 years post-HCT in consenting surviving patients.

Keywords
Wiskott-Aldrich Syndrome, Hematopoietic Stem Cell Transplantation, Genetic Therapy
Medical subject headings
Wiskott-Aldrich Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT02064933
Study groupRoleWhat they receive
Retrospective Cohort (Longitudinal Analysis)Participants with WAS treated at consortium centers since 1990 who have received transplant (Stratum A) or gene therapy (Stratum B)
Prospective Cohort (Longitudinal Analysis)Participants treated at consortium centers since 1990 who will receive transplant (Stratum A) or gene therapy (Stratum B)
Cross-Sectional Cohort (Cross-sectional Analysis)Living participants with WAS who have received transplant (Stratum A) or gene therapy (Stratum B) at Consortium Centers 1990-Present And >= 2 Years Post-Transplant

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Longitudinal Analysis: Overall Survival From Time of HCT/Gene Therapy

    The event analyzed is death from any cause. The time to this event is the time from HCT/gene therapy to death or last follow-up.

    Measured: an expected average of 5 years

  2. Cross-Sectional Analysis: Proportion of Participants Achieving Full T Cell Reconstitution

    Full T cell reconstitution is defined by all of the following:

    • CD3 cell count within normal range for age.
    • CD4 cell count within normal range for age.
    • CD8 cell count within normal range for age
    • Donor T cell chimerism > 95%
    • Lymphocyte proliferation to PHA. Proliferative responses to PHA within the normal range (at or above the lower limit of normal).

    When possible the proliferation to PHA should be calculated as a percentage of the lower limit of normal controls for that laboratory; alternatively, the lower limit of normal controls for the day may be used

    Measured: an expected average of 5 years

  3. Cross-Sectional Analysis: Proportion of Participants Achieving Full B Cell Reconstitution

    Full B cell reconstitution is defined by all of the following:

    • Quantitative immunoglobulins (IgG, IgM, and IgA) within normal range; each immunoglobulin level will be assessed separately.
    • Serologic confirmation of post immunization tetanus titer in protective range.
    • Serologic confirmation of post immunization pneumococcal titers in protective range (protective titers for > 50% of the serotypes contained in the vaccine) following immunization

    Measured: an expected average of 5 years

  4. Cross-Sectional Analysis: Proportion of Participants Achieving Resolution of thrombocytopenia

    Resolution of thrombocytopenia defined by Platelets ≥ 150,000/microliter (transfusion independent for at least 7 consecutive days)

    Measured: an expected average of 5 years

  5. Cross-Sectional Analysis: Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL

    Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL is defined as the first day of at least 3 different days the ANC is measured as 500 / uL or greater

    Measured: an expected average of 5 years

  6. Cross-sectional Analysis: Day of Recovery of Platelet Count to 20,000 / uL

    Day of Recovery of Platelet Count to 20,000 / uL is defined as the first day of at least 3 laboratory values obtained on different days where the platelet count was measured as 20,000 / uL or greater, AND without platelet transfusions for at least 7 consecutive days immediately preceding this day.

    Measured: an expected average of 5 years

  7. Cross-sectional Analysis: Day of Recovery of Platelet Count to 50,000 / uL

    Day of Recovery of Platelet Count to 50,000 / uL is defined as the first day of at least 3 laboratory values obtained on different days where the platelet count was measured as 50,000 / uL or greater, AND without platelet transfusions for at least 7 consecutive days immediately preceding this day.

    Measured: an expected average of 5 years

Secondary outcomes (24)

  1. Longitudinal Analysis: Proportion of Participants Achieving Hematologic Reconstitution

    Hematologic Reconstitution is defined as attainment of each of the following lab test values:

    • Hemoglobin within normal range for age
    • WBC count within normal range for age
    • Absolute neutrophil count (ANC) within normal range for age
    • Platelet count ≥ 150,000/microL and without transfusion for at least 7 consecutive days

    Measured: an expected average of 5 years

  2. Longitudinal Analysis: Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL

    Day of Recovery of Absolute Neutrophil Count (ANC) to 500 / uL is defined as the first day of at least 3 different days the ANC is measured as 500 / uL or greater.

    Measured: an expected average of 5 years

  3. Longitudinal Analysis: Day of Recovery of Platelet Count to 20,000 / uL

    Day of Recovery of Platelet Count to 20,000 / uL is defined as the first day of at least 3 laboratory values obtained on different days where the platelet count was measured as 20,000 / uL or greater, AND without platelet transfusions for at least 7 consecutive days immediately preceding this day.

    Measured: an expected average of 5 years

  4. Longitudinal Analysis: Day of Recovery of Platelet Count to 50,000 / uL

    Day of Recovery of Platelet Count to 50,000 / uL is defined as the first day of at least 3 laboratory values obtained on different days where the platelet count was measured as 50,000 / uL or greater, AND without platelet transfusions for at least 7 consecutive days immediately preceding this day.

    Measured: an expected average of 5 years

  5. Longitudinal Analysis: Proportion of Participants Achieving Full T Cell Immune Reconstitution

    • Absolute CD3 T cell count within normal range for age
    • Absolute CD4 T cell count within normal range for age
    • Absolute CD8 T cell count within normal range for age
    • Proliferative responses to PHA within the normal range (at or above the lower limit of normal).
    • When possible the proliferation to PHA should be calculated as a percentage of the lower limit of normal controls for that laboratory; alternatively, the lower limit of normal controls for the day may be used.

    Measured: an expected average of 5 years

  6. Longitudinal Analysis: Proportion of Participants Achieving Full B Cell Immune Reconstitution

    • Quantitative immunoglobulins (IgG, IgM, and IgA) within normal range for age; each immunoglobulin level will be assessed separately.
    • Serologic confirmation of post immunization tetanus titer in protective range.
    • Serologic confirmation of post immunization pneumococcal titers in protective range (protective titers for > 50% of the serotypes contained in the vaccine) following immunization.
    • Patients who remain on IVIG will be considered not B cell reconstituted.
    • Normalization of isohemagglutinin titers.

    Measured: an expected average of 5 years

  7. Longitudinal Analysis: State of Lineage Specific Chimerism (HCT Stratum)

    Peripheral blood chimerism will be assessed by FISH XX/XY, STRs, or WASP expression.

    Measured: an expected average of 5 years

  8. Longitudinal Analysis: Definition of Graft Failure / Rejection

    • Less than 5% of donor cells in all lineages or in whole blood by 100 days post-HCT using standard PCR based or in situ hybridization techniques OR
    • Second transplant by 100 days post-HCT (unless > 5% CD3 and purpose is to boost immune recovery).

    Failure to achieve ≥5% donor cells in all lineages or in whole blood by 100 days post-HCT will be defined as graft failure/rejection. Patients who receive a second transplant by day 100 will be considered graft failure.

    Measured: an expected average of 5 years

  9. Longitudinal Analysis: Severe bleeding episodes

    Any severe bleeding episode requiring platelet and/or RBC transfusion(s)

    Measured: an expected average of 5 years

  10. Longitudinal Analysis: Malignancy

    New onset or relapse of lymphoid malignancy confirmed by relevant pathologic and genetic features.

    Measured: an expected average of 5 years

  11. Longitudinal Analysis: Growth

    Z score of weight and height pre-HCT/gene therapy and post-HCT/gene therapy.

    Measured: an expected average of 5 years

  12. Longitudinal Analysis: Incidence of Acute GVHD

    The occurrence of skin, gastrointestinal or liver abnormalities fulfilling the Consensus criteria of Grades IIIV or grades III-IV acute GVHD1 are considered events. Death is a competing risk, and patients alive without acute GVHD will be censored at the time of last follow-up.

    Measured: an expected average of 5 years

  13. Longitudinal Analysis: Incidence of Chronic GVHD

    Chronic GVHD will be graded as limited or extensive.2 Occurrence of symptoms in any organ system that meet the criteria of chronic GVHD will be recorded. Death is a competing risk, and patients alive without chronic GVHD will be censored at time of last follow-up.

    Measured: an expected average of 5 years

  14. Longitudinal Analysis: Autoimmunity disorders

    Incidence of documented autoimmunity disorders

    Measured: an expected average of 5 years

  15. Longitudinal Analysis: Infections / blood borne infections

    1. Clinical resolution of any pre-HCT opportunistic infections including but not limited to CMV, HSV1, adenovirus, EBV, and VZV. Approximate time to resolution (clinically well, off treatment, and/or negative culture/PCR assay) will be measured from the day of HCT.
    2. Incidence of documented severe (requiring hospitalization or resulting in death) and/or recurrent bacterial, viral or fungal infection post HCT. These will be reported by site of disease, organism, date of onset post HCT, and whether or not the infection resolved.
    3. Presence and resolution of severe warts (verruca vulgaris, flat warts) from the day of HCT. Whether the subject had complete resolution, partial resolution, persistent or recurrent warts will be recorded.
    4. New episodes of infections due to meningococcus, pneumococcus or hemophilus.
    5. Lymphoproliferative disease due to EBV.

    Measured: an expected average of 5 years

  16. Cross-sectional Analysis: Current State of Lineage Specific Chimerism (HCT Stratum)

    Peripheral blood donor chimerism will be assessed by FISH XX/XY, STR, and/or WASP expression

    Measured: an expected average of 5 years

  17. Cross-sectional Analysis: Current frequency and severity of infections

    Measured: an expected average of 5 years

  18. Cross-sectional Analysis: Current Status of Growth

    Current Z score of weight and height pre-HCT/gene therapy and post-HCT/gene therapy.

    Measured: an expected average of 5 years

  19. Cross-Sectional Analysis: Graft-versus-host Disease (GvHD)

    Presence of chronic GVHD, current assessment; graded as limited or extensive

    Measured: an expected average of 5 years

  20. Cross-Sectional Analysis: Autoimmunity Disorders

    Presence of autoimmunity disorders

    Measured: an expected average of 5 years

  21. Cross-sectional Analysis: Severe Bleeding Episodes

    Any severe bleeding episode requiring platelet and/or RBC transfusion(s) during the previous year.

    Measured: an expected average of 5 years

  22. Cross-sectional Analysis: fertility

    Whether the subject has biological offspring will be recorded.

    Measured: an expected average of 5 years

  23. Cross-sectional Analysis: malignancy

    New onset or relapse of lymphoid malignancy confirmed by relevant pathologic and genetic features.

    Measured: an expected average of 5 years

  24. Cross-sectional Analysis: Quality of Life Questionnaire

    Age appropriate testing will be performed at the cross-sectional visit inpatients surviving at least two years post-transplant

    Measured: an expected average of 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Feb 2014 (estimated)
  2. Study start2 Feb 2014
  3. Primary completion1 May 2019
  4. Study completion1 May 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Other
Enrolment
305 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02064933
Sponsor's protocol ID
DAIT RDCRN PIDTC-6904
Status last verified
Aug 2020
First submitted
13 Feb 2014
Last updated
10 Aug 2020
Results posted
No
Data monitoring committee
No

Publications

  • Griffith LM, Cowan MJ, Kohn DB, Notarangelo LD, Puck JM, Schultz KR, Buckley RH, Eapen M, Kamani NR, O'Reilly RJ, Parkman R, Roifman CM, Sullivan KE, Filipovich AH, Fleisher TA, Shearer WT. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. J Allergy Clin Immunol. 2008 Dec;122(6):1087-96. doi: 10.1016/j.jaci.2008.09.045. Epub 2008 Nov 6.

    PubMed 18992926

  • Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC; workshop participants. Primary Immune Deficiency Treatment Consortium (PIDTC) report. J Allergy Clin Immunol. 2014 Feb;133(2):335-47. doi: 10.1016/j.jaci.2013.07.052. Epub 2013 Oct 15.

    PubMed 24139498

  • Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Shearer WT, Burroughs LM, Torgerson TR, Decaluwe H, Haddad E; workshop participants. Primary Immune Deficiency Treatment Consortium (PIDTC) update. J Allergy Clin Immunol. 2016 Aug;138(2):375-85. doi: 10.1016/j.jaci.2016.01.051. Epub 2016 Apr 22.

    PubMed 27262745

  • Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, Kapoor N, Hanson IC, Filipovich AH, Jyonouchi S, Sullivan KE, Small TN, Burroughs L, Skoda-Smith S, Haight AE, Grizzle A, Pulsipher MA, Chan KW, Fuleihan RL, Haddad E, Loechelt B, Aquino VM, Gillio A, Davis J, Knutsen A, Smith AR, Moore TB, Schroeder ML, Goldman FD, Connelly JA, Porteus MH, Xiang Q, Shearer WT, Fleisher TA, Kohn DB, Puck JM, Notarangelo LD, Cowan MJ, O'Reilly RJ. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014 Jul 31;371(5):434-46. doi: 10.1056/NEJMoa1401177.

    PubMed 25075835

  • Haddad E, Allakhverdi Z, Griffith LM, Cowan MJ, Notarangelo LD. Survey on retransplantation criteria for patients with severe combined immunodeficiency. J Allergy Clin Immunol. 2014 Feb;133(2):597-9. doi: 10.1016/j.jaci.2013.10.022. Epub 2013 Dec 10. No abstract available.

    PubMed 24331379

  • Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, Puck JM, Logan BR, Griffith LM, Kohn DB, O'Reilly RJ, Fleisher TA, Pai SY, Martinez CA, Buckley RH, Cowan MJ. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience. J Allergy Clin Immunol. 2014 Apr;133(4):1092-8. doi: 10.1016/j.jaci.2013.09.044. Epub 2013 Nov 28.

    PubMed 24290292

  • Dvorak CC, Cowan MJ, Logan BR, Notarangelo LD, Griffith LM, Puck JM, Kohn DB, Shearer WT, O'Reilly RJ, Fleisher TA, Pai SY, Hanson IC, Pulsipher MA, Fuleihan R, Filipovich A, Goldman F, Kapoor N, Small T, Smith A, Chan KW, Cuvelier G, Heimall J, Knutsen A, Loechelt B, Moore T, Buckley RH. The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol. 2013 Oct;33(7):1156-64. doi: 10.1007/s10875-013-9917-y. Epub 2013 Jul 2.

    PubMed 23818196

  • Griffith LM, Cowan MJ, Notarangelo LD, Puck JM, Buckley RH, Candotti F, Conley ME, Fleisher TA, Gaspar HB, Kohn DB, Ochs HD, O'Reilly RJ, Rizzo JD, Roifman CM, Small TN, Shearer WT; Workshop Participants. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. J Allergy Clin Immunol. 2009 Dec;124(6):1152-60.e12. doi: 10.1016/j.jaci.2009.10.022.

    PubMed 20004776

  • Burroughs LM, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing JJ, Edwards S, Dvorak CC, Chaudhury S, Prockop SE, Quinones R, Goldman FD, Quigg TC, Chandrakasan S, Smith AR, Parikh S, Davila Saldana BJ, Thakar MS, Phelan R, Shenoy S, Forbes LR, Martinez C, Chellapandian D, Shereck E, Miller HK, Kapoor N, Barnum JL, Chong H, Shyr DC, Chen K, Abu-Arja R, Shah AJ, Weinacht KG, Moore TB, Joshi A, DeSantes KB, Gillio AP, Cuvelier GDE, Keller MD, Rozmus J, Torgerson T, Pulsipher MA, Haddad E, Sullivan KE, Logan BR, Kohn DB, Puck JM, Notarangelo LD, Pai SY, Rawlings DJ, Cowan MJ. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood. 2020 Jun 4;135(23):2094-2105. doi: 10.1182/blood.2019002939.

    PubMed 32268350

  • Alexander JL, Davila Saldana BJ, Brazauskas R, Dammalapati SG, Griffith LM, Shah AJ, Shimano KA, Ochs HD, Bleesing JJ, Ebens CL, Kapadia M, Bauchat A, Kapoor N, Oved JH, Eissa H, Lust H, Keller MD, Haines H, Chandrakasan S, Talano JA, Rayes A, Madden L, Shereck E, Miller HK, Satter LF, Martinez C, Rozmus J, Bednarski JJ, Yu LC, Chellapandian D, Aquino VM, Knutsen A, Chong H, Chopek A, Gillio AP, Joshi A, Rangarajan H, Moore TB, Andolina JR, DeSantes KB, Vander Lugt M, Prockop SE, Shyr DC, Sullivan KE, Parikh S, Weinacht KG, Torgerson TR, Marsh R, Dvorak CC, Chan AY, Haddad E, Heimall JR, Pulsipher MA, Leiding JW, Kohn DB, Puck JM, Notarangelo LD, Rawlings DJ, Cowan MJ, Petrovic A, Pai SY, Burroughs LM. Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood Adv. 2026 Mar 10;10(5):1783-1798. doi: 10.1182/bloodadvances.2025017662.

    PubMed 41346295

Questions about this trial

What is this clinical trial studying?

An observational study of Wiskott-Aldrich Syndrome, run by National Institute of Allergy and Infectious Diseases (NIAID) at 43 sites in 2 countries.

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 10 inclusion and 1 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: finished, as of the sponsor's last update on 10 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 43 sites in United States, Canada. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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.

What does the primary outcome measure?

Longitudinal Analysis: Overall Survival From Time of HCT/Gene Therapy (time frame: an expected average of 5 years). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02064933, which also lists 10 linked publications.

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 10 Aug 2020.

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