NCT01346150

A Retrospective and Cross-Sectional Analysis of Patients Treated for SCID Since January 1,1968 (RDCRN PIDTC-6902)

Status unknown Last updated 9 November 2020

What this trial tests

trial in SCID in 1,007 participants. Status unknown.

Timeline

15 May 2011

Primary endpoint
1 August 2023

1 August 2023

Quick facts

Lead sponsor	National Institute of Allergy and Infectious Diseases (NIAID)
Status	Status unknown
Study type	OBSERVATIONAL
Enrollment	1,007
Start date	15 May 2011
Primary completion	1 August 2023
Estimated completion	1 August 2023
Sites	36 locations across United States, Canada

Conditions studied

• SCID — all drugs for SCID →
• ADA-SCID — all drugs for ADA-SCID →
• XSCID — all drugs for XSCID →
• Leaky SCID — all drugs for Leaky SCID →

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Who can join

Eligibility, any sex, with SCID or ADA-SCID. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Retrospective Study - Part 1
  Time frame: 1, 5, 10, 20, >20 years
  Overall survival
• Cross-Sectional Study - Part 2
  Time frame: 2 to > 20 years
  Full immune reconstitution

Sponsor's own description

Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant, gene therapy, or enzyme replacement in the past may be eligible for this study. The purpose of study is to look backwards at what has already been done in the. Over 800 patients with SCID are expected to be enrolled, making this one of the largest studies ever to describe outcomes for patients with SCID treated at many different hospitals around North America.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience.
   Shearer WT, Dunn E, Notarangelo LD, Dvorak CC, et al · · 2014 · cited 229× · PMID 24290292 · DOI 10.1016/j.jaci.2013.09.044
2. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery.
   Haddad E, Logan BR, Griffith LM, Buckley RH, et al · · 2018 · cited 136× · PMID 30154114 · DOI 10.1182/blood-2018-03-840702
3. Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium.
   Thakar MS, Logan BR, Puck JM, Dunn EA, et al · · 2023 · cited 76× · PMID 37352885 · DOI 10.1016/s0140-6736(23)00731-6
4. The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018).
   Dvorak CC, Haddad E, Buckley RH, Cowan MJ, et al · · 2019 · cited 62× · PMID 30193840 · DOI 10.1016/j.jaci.2018.08.027
5. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.
   Cuvelier GDE, Logan BR, Prockop SE, Buckley RH, et al · · 2022 · cited 39× · PMID 35671392 · DOI 10.1182/blood.2022016196
6. Primary Immune Deficiency Treatment Consortium (PIDTC) report.
   Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, et al · · 2014 · cited 35× · PMID 24139498 · DOI 10.1016/j.jaci.2013.07.052
7. Primary Immune Deficiency Treatment Consortium (PIDTC) update.
   Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, et al · · 2016 · cited 32× · PMID 27262745 · DOI 10.1016/j.jaci.2016.01.051
8. B-cell differentiation and IL-21 response in <i>IL2RG/JAK3</i> SCID patients after hematopoietic stem cell transplantation.
   Miggelbrink AM, Logan BR, Buckley RH, Parrott RE, et al · · 2018 · cited 30× · PMID 29728406 · DOI 10.1182/blood-2017-10-809822