Protocol Details

A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number	14-I-0072
Sponsoring Institute	National Institute of Allergy and Infectious Diseases (NIAID)
Recruitment Detail	Type: Completed Study; data analyses ongoing Gender: Male & Female Min Age: 2 Years Max Age: N/A
Referral Letter Required	Yes
Population Exclusion(s)	None
Keywords	Leaky SCID; Gamma Chain; Transplant; Gene Therapy; JAK 3; Natural History
Recruitment Keyword(s)	None
Condition(s)	Severe Combined Immune Deficiency (SCID)
Investigational Drug(s)	None
Investigational Device(s)	None
Intervention(s)	None
Supporting Site	National Institute of Allergy and Infectious Diseases

This protocol is prospective natural history study to study the natural history of subjects with severe combined immunodeficiency (SCID) disorders at the NIH CC.

This protocol is a prospective natural history study in which investigators at 43 centers caring for patients with SCID Disorder in North America will participate. Investigators will: 1) evaluate and treat patients with SCID Spectrum Disorder according to their institutional practice and protocols, and also 2) enroll the same patients as subjects in this prospective natural history protocol. Thus, subjects will receive comparable baseline and follow up evaluations across all transplant centers, irrespective of the transplant (or other treatment) strategy and methods used at an individual site.

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Eligibility

INCLUSION CRITERIA:

Subjects must be over the age of 2 and actively enrolled and receiving treatment under a SCID protocol at NIAID.

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Citations:

Railey MD, Lokhnygina Y, Buckley RH. Long-term clinical outcome of patients with severe combined immunodeficiency who received related donor bone marrow transplants without pretransplant chemotherapy or post-transplant GVHD prophylaxis. J Pediatr. 2009 Dec;155(6):834-840.e1. doi: 10.1016/j.jpeds.2009.07.049. Epub 2009 Oct 9.

Neven B, Leroy S, Decaluwe H, Le Deist F, Picard C, Moshous D, Mahlaoui N, Debr(SqrRoot)(Copyright) M, Casanova JL, Dal Cortivo L, Madec Y, Hacein-Bey-Abina S, de Saint Basile G, de Villartay JP, Blanche S, Cavazzana-Calvo M, Fischer A. Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency. Blood. 2009 Apr 23;113(17):4114-24. doi: 10.1182/blood-2008-09-177923. Epub 2009 Jan 23.

Chinen J, Davis J, De Ravin SS, Hay BN, Hsu AP, Linton GF, Naumann N, Nomicos EY, Silvin C, Ulrick J, Whiting-Theobald NL, Malech HL, Puck JM. Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency. Blood. 2007 Jul 1;110(1):67-73. Epub 2007 Mar 16.

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Contacts:

Principal Investigator	Referral Contact	For more information:
Harry L. Malech, M.D. National Institute of Allergy and Infectious Diseases (NIAID) NIHBC 10 - CRC BG RM 5-3750 10 CENTER DR BETHESDA MD 20892 (301) 480-6916 hmalech@nih.gov	Patricia L. Littel, R.N. National Institute of Allergy and Infectious Diseases (NIAID) National Institutes of Health Building 10 Room 1C129A 10 Center Drive Bethesda, Maryland 20892 (301) 335-1744 plittel@mail.nih.gov	Office of Patient Recruitment National Institutes of Health Clinical Center (CC) Building 61, 10 Cloister Court Bethesda, Maryland 20892 Toll Free: 1-800-411-1222 Local Phone: 301-451-4383 TTY: TTY Users Dial 7-1-1 ccopr@nih.gov

Clinical Trials Number:

NCT01186913

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