Natural History Study of SCID Disorders

Conditions

• ADA SCID
• Leaky SCID
• Omenn Syndrome
• Reticular Dysgenesis
• Severe Combined Immunodeficiency (SCID)
• XSCID

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Study Details

This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important in determining outcome, study investigators will uniformly collect pre-, post- and peri-transplant (or other treatment) information on all children enrolled into this study. Children will be divided into three strata: * Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell function * Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function), and * Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy. Each Group/Cohort Stratum will be analyzed separately.

Key Dates

Start date

Sep 2, 2010

Status verified

Nov 2020

Primary completion

Sep 30, 2028

Completion

Sep 30, 2028

Study Design

Enrollment

690 participants (estimated)

Arms

• Arm: Stratum A: Typical SCID +HCT
  Stratum A: Typical Severe Combined Immunodeficiency (SCID) treated with HCT therapy. Participants with typical (formerly referred to as classic) SCID + allogeneic hematopoietic stem cell transplantation (HCT) therapy according to standard of care, per local protocol.
• Arm: Stratum B: Atypical SCID +HCT
  Stratum B: Atypical Severe Combined Immunodeficiency (SCID) treated with HCT therapy. Participants with leaky SCID, Omenn syndrome, or Reticular Dysgenesis (RS) + allogeneic hematopoietic stem cell transplantation (HCT) therapy according to standard of care, per local protocol.
• Arm: Stratum C:SCID +Non-HCT
  Stratum C: Severe Combined Immunodeficiency (SCID) who receive alternative therapy per standard of care, non-standard care and/or investigational. This stratum includes: * Adenosine Deaminase-Deficient SCID (ADA Deficient SCID) with intention to treat with Polyethylene Glycol -Adenosine Deaminase Enzyme Replacement Therapy (PEG-ADA ERT) * ADA Deficient SCID with intention to treat with gene therapy * X-linked SCID (XSCID) with intention to treat with gene therapy * Any individual with SCID previously treated with a thymus transplant (includes intention to treat with HCT, as well as PEG-ADA ERT or gene therapy)

Primary Outcome Measure

Overall Survival (OS) at Month 6 Post HCT [ Time Frame: Month 6 Post HCT ]

Locations (39)

Find similar trials in Birmingham, AL

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