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New treatment option for Primary Immunodeficiency (PID)
Official title Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
ClinicalTrials.gov ID: NCT01962415
What this study is testing
What is Hydroxyurea?
Hydroxyurea is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for primary immunodeficiency (pid).
Also referred to as hydroxycarbamide, Hydrea.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 55
You may be able to join if
- Inclusion:
- A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x...
- Adequate organ function as measured by:
- Creatinine ≤ 2.0 mg/dL and creatinine clearance ≥ 50 mL/min/1.73 m2.
- Hepatic transaminases (ALT/AST) ≤ 4 x upper limit of normal (ULN).
See the full eligibility criteria
- Inclusion:
- A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x 10e7 cells/kg, or double unit grafts, each cord blood unit delivering at...
- Adequate organ function as measured by:
- Creatinine ≤ 2.0 mg/dL and creatinine clearance ≥ 50 mL/min/1.73 m2.
- Hepatic transaminases (ALT/AST) ≤ 4 x upper limit of normal (ULN).
- Adequate cardiac function by echocardiogram or radionuclide scan (shortening fraction \> 26% or ejection fraction \> 40% or \> 80% of normal value for age).
- Pulmonary evaluation testing demonstrating CVC or FEV1/FVC of ≥ 50% of predicted for age and/or resting pulse oximeter ≥ 92% on room air or clearance by the pediatric or adult pulmonologist. For adult patients DLCO...
- Written informed consent and/or assent according to FDA guidelines.
- Negative pregnancy test if pubertal and/or menstruating.
- HIV negative.
- A non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to:
- Primary Immunodeficiency syndromes including but not limited to:
- Severe Combined Immune Deficiency (SCID) with NK cell activity
- Omenn Syndrome
- Bare Lymphocyte Syndrome (BLS)
- Combined Immune Deficiency (CID) syndromes
- Combined Variable Immune Deficiency (CVID) syndrome
- Wiskott-Aldrich Syndrome
- Leukocyte adhesion deficiency
- Chronic granulomatous disease (CGD)
- X-linked Hyper IgM (XHIM) syndrome
- IPEX syndrome
- Chediak - Higashi Syndrome
- Autoimmune Lymphoproliferative Syndrome (ALPS)
- Hemophagocytic Lymphohistiocytosis (HLH) syndromes
- Lymphocyte Signaling defects
- Other primary immune defects where hematopoietic stem cell transplantation may be beneficial
- Congenital bone marrow failure syndromes including but not limited to:
- Dyskeratosis Congenita (DC)
- Congenital Amegakaryocytic Thrombocytopenia (CAMT)
- Osteopetrosis
- Inherited Metabolic Disorders (IMD) including but not limited to:
- Mucopolysaccharidoses
- Hurler syndrome (MPS I)
- Hunter syndrome (MPS II)
- Leukodystrophies
- Krabbe Disease, also known as globoid cell leukodystrophy
- Metachromatic leukodystrophy (MLD)
- X-linked adrenoleukodystrophy (ALD)
- Hereditary diffuse leukoencephalopathy with spheroids (HDLS)
- Other inherited metabolic disorders
- alpha mannosidosis
- Gaucher Disease
- Other inheritable metabolic diseases where hematopoietic stem cell transplantation may be beneficial.
- Hereditary anemias
- Thalassemia major
- Sickle cell disease (SCD) - patients with sickle disease must have one or more of the following:
- Overt or silent stroke
- Pain crises ≥ 2 episodes per year for past year
- One or more episodes of acute chest syndrome
- Osteonecrosis involving ≥ 1 joints
- Priapism
- Diamond Blackfan Anemia (DBA)
- Other congenital transfusion dependent anemias
- Inflammatory Conditions
- Crohn's Disease/Inflammatory Bowel Disease Exclusion:
- Allogeneic hematopoietic stem cell transplant within the previous 6 months.
- Any active malignancy or MDS.
- Severe acquired aplastic anemia.
- Uncontrolled bacterial, viral or fungal infection (currently taking medication and with progression of clinical symptoms).
- Pregnancy or nursing mother.
- Poorly controlled pulmonary hypertension.
- Any condition that precludes serial follow-up.
The study team makes the final eligibility decision.
Where it's taking place
- Pittsburgh, Pennsylvania, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 months to 55 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Pittsburgh, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.