Clinical trial 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?
This is an investigational oral medicine being studied as part of a treatment plan for Primary Immunodeficiency. It is designed to lower the number of certain blood cells in the body.
Also referred to as hydroxycarbamide, Hydrea, Droxia.
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
- This study is testing whether a reduced‑intensity transplant plan works for people with non‑cancer blood disorders. It looks at how well the transplant keeps working and what side effects may appear later.
- Who it's for
- You may be able to join if you are a child or teen with a non‑cancer blood disorder that can be treated with a stem cell transplant.
- What's involved
- You would receive a stem cell transplant from a cord blood or blood donor. After the transplant, you will have regular check‑ups to see how your body is doing and to watch for any late side effects.
- Time commitment
- The study lasts about 2 years, with visits at the start, after the transplant, and at regular intervals for follow‑up.
- 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
- 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...
- 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...
- 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...
See the full eligibility criteria
- 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...
- 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.
- Written informed consent and/or assent according to FDA guidelines.
- Negative pregnancy test if pubertal and/or menstruating.
- HIV negative.
- 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
- Dyskeratosis Congenita (DC)
- Congenital Amegakaryocytic Thrombocytopenia (CAMT)
- Osteopetrosis
- 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
- 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
- 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 is enrolling
- 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, but 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?
This study is enrolling now at Pittsburgh, Pennsylvania, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.