NCT01962415 Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
| NCT ID | NCT01962415 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Paul Szabolcs |
| Condition | Primary Immunodeficiency (PID) |
| Study Type | INTERVENTIONAL |
| Enrollment | 100 participants |
| Start Date | 2014-02-04 |
| Primary Completion | 2026-11 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 100 participants in total. It began in 2014-02-04 with a primary completion date of 2026-11.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion: 1. 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 least 2 x 10e7 cells/kg OR an 8 of 8 or 7 of 8 HLA allele level matched unrelated donor bone marrow or peripheral blood progenitor graft. 2. Adequate organ function as measured by: 1. Creatinine ≤ 2.0 mg/dL and creatinine clearance ≥ 50 mL/min/1.73 m2. 2. Hepatic transaminases (ALT/AST) ≤ 4 x upper limit of normal (ULN). 3. Adequate cardiac function by echocardiogram or radionuclide scan (shortening fraction \> 26% or ejection fraction \> 40% or \> 80% of normal value for age). 4. 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 (corrected for hemoglobin) should be ≥ 50% of predicted if the DLCO can be obtained. 3. Written informed consent and/or assent according to FDA guidelines. 4. Negative pregnancy test if pubertal and/or menstruating. 5. HIV negative. 6. A non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to: 1. 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 2. Congenital bone marrow failure syndromes including but not limited to: * Dyskeratosis Congenita (DC) * Congenital Amegakaryocytic Thrombocytopenia (CAMT) * Osteopetrosis 3. 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. 4. 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 5. Inflammatory Conditions * Crohn's Disease/Inflammatory Bowel Disease Exclusion: 1. Allogeneic hematopoietic stem cell transplant within the previous 6 months. 2. Any active malignancy or MDS. 3. Severe acquired aplastic anemia. 4. Uncontrolled bacterial, viral or fungal infection (currently taking medication and with progression of clinical symptoms). 5. Pregnancy or nursing mother. 6. Poorly controlled pulmonary hypertension. 7. Any condition that precludes serial follow-up.
Contact & Investigator
Paul Szabolcs, MD
PRINCIPAL INVESTIGATOR
University of Pittsburgh
Frequently Asked Questions
Who can join the NCT01962415 clinical trial?
This trial is open to participants of all sexes, aged 2 Months or older, up to 55 Years, studying Primary Immunodeficiency (PID). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT01962415 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT01962415 currently recruiting?
Yes, NCT01962415 is actively recruiting participants. Contact the research team at paul.szabolcs@chp.edu for enrollment information.
Where is the NCT01962415 trial being conducted?
This trial is being conducted at Pittsburgh, United States.
Who is sponsoring the NCT01962415 clinical trial?
NCT01962415 is sponsored by Paul Szabolcs. The principal investigator is Paul Szabolcs, MD at University of Pittsburgh. The trial plans to enroll 100 participants.