No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
2 Months – 55
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
100 (estimated)
Sponsor
Paul Szabolcs · Other
Who this trial is looking for
This trial is looking for patients with certain non-cancerous disorders who may benefit from a special type of stem cell transplant. Participants will receive treatment and be monitored for how well the transplant works and any long-term effects.
Are You a Good Fit for This Trial?
You may be able to join if
I have a non-malignant disorder that can be treated with a stem cell transplant.
I have a matching umbilical cord blood or donor bone marrow.
I have signed a consent form to participate.
I am HIV negative.
I have good organ function.
I am not pregnant or nursing.
I can attend follow-up appointments.
You may not be able to join if
I had a stem cell transplant in the last 6 months.
I have an active cancer.
I have a severe infection that is not controlled.
I have poorly controlled pulmonary hypertension.
I have severe acquired aplastic anemia.
I cannot follow up for appointments.
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer three quick questions written from this study's own rules to see if you may qualify. No account needed to find out.
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). …
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.
Trial Locations
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Status
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 ma…
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.
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