Primary Immunodeficiency (PID)Phase 2HSCT+RICNCT01962415
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
A Phase 2 study testing Hydroxyurea, Alemtuzumab and Fludarabine in people with Primary Immunodeficiency (PID) and Congenital Bone Marrow Failure Syndromes, run by Paul Szabolcs at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 months to 55 years
- Conditions the study is for
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD)
- What is being tested?
- Hydroxyurea, Alemtuzumab and Fludarabine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Feb 2014to Nov 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 months to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- 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 (corrected for hemoglobin) should be ≥ 50% of predicted if the DLCO can be obtained.
Show all 55Show fewer
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Shawna McIntyre, RN, 412-692-5552, mcintyresm@upmc.edu
Paulina Horvei, MD
About this study
Official title: A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults ≤55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation
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.
Read the detailed description
For some non-malignant diseases (NMD; i.e., thalassemia, sickle cell disease, most immune deficiencies) a hematopoietic stem cell transplant may be curative by healthy donor stem cell engraftment alone. HSCT in patients with NMD differs from that in malignant disorders for two important reasons: 1) these patients are typically naïve to chemotherapy and immunosuppression. This may potentially lead to difficulties with engraftment. And 2) RIC with subsequent bone marrow chimerism may be beneficial even in mixed chimerism and result in decreased transplant-related mortality (TRM). Nevertheless, any previous organ damage, as a result of the underlying disease, may remain present after the HSCT.
For other diseases (metabolic disorders, some immunodeficiencies, etc.), a transplant is not curative. For these diseases, the main intent of the transplant is to slow down, or stop, the progress of the disease. In select few cases/diseases, the presence of healthy bone marrow derived cells may even prevent progression and prevent neurological decline.
Research funds are not available to assist with enrollment on this trial.
In this research study, instead of using the standard myeloablative conditioning, the study doctor is using RIC, in which significantly lower doses of chemotherapy will be used. The lower doses may not eradicate every stem cell in the patient's bone marrow, however, in the presented combination, the intention is to eliminate already formed immune cells and provide maximum growth advantage to healthy donor stem cells. This paves the way to successful engraftment of donor stem cells. Engrafting donor stem cells can outcompete, and donor lymphocytes could suppress, the patients' surviving stem cells. With RIC, the side effects on the brain, heart, lung, liver, and other organ functions are less severe and late toxic effects should also be reduced.
The purpose of this study is to collect data from the patients undergoing reduced-intensity conditioning before HSCT, and compare it to the standard myeloablative conditioning. It is expected there will be therapeutic benefits, paired with better survival rate, less organ toxicity and improved quality of life, following the RIC compared to the myeloablative regimen.
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA)
- Being tested
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Keywords
- Severe Combined Immune Deficiency (SCID), 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, Dyskeratosis Congenita (DC), Congenital Amegakaryocytic Thrombocytopenia (CAMT), Osteopetrosis, Mucopolysaccharidoses, Hurler syndrome (MPS I), Hunter syndrome (MPS II)
- Medical subject headings
- Primary Immunodeficiency Diseases, Congenital Bone Marrow Failure Syndromes, Arthritis, Juvenile, Severe Combined Immunodeficiency, Syndrome, Wiskott-Aldrich Syndrome, Leukocyte adhesion deficiency type 1, Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
Also called hydroxycarbamide, Hydrea, Droxia
Oral administration
AlemtuzumabDrug
Also called Campath
Intravenous (IV) administration.
FludarabineDrug
Also called Fludara
IV administration
MelphalanDrug
Also called Melphalan hydrochloride, Alkeran
IV administration
ThiotepaDrug
IV administration
| Study group | Role | What they receive |
|---|---|---|
| UCBT:transfusion dependent anemias or increased rejection risk | Experimental | Alemtuzumab, Hydroxyurea, Fludarabine, Melphalan, and Thiotepa conditioning regimen prior to allogenic HSCT. Receives: Drug: Hydroxyurea; Drug: Alemtuzumab; Drug: Fludarabine; Drug: Melphalan; Drug: Thiotepa |
| BMT, PBSCT and not transfusion dependent UCBT | Experimental | Alemtuzumab, Hydroxyurea, Fludarabine, Melphalan, and Thiotepa conditioning regimen prior to allogenic HSCT. Receives: Drug: Hydroxyurea; Drug: Alemtuzumab; Drug: Fludarabine; Drug: Melphalan; Drug: Thiotepa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Post-transplant treatment-related mortality (TRM)
The number of deaths related to the research intervention at day 100, 6 months, and 1 year post-transplant.
Measured: 1 year post-transplant
Neurodevelopmental milestones
Evaluation of the pace of attaining neurodevelopmental milestones after reduced-intensity conditioning as compared to myeloablative conditioning historical controls from the target population(s).
Measured: 1 year post-transplant
Immune Reconstitution
Evaluation of the pace of immune reconstitution.
Measured: 1 year post-transplant
Severe opportunistic infections
Evaluation of the incidence of severe opportunistic infections.
Measured: 1 year post-transplant
GVHD occurrence
Description of the incidence of acute graft versus host disease (GVHD) (II-IV) and chronic extensive GVHD.
Measured: 1 year post-transplant
Secondary outcomes (6)
Donor cell engraftment
Determination of the feasibility of attaining robust donor cell engraftment (>50% donor chimerism at 6 months) following reduced-intensity conditioning (RIC) regimens prior to HSCT in the target population(s).
Measured: 6 months post-transplant
Normal enzyme level
Determination of the feasibility of attaining and sustaining normal enzyme levels in the target population(s).
Measured: 1 year post-transplant
Neutrophil recovery
Determination of the pace of neutrophil recovery.
Measured: 1 year post-transplant
Platelet recovery
Determination of the pace of platelet recovery.
Measured: 1 year post-transplant
Grade 3-4 organ toxicity
The number of grade 3-4 organ adverse events.
Measured: 1 year post-transplant
Late graft failure
Evaluation of the incidence of late graft failure.
Measured: 1 year post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2013 (estimated)
- Study start4 Feb 2014
- Primary completionNov 2026 (estimated)
- Study completionNov 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary Immunodeficiency (PID)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Paul Szabolcs
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Paul Szabolcs, University of Pittsburgh
- Investigators
- Paul Szabolcs, MD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01962415
- Sponsor's protocol ID
- STUDY19060337
- Acronym
- HSCT+RIC
- Status last verified
- Dec 2025
- First submitted
- 10 Oct 2013
- Last updated
- 15 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Vander Lugt MT, Chen X, Escolar ML, et al. Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Adv. 2020;4(13):3041-3052. Blood Adv. 2020 Aug 11;4(15):3508. doi: 10.1182/bloodadvances.2020002967. No abstract available.
PubMed 32750127Vander Lugt MT, Chen X, Escolar ML, Carella BA, Barnum JL, Windreich RM, Hill MJ, Poe M, Marsh RA, Stanczak H, Stenger EO, Szabolcs P. Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Adv. 2020 Jul 14;4(13):3041-3052. doi: 10.1182/bloodadvances.2020001940.
PubMed 32634238
Study at a glance
From the registry record, last updated by the sponsor on 15 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults ≤55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT01962415
- Acronym / sponsor ID
- HSCT+RIC / STUDY19060337
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA)
- Keywords
- Severe Combined Immune Deficiency (SCID), 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)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Paul SzabolcsUniversity, hospital or other
- Study start
- 4 Feb 2014 (actual)
- Primary completion
- Nov 2026 (estimated)
- Study completion
- Nov 2027 (estimated)
- First posted
- 14 Oct 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
For some non-malignant diseases (NMD; i.e., thalassemia, sickle cell disease, most immune deficiencies) a hematopoietic stem cell transplant may be curative by healthy donor stem cell engraftment alone. HSCT in patients with NMD differs from that in malignant disorders for two important reasons: 1) these patients are typically naïve to chemotherapy and immunosuppression. This may potentially lead to difficulties with engraftment. And 2) RIC with subsequent bone marrow chimerism may be beneficial even in mixed chimerism and result in decreased transplant-related mortality (TRM). Nevertheless, any previous organ damage, as a result of the underlying disease, may remain present after the HSCT.
For other diseases (metabolic disorders, some immunodeficiencies, etc.), a transplant is not curative. For these diseases, the main intent of the transplant is to slow down, or stop, the progress of the disease. In select few cases/diseases, the presence of healthy bone marrow derived cells may even prevent progression and prevent neurological decline.
Research funds are not available to assist with enrollment on this trial.
In this research study, instead of using the standard myeloablative conditioning, the study doctor is using RIC, in which significantly lower doses of chemotherapy will be used. The lower doses may not eradicate every stem cell in the patient's bone marrow, however, in the presented combination, the intention is to eliminate already formed immune cells and provide maximum growth advantage to healthy donor stem cells. This paves the way to successful engraftment of donor stem cells. Engrafting donor stem cells can outcompete, and donor lymphocytes could suppress, the patients' surviving stem cells. With RIC, the side effects on the brain, heart, lung, liver, and other organ functions are less severe and late toxic effects should also be reduced.
The purpose of this study is to collect data from the patients undergoing reduced-intensity conditioning before HSCT, and compare it to the standard myeloablative conditioning. It is expected there will be therapeutic benefits, paired with better survival rate, less organ toxicity and improved quality of life, following the RIC compared to the myeloablative regimen.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| UCBT:transfusion dependent anemias or increased rejection risk | Experimental | Drug: Hydroxyurea, Drug: Alemtuzumab, Drug: Fludarabine, Drug: Melphalan, Drug: ThiotepaAlemtuzumab, Hydroxyurea, Fludarabine, Melphalan, and Thiotepa conditioning regimen prior to allogenic HSCT. |
| BMT, PBSCT and not transfusion dependent UCBT | Experimental | Drug: Hydroxyurea, Drug: Alemtuzumab, Drug: Fludarabine, Drug: Melphalan, Drug: ThiotepaAlemtuzumab, Hydroxyurea, Fludarabine, Melphalan, and Thiotepa conditioning regimen prior to allogenic HSCT. |
Who may qualify
- Ages
- 2 months to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (55 listed)
- 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.
- Adequate organ function as measured by:
- Written informed consent and/or assent according to FDA guidelines.
- Negative pregnancy test if pubertal and/or menstruating.
- HIV negative.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxyureaAlso: hydroxycarbamide, Hydrea, DroxiaOral administration | Drug | BMT, PBSCT and not transfusion dependent UCBT, UCBT:transfusion dependent anemias or increased rejection risk |
| AlemtuzumabAlso: CampathIntravenous (IV) administration. | Drug | BMT, PBSCT and not transfusion dependent UCBT, UCBT:transfusion dependent anemias or increased rejection risk |
| FludarabineAlso: FludaraIV administration | Drug | BMT, PBSCT and not transfusion dependent UCBT, UCBT:transfusion dependent anemias or increased rejection risk |
| MelphalanAlso: Melphalan hydrochloride, AlkeranIV administration | Drug | BMT, PBSCT and not transfusion dependent UCBT, UCBT:transfusion dependent anemias or increased rejection risk |
| ThiotepaIV administration | Drug | BMT, PBSCT and not transfusion dependent UCBT, UCBT:transfusion dependent anemias or increased rejection risk |
Primary outcome
Post-transplant treatment-related mortality (TRM)
Time frame: 1 year post-transplant
The number of deaths related to the research intervention at day 100, 6 months, and 1 year post-transplant.
Neurodevelopmental milestones
Time frame: 1 year post-transplant
Evaluation of the pace of attaining neurodevelopmental milestones after reduced-intensity conditioning as compared to myeloablative conditioning historical controls from the target population(s).
Immune Reconstitution
Time frame: 1 year post-transplant
Evaluation of the pace of immune reconstitution.
Severe opportunistic infections
Time frame: 1 year post-transplant
Evaluation of the incidence of severe opportunistic infections.
GVHD occurrence
Time frame: 1 year post-transplant
Description of the incidence of acute graft versus host disease (GVHD) (II-IV) and chronic extensive GVHD.
6 secondary outcomes
Donor cell engraftment
Time frame: 6 months post-transplant
Normal enzyme level
Time frame: 1 year post-transplant
Neutrophil recovery
Time frame: 1 year post-transplant
Platelet recovery
Time frame: 1 year post-transplant
Grade 3-4 organ toxicity
Time frame: 1 year post-transplant
Late graft failure
Time frame: 1 year post-transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Dec 2025.
- Results publication: PubMed 32750127Vander Lugt MT, Chen X, Escolar ML, et al. Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Adv. 2020;4(13):3041-3052. Blood Adv. 2020 Aug 11;4(15):3508. doi: 10.1182/bloodadvances.2020002967. No abstract available.
- Derived from this study: PubMed 32634238Vander Lugt MT, Chen X, Escolar ML, Carella BA, Barnum JL, Windreich RM, Hill MJ, Poe M, Marsh RA, Stanczak H, Stenger EO, Szabolcs P. Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Adv. 2020 Jul 14;4(13):3041-3052. doi: 10.1182/bloodadvances.2020001940.
- Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disordersLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Paul Szabolcs, MD principal investigator
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxyurea, Alemtuzumab and Fludarabine in people with Primary Immunodeficiency (PID) and Congenital Bone Marrow Failure Syndromes, run by Paul Szabolcs at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 55 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Paul Szabolcs, MD (412-692-5427, paul.szabolcs@chp.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Post-transplant treatment-related mortality (TRM) (time frame: 1 year post-transplant). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01962415, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Dec 2025.