NCT04414046 TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children
| NCT ID | NCT04414046 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Johns Hopkins All Children's Hospital |
| Condition | Primary Immune Deficiency Disorders |
| Study Type | INTERVENTIONAL |
| Enrollment | 17 participants |
| Start Date | 2020-07-22 |
| Primary Completion | 2027-06-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
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 17 participants in total. It began in 2020-07-22 with a primary completion date of 2027-06-30.
⚠ 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
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.
Eligibility Criteria
Inclusion Criteria: 1. Patient with any form of primary immune deficiency/dysregulatory disorders characterized by aberrant immune function, abnormal hematopoiesis, systemic or organ specific autoimmunity and/or non-malignant lymphoproliferation. This includes, but not limited to: I. Disorders of phagocytes: Chronic granulomatous disease, Leukocyte adhesion deficiency, defects of IL-10 pathway, MonoMac syndrome II. Defects of cellular and humoral immunity: Severe Combined Immunodeficiency Disorder (infants with classic SCID up to 2 years of age will be excluded due to other open protocol), X-linked hyper-IgM syndrome, DOCK8 deficiency, ZAP70 deficiency, common variable immunodeficiency (CVID), Wiskott-Aldrich syndrome, NEMO deficiency. III. Disorder of immune dysregulation: Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome, CTLA4 deficiency, LRBA deficiency, STAT1 GOF, STAT3 GOF, X-linked lymphoproliferative disease etc. IV. Other PIDs and immune dysregulatory disorders who can be benefitted by HCT as deemed appropriate by the PI and the treating immunologist. 2. Histiocytic disorders including hemophagocytic lymphohistiocytosis (familial HLH (types 1-5), secondary HLH (refractory to therapy or with recurrent episodes of hyper inflammation) and multisystem refractory Langerhans cell histiocytosis. 3. Metabolic disorders that could improve or stabilize after stem cell transplantation such as mucopolysaccharidoses, neurodegenerative disorders, osteopetrosis, etc. Inclusion Criteria: 1. Patient has a suitable genotypic identical match of 5/10. The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-C, HLA-DRB1 and HLA-DQB1. 2. Patients must have adequate organ function measured by: 1. Cardiac: asymptomatic or if symptomatic then LVEF at rest must be ≥ 40% or SF ≥ 26% 2. Pulmonary: asymptomatic or if symptomatic DLCO ≥ 40% of predicted (corrected for hemoglobin) or pulse oximetry ≥ 92% on room air if the patient is unable to perform pulmonary function testing. 3. Renal: Creatinine clearance (CrCl) or glomerular filtration rate (GFR) must be \> 50 mL/min/1.73 m2. 4. Hepatic: Serum conjugated (direct) bilirubin \< 2.0 x ULN for age; AST and ALT \< 5.0 x ULN for age. 5. Karnofsky or Lansky (age-dependent) performance score ≥ 50 3. Signed written informed consent Exclusion Criteria: 1. Participants who have an HLA-matched sibling who is able and willing to donate bone marrow. Patients with a HLA-matched unrelated donors are not excluded. 2. Pregnant or breastfeeding females. 3. Patient has HIV or uncontrolled fungal, bacterial or viral infections. 4. Patient has received prior solid organ transplant. 5. Patient has active GVHD (\> grade II) or chronic extensive GVHD due to a previous allograft at the time of inclusion.
Contact & Investigator
Deepak Chellapandian, MD
PRINCIPAL INVESTIGATOR
Johns Hopkins All Children's Hospital
Frequently Asked Questions
Who can join the NCT04414046 clinical trial?
This trial is open to participants of all sexes, up to 21 Years, studying Primary Immune Deficiency Disorders. 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 NCT04414046 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 NCT04414046 currently recruiting?
Yes, NCT04414046 is actively recruiting participants. Contact the research team at jade.hanson@jhmi.edu for enrollment information.
Where is the NCT04414046 trial being conducted?
This trial is being conducted at St. Petersburg, United States.
Who is sponsoring the NCT04414046 clinical trial?
NCT04414046 is sponsored by Johns Hopkins All Children's Hospital. The principal investigator is Deepak Chellapandian, MD at Johns Hopkins All Children's Hospital. The trial plans to enroll 17 participants.