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NCT04232085
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Phase 2 trial testing Alemtuzumab in Primary Immune Deficiency Disorder in 27 participants. Currently enrolling.
31 December 2027
Quick facts
| Lead sponsor | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins |
|---|---|
| Phase | Phase 2 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 27 |
| Start date | 12 February 2020 |
| Primary completion | 31 December 2027 |
| Estimated completion | 31 December 2028 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Alemtuzumab (ALEMTUZUMAB) — full drug profile →
- Fludarabine (FLUDARABINE) — full drug profile →
- Melphalan
- Low Dose Total Body Irradiation
- Cyclophosphamide (cyclophosphamide) — full drug profile →
- Tacrolimus (TACROLIMUS) — full drug profile →
- Mycophenolate Mofetil (MYCOPHENOLATE MOFETIL) — full drug profile →
Conditions studied
- Primary Immune Deficiency Disorder — all drugs for Primary Immune Deficiency Disorder →
- Immune Deficiency Disease — all drugs for Immune Deficiency Disease →
- Bone Marrow Failure — all drugs for Bone Marrow Failure →
- Short Telomere Length — all drugs for Short Telomere Length →
Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins — full company profile →
Who can join
Adults 4 Months to 50, any sex, with Primary Immune Deficiency Disorder or Immune Deficiency Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.
Publications & conference data
3 peer-reviewed publications reference this trial (live from Europe PMC):
-
Post-transplantation Cyclophosphamide: From HLA-Haploidentical to Matched-Related and Matched-Unrelated Donor Blood and Marrow Transplantation.
Williams L, Cirrone F, Cole K, Abdul-Hay M, et al · · 2020 · cited 32× · PMID 32373119 · DOI 10.3389/fimmu.2020.00636 -
New therapeutic targets and biomarkers for acute graft-versus-host disease (GVHD).
Choe H, Ferrara JLM. · · 2021 · cited 25× · PMID 34669521 · DOI 10.1080/14728222.2021.1992383 -
Reduced Intensity Bone Marrow Transplantation with Post-Transplant Cyclophosphamide for Pediatric Inherited Immune Deficiencies and Bone Marrow Failure Syndromes.
Klein OR, Bapty S, Lederman HM, Younger MEM, et al · · 2021 · cited 14× · PMID 33159275 · DOI 10.1007/s10875-020-00898-0
Verify or expand the search:
- PubMed search for NCT04232085
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of Alemtuzumab
Trials testing the same drug.
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- NCT06872333 — Allo HSCT for High Risk Hemoglobinopathies · Phase 2 · recruiting
- NCT05501756 — Precision Alemtuzumab Dosing for Allogeneic Hematopoietic Cell Transplantation · Phase 2 · recruiting
- NCT05384756 — TMLI and Alemtuzumab for Treatment of Sickle Cell Disease · Phase 1 · active not recruiting
- NCT06310343 — ADAs to Alemtuzumab · completed
Other recruiting trials for Primary Immune Deficiency Disorder
Currently open trials in the same condition.
- NCT03266653 — EBV-specific Cytotoxic T-lymphocytes (CTLs) for Refractory EBV Infection · Phase 2 · recruiting
- NCT04197596 — Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs) · Phase 1, PHASE2 · recruiting
- NCT03266640 — Virus Specific Cytotoxic T-Lymphocytes (CTLs) for Refractory Cytomegalovirus (CMV) · Phase 2 · recruiting
- NCT03266627 — Adenovirus-specific Cytotoxic T-lymphocytes for Refractory Adenovirus Infection · Phase 2 · recruiting
Other Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins trials
Trials by the same sponsor.
- NCT07424222 — Ruxolitinib for Immune Effector Cell Associated Hemophagocytic Lymphohistiocytosis-like Syndrome (RISE) · Phase 1 · not yet recruiting
- NCT06210854 — Assessing Administration of pBI-11 Via Electroporation for the Treatment of Patients With HPV16/18+ · Phase 2 · not yet recruiting
- NCT07360119 — Total Pancreatectomy With Islet Autotransplantation (TPIAT) for High-Risk Patients With Pancreatic Tumors · NA · not yet recruiting
- NCT07140315 — DK222 Study at Hopkins · Phase 1 · recruiting
- NCT07071155 — Momelotinib in Combination With Hypomethylating Agent for Chronic Phase Myelodysplastic Syndromes/Myeloproliferative Ove · EARLY_PHASE1 · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04232085 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Last refreshed: 28 November 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04232085.
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