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Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

StatusRecruiting
PhasePhase 2
Started
View on ClinicalTrials.gov ↗

Record History

The 50 most recent of 341 record versions are accounted for below. See every version on ClinicalTrials.gov ↗

  1. Minor 4 versions Re-verified since Jun 16, 2026, no change to tracked fieldsv338-v341
  2. Notable Primary completion pushed from Jun 1, 2026 to Jun 1, 2027v337
  3. Minor 6 versions Re-verified since Apr 23, 2026, no change to tracked fieldsv331-v336
  4. Minor Eligibility criteria shortened, about 1 wordv330
  5. Minor Eligibility criteria expanded, about 35 wordsv329
Show 37 earlier versionsHide 37 earlier versions
  1. Minor 7 versions Re-verified since Aug 23, 2025, no change to tracked fieldsv322-v328
  2. Minor Eligibility criteria revisedv321
  3. Minor 2 versions Re-verified since Jun 18, 2025, no change to tracked fieldsv319-v320
  4. Notable Primary completion date: Jun 1, 2025 to Jun 1, 2026v318
  5. Minor 10 versions Re-verified since May 25, 2024, no change to tracked fieldsv308-v317
  6. Minor Eligibility criteria expanded, about 99 wordsv307
  7. Minor 2 versions Re-verified since Feb 23, 2024, no change to tracked fieldsv305-v306
  8. Minor Study Status, Study Descriptionv304
  9. Minor 4 versions Re-verified since Dec 7, 2023, no change to tracked fieldsv300-v303
  10. Minor Study Status, Eligibilityv299
  11. Minor 7 versions Re-verified since Jul 5, 2022, no change to tracked fieldsv292-v298
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants. Objectives: To see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy. Eligibility: Recipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75 Design: Recipients will have: * Blood, urine, heart, and lung tests * Scans * Bone marrow sample Recipients will need a caregiver for several months. They may make fertility plans and a power of attorney. Donors will have blood and tissue tests, then injections to boost stem cells for 5-7 days. Donors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg. In the hospital for about 1 month, recipients will have: * Central line inserted in the neck or chest * Medicines for side effects * Chemotherapy over 8 days and radiation 1 time * Stem cell transplant over 4 hours Up to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests. At day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.
Trial Details
NCT Number NCT03520647
Lead Sponsor National Heart, Lung, and Blood Institute (NHLBI)
Conditions
(from sponsor)
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
Related terms Anemia, Aplastic, Hemoglobinuria, Paroxysmal
Enrollment 56 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov