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NCT03520647 · ClinicalTrials.gov registry record · Phase 2

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

A Phase 2 study of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Severe Aplastic Anemia (SAA), sponsored by National Heart, Lung, and Blood Institute (NHLBI).

Recruiting
Registry status
Phase 2
Development phase
56
Enrollment target
1
Study location

NCT03520647: Recruiting Phase 2 study of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Severe Aplastic Anemia (SAA), sponsored by National Heart, Lung, and Blood Institute (NHLBI).

NCT03520647 is a Phase 2 study of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Severe Aplastic Anemia (SAA) that is actively recruiting participants, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 56 participants, below the 115-participant average among 5 other Paroxysmal Nocturnal Hemoglobinuria (PNH) trials with a reported enrollment target (51% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT03520647, a Phase 2 study of Paroxysmal Nocturnal Hemoglobinuria (PNH) and Severe Aplastic Anemia (SAA), is actively recruiting participants, sponsored by National Heart, Lung, and Blood Institute (NHLBI).

RECRUITING
Registry status
Phase 2
Development phase
56 participants
Enrollment target
1
Study location

Study Summary

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.

Primary Outcome

evaluate 1 year chronic GVHD-free survival rate (defined by the percentage of patients who are alive with no evidence of moderate or severe chronic GVHD at 1 year) of using G-CSF mobilized peripheral stem cells and post haplo-identical transplantation cyclophosphamide in subjects with severe aplastic anemia or refractory anemia (RA) or SAA subjects

Conditions Studied

Paroxysmal Nocturnal Hemoglobinuria (PNH) Severe Aplastic Anemia (SAA) Hypo-Plastic Myelodysplastic Syndrome (MDS)

Interventions

  • DRUG Cyclophosphamide
  • OTHER Peripheral Blood Stem Cells

Study Locations (1)

Maryland

  • National Institutes of Health Clinical Center - Bethesda

Trial Details

FieldValue
Enrollment Target 56 participants
Start Date 2019-02-19
Est. Completion 2028-06-01
Phase Phase 2
National Heart, Lung, and Blood Institute (NHLBI)

501 total trials

What NCT03520647 shows while recruiting

NCT03520647 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 56 participants, a relatively small participant target, below the 115-participant average among 5 other Paroxysmal Nocturnal Hemoglobinuria (PNH) trials with a reported enrollment target (51% lower).

The record links to 3 conditions, with Paroxysmal Nocturnal Hemoglobinuria (PNH) appearing as the primary indexed condition, and to 2 interventions - of which Cyclophosphamide is the first listed.

NCT03520647 reports a single indexed study location in Maryland.

Frequently Asked Questions

What is clinical trial NCT03520647 about?

NCT03520647 is a clinical study titled "Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis". 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...

What is the current status of trial NCT03520647?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 56 participants. The study started on 2019-02-19. Estimated completion is 2028-06-01.

What conditions does trial NCT03520647 study?

This clinical trial studies the following conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH), Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS).

What interventions are being tested in trial NCT03520647?

The interventions under investigation include: Cyclophosphamide (DRUG), Peripheral Blood Stem Cells (OTHER).

Who is sponsoring clinical trial NCT03520647?

This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT03520647 being conducted?

This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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More recruiting context for NCT03520647

Source: ClinicalTrials.gov NCT03520647, the US trial registry maintained by the National Library of Medicine. NCT03520647 (small enrollment · single site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.