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NCT03653338 · ClinicalTrials.gov registry record · Phase 1

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

A Phase 1 study of Sickle Cell Anemia and Diamond-Blackfan Anemia, sponsored by Paul Szabolcs.

Recruiting
Registry status
Phase 1
Development phase
5
Enrollment target
1
Study location

NCT03653338 is a Phase 1 study of Sickle Cell Anemia and Diamond-Blackfan Anemia that is actively recruiting participants, run by Paul Szabolcs. The registered enrollment target is 5 participants, below the 100-participant average among 22 other Sickle Cell Anemia trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state.

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The verdict

NCT03653338, a Phase 1 study of Sickle Cell Anemia and Diamond-Blackfan Anemia, is actively recruiting participants, sponsored by Paul Szabolcs.

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

Study Summary

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.

Interventions

  • DRUG Alemtuzumab
  • DRUG Hydroxyurea
  • DRUG Rituximab
  • BIOLOGICAL CD3/CD19 depleted leukocytes
  • BIOLOGICAL CD45RA depleted leukocytes

Study Locations (1)

Pennsylvania

  • Children's Hospital of Pittsburgh of UPMC - Pittsburgh

Trial Details

FieldValue
Enrollment Target 5 participants
Start Date 2018-08-02
Est. Completion 2027-08-01
Phase Phase 1

Sponsor

Paul Szabolcs

7 total trials

What the Registry Record Tells You About NCT03653338

The ClinicalTrials.gov registry entry for NCT03653338 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 5 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 100-participant average among 22 other Sickle Cell Anemia trials with a reported enrollment target (95% lower). The listed sponsor is Paul Szabolcs, which has 7 total studies on file at ClinicalTrials.gov.

The record links to 3 conditions, with Sickle Cell Anemia appearing as the primary indexed condition, and to 5 interventions - of which Alemtuzumab is the first listed.

NCT03653338 reports 1 study location spanning 1 distinct geographic area - top geographies include Pennsylvania.

Frequently Asked Questions

What is clinical trial NCT03653338 about?

NCT03653338 is a clinical study titled "T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias". The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidenti...

What is the current status of trial NCT03653338?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2018-08-02. Estimated completion is 2027-08-01.

What conditions does trial NCT03653338 study?

This clinical trial studies the following conditions: Sickle Cell Anemia, Diamond-Blackfan Anemia, Beta-thalassemia Major.

What interventions are being tested in trial NCT03653338?

The interventions under investigation include: Alemtuzumab (DRUG), Hydroxyurea (DRUG), Rituximab (DRUG), CD3/CD19 depleted leukocytes (BIOLOGICAL), CD45RA depleted leukocytes (BIOLOGICAL).

Who is sponsoring clinical trial NCT03653338?

This trial is sponsored by Paul Szabolcs, which has 7 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT03653338 being conducted?

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

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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