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NCT04784052 · ClinicalTrials.gov registry record · Phase 1

Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab

A Phase 1 study of Fanconi Anemia, sponsored by Porteus, Matthew, MD.

Recruiting
Registry status
Phase 1
Development phase
18
Enrollment target
1
Study location

NCT04784052 is a Phase 1 study of Fanconi Anemia that is actively recruiting participants, run by Porteus, Matthew, MD. The registered enrollment target is 18 participants, below the 335-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state.

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The verdict

NCT04784052, a Phase 1 study of Fanconi Anemia, is actively recruiting participants, sponsored by Porteus, Matthew, MD.

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

Study Summary

The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191 as a part of conditioning. This experimental treatment will hopefully cause fewer side effects than chemotherapy (the current standard of care method). Participants will be administered the conditioning regimen, are assessed until they receive the depleted stem cell infusion, and will be followed for up to 2 years after the cell infusion.

Conditions Studied

Fanconi Anemia

Interventions

  • DRUG Cyclophosphamide
  • DEVICE CliniMACS Prodigy System
  • DRUG JSP191
  • BIOLOGICAL Depleted Stem Cell Transplant
  • BIOLOGICAL Rabbit Anti-Thymoglobulin (rATG)

Study Locations (1)

California

  • Stanford University - Stanford

Trial Details

FieldValue
Enrollment Target 18 participants
Start Date 2021-12-07
Est. Completion 2028-12
Phase Phase 1

Sponsor

Porteus, Matthew, MD

2 total trials

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What the Registry Record Tells You About NCT04784052

The ClinicalTrials.gov registry entry for NCT04784052 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 18 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 335-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (95% lower). The listed sponsor is Porteus, Matthew, MD, which has 2 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Fanconi Anemia appearing as the primary indexed condition, and to 5 interventions - of which Cyclophosphamide is the first listed.

NCT04784052 reports 1 study location spanning 1 distinct geographic area - top geographies include California.

Frequently Asked Questions

What is clinical trial NCT04784052 about?

NCT04784052 is a clinical study titled "Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab". The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor with and without using an experimental antibody treatment called JSP-191...

What is the current status of trial NCT04784052?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 18 participants. The study started on 2021-12-07. Estimated completion is 2028-12.

What conditions does trial NCT04784052 study?

This clinical trial studies the following conditions: Fanconi Anemia.

What interventions are being tested in trial NCT04784052?

The interventions under investigation include: Cyclophosphamide (DRUG), CliniMACS Prodigy System (DEVICE), JSP191 (DRUG), Depleted Stem Cell Transplant (BIOLOGICAL), Rabbit Anti-Thymoglobulin (rATG) (BIOLOGICAL).

Who is sponsoring clinical trial NCT04784052?

This trial is sponsored by Porteus, Matthew, MD, which has 2 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04784052 being conducted?

This trial has 1 study location across California. 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

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.