Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.

NCT04232085 · ClinicalTrials.gov registry record · Phase 2

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

A Phase 2 study of Fanconi Anemia and Dyskeratosis Congenita, sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

Recruiting
Registry status
Phase 2
Development phase
27
Enrollment target
1
Study location

NCT04232085 is a Phase 2 study of Fanconi Anemia and Dyskeratosis Congenita that is actively recruiting participants, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins. The registered enrollment target is 27 participants, below the 334-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (92% lower). The trial reports 1 study location across 1 state.

View on ClinicalTrials.gov ↗

View your shortlist →

The verdict

NCT04232085, a Phase 2 study of Fanconi Anemia and Dyskeratosis Congenita, is actively recruiting participants, sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

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

Study Summary

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.

Conditions Studied

Fanconi Anemia Dyskeratosis Congenita Bone Marrow Failure Primary Immune Deficiency Disorder Hoyeraal-Hreidarsson Syndrome Telomere Biology Disorder Immune Deficiency Disease Short Telomere Length Non Fanconi DNA-DSB Repair Disorder Short Telomere Syndrome

Interventions

  • DRUG Cyclophosphamide
  • DRUG Fludarabine
  • DRUG Melphalan
  • DRUG Alemtuzumab
  • RADIATION Low Dose Total Body Irradiation

Study Locations (1)

Maryland

  • Johns Hopkins University - Baltimore

Trial Details

FieldValue
Enrollment Target 27 participants
Start Date 2020-02-12
Est. Completion 2028-12-31
Phase Phase 2

Sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

523 total trials

Interested in This Trial?

Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04232085

The ClinicalTrials.gov registry entry for NCT04232085 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 27 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 334-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (92% lower). The listed sponsor is Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, which has 523 total studies on file at ClinicalTrials.gov.

The record links to 10 conditions, with Fanconi Anemia appearing as the primary indexed condition, and to 5 interventions - of which Cyclophosphamide is the first listed.

NCT04232085 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.

Frequently Asked Questions

What is clinical trial NCT04232085 about?

NCT04232085 is a clinical study titled "Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures". 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 bo...

What is the current status of trial NCT04232085?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 27 participants. The study started on 2020-02-12. Estimated completion is 2028-12-31.

What conditions does trial NCT04232085 study?

This clinical trial studies the following conditions: Fanconi Anemia, Dyskeratosis Congenita, Bone Marrow Failure, Primary Immune Deficiency Disorder, Hoyeraal-Hreidarsson Syndrome.

What interventions are being tested in trial NCT04232085?

The interventions under investigation include: Cyclophosphamide (DRUG), Fludarabine (DRUG), Melphalan (DRUG), Alemtuzumab (DRUG), Low Dose Total Body Irradiation (RADIATION).

Who is sponsoring clinical trial NCT04232085?

This trial is sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, which has 523 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04232085 being conducted?

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

Learn More About Clinical Trials

Similar trials for Fanconi Anemia

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Explore more on PlainTrial

Fanconi Anemia

All registered trials for this condition

Dyskeratosis Congenita

All registered trials for this condition

Maryland

All trials with a study site in Maryland

Recruiting now

Find trials open to new participants near you

Rankings

Top sponsors, conditions, and phases

Read our methodology - how this data is sourced, computed, and verified.

Related

Browse all → | Rankings →
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.