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

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. According to ClinicalTrials.gov, the official US trial registry.

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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.

Primary Outcome

Rate of donor engraftment ≥95% at day 60 as measured by donor chimerism in whole blood.

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
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

523 total trials

What NCT04232085 shows while recruiting

NCT04232085 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 27 participants, a relatively small participant target, below the 334-participant average among 15 other Fanconi Anemia trials with a reported enrollment target (92% lower).

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 a single indexed study location in 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.

Where NCT04232085's enrollment target sits among peer trials

27 9th of 15 higher than 7 of 15 other Fanconi Anemia trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Fanconi Anemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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More recruiting context for NCT04232085

Source: ClinicalTrials.gov NCT04232085, the US trial registry maintained by the National Library of Medicine. NCT04232085 (small enrollment · single site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.