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NCT01050855 · ClinicalTrials.gov registry record · Phase 2

Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

A Phase 2 study of Hemoglobinopathies and Immunodeficiencies, sponsored by Children's Hospital of Philadelphia.

Active
Registry status
Phase 2
Development phase
75
Enrollment target
1
Study location

NCT01050855: Active Phase 2 study of Hemoglobinopathies and Immunodeficiencies, sponsored by Children's Hospital of Philadelphia.

NCT01050855 is a Phase 2 study of Hemoglobinopathies and Immunodeficiencies that is active but no longer recruiting, run by Children's Hospital of Philadelphia. The registered enrollment target is 75 participants, below the 1,799-participant average among 17 other Hemoglobinopathies trials with a reported enrollment target (96% 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

NCT01050855, a Phase 2 study of Hemoglobinopathies and Immunodeficiencies, is active but no longer recruiting, sponsored by Children's Hospital of Philadelphia.

ACTIVE NOT RECRUITING
Registry status
Phase 2
Development phase
75 participants
Enrollment target
1
Study location

Study Summary

This is a Phase II pilot study to evaluate engraftment and toxicity of patients with non-malignant diseases using a reduced intensity conditioning regimen in the setting of allogeneic transplant for non malignant diseases. Bone Marrow or cord blood will be acceptable as a stem cell source. Recently, reduced intensity conditioning (RIC) regimens have been used for both adult patients with leukemias and pediatric patients with non-malignant diseases. These regimens are better tolerated, resulting in less transplant related morbidity and mortality. Stable mixed chimerism, while insufficient for eradication of leukemias, may be sufficient to cure patients with non-malignant diseases.

Primary Outcome

engraftment of patients with non-malignant disorders will be evaluated using a reduced-intensity conditioning regimen

Interventions

  • DRUG RIC: Distal Campath
  • DRUG RIC:Intermediate Campath
  • DRUG RIC: Mini Busulfan

Study Locations (1)

Pennsylvania

  • The Children's Hospital of Philadelphia - Philadelphia

Trial Details

FieldValue
Enrollment Target 75 participants
Start Date 2008-01
Est. Completion 2026-12
Phase Phase 2
Children's Hospital of Philadelphia

431 total trials

What the registry record for NCT01050855 still lists

NCT01050855 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 75 participants, a relatively small participant target, below the 1,799-participant average among 17 other Hemoglobinopathies trials with a reported enrollment target (96% lower).

The record links to 3 conditions, with Hemoglobinopathies appearing as the primary indexed condition, and to 3 interventions - of which RIC: Distal Campath is the first listed.

NCT01050855 reports a single indexed study location in Pennsylvania.

Frequently Asked Questions

What is clinical trial NCT01050855 about?

NCT01050855 is a clinical study titled "Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders". This is a Phase II pilot study to evaluate engraftment and toxicity of patients with non-malignant diseases using a reduced intensity conditioning regimen in the setting of allogeneic transplant for non malignant diseases. Bone Marrow or cord blood will be acceptable as a stem cell source. Recently...

What is the current status of trial NCT01050855?

This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 75 participants. The study started on 2008-01. Estimated completion is 2026-12.

What conditions does trial NCT01050855 study?

This clinical trial studies the following conditions: Hemoglobinopathies, Immunodeficiencies, Non Malignant Diseases.

What interventions are being tested in trial NCT01050855?

The interventions under investigation include: RIC: Distal Campath (DRUG), RIC:Intermediate Campath (DRUG), RIC: Mini Busulfan (DRUG).

Who is sponsoring clinical trial NCT01050855?

This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT01050855 being conducted?

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

Similar trials for Hemoglobinopathies

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 NCT01050855's enrollment target sits among peer trials

75 3rd of 17 higher than 15 of 17 other Hemoglobinopathies trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Hemoglobinopathies 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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Source: ClinicalTrials.gov NCT01050855, the US trial registry maintained by the National Library of Medicine. NCT01050855 (small enrollment · single site footprint · active not recruiting) retrieved and formatted by PlainTrial, see methodology.