Trial · NCT00004143

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

NCT00004143, Phase 2, Completed, David Rizzieri, MD; results first posted 2013-11-11

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes. Trials, record NCT00004143. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00004143

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
NCT number: NCT00004143
Status: Completed
Phases: Phase 2
Enrollment: 2 participants, actual
Lead sponsor: David Rizzieri, MD
Conditions: Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
Interventions: Campath, Chemo and/or TBI Allo SCT
First posted: 2003-01-27
Last updated: 2014-12-05
Results first posted: 2013-11-11
Limitations and caveats: Small number of subjects analyzed

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Patients With Neutrophil Engraftment	1 year post transplant	Number of patients with neutrophil engraftment: Absolute Neutrophil Count (ANC) \> 500/μL and hemoglobin level remaining above 10 g/dL without transfusion support, with tests showing at least 2.5% donor cells present. Primary graft failure is defined as absence of establishment of adequate donor hematopoiesis by day 42 with bone marrow cellularity \< 5%, peripheral White Blood Count (WBC) \< 500/μL, peripheral ANC \< 100/μL, and/or platelets \< 10,000/μL by day 120 with absence of megakaryocytes in the bone marrow (in the absence of disease relapse).
Primary	Number of Patients With Platelet Engraftment	1 year post transplant	Number of patients with platelet engraftment - Platelets \> 20,000/μL and hemoglobin level remaining above 10 g/dL without transfusion support, with tests showing at least 2.5% donor cells present. Primary graft failure is defined as absence of establishment of adequate donor hematopoiesis by day 42 with bone marrow cellularity \< 5%, peripheral White Blood Count (WBC) \< 500/μL, peripheral ANC \< 100/μL, and/or platelets \< 10,000/μL by day 120 with absence of megakaryocytes in the bone marrow (in the absence of disease relapse).
Primary	Number of Patients With Grade 3-4 Acute Graft Versus Host Disease (GVHD)	60 days post transplant	Number of patients with Grade 3-4 acute Graft Versus Host Disease (GVHD). GVHD will be monitored at least two times per week through day 45, then weekly through day 60 and graded by 2 persons at each institution, to ensure internal consistency in grading.
Primary	Number of Participants With Grade 3-4 Unexpected Adverse Events	45 days post transplant	An unexpected adverse event is one that differs in the nature, severity, or frequency from (a) the research procedures that are described in the protocol-related documents, (such as the IRB-approved research protocol and informed consent document) as expected, and/or (b) the characteristics of the subject population being studied.
Primary	Number of Participants With Transplant-related Mortality	100 days	Number of patients who died due to transplant-related complications
Secondary	Overall Survival	2 years	Number of patients alive 2 years after transplant

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Units	Measured as
PRIMARY	Number of Patients With Neutrophil Engraftment	1 year post transplant	participants	NUMBER
PRIMARY	Number of Patients With Platelet Engraftment	1 year post transplant	participants	NUMBER
SECONDARY	Overall Survival	2 years	participants	NUMBER
PRIMARY	Number of Patients With Grade 3-4 Acute Graft Versus Host Disease (GVHD)	60 days post transplant	participants	NUMBER
PRIMARY	Number of Participants With Grade 3-4 Unexpected Adverse Events	45 days post transplant	participants	NUMBER
PRIMARY	Number of Participants With Transplant-related Mortality	100 days	participants	NUMBER

Source ClinicalTrials.gov

Results by group6

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Units
Number of Patients With Neutrophil Engraftment	All Patients	2	participants
Number of Patients With Platelet Engraftment	All Patients	2	participants
Overall Survival	All Patients	2	participants
Number of Patients With Grade 3-4 Acute Graft Versus Host Disease (GVHD)	All Patients	2	participants
Number of Participants With Grade 3-4 Unexpected Adverse Events	All Patients	0	participants
Number of Participants With Transplant-related Mortality	All Patients	0	participants

Source ClinicalTrials.gov

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	All Patients	2
Overall Study	COMPLETED	All Patients	2
Overall Study	NOT COMPLETED	All Patients	0

Source ClinicalTrials.gov

Adverse events4

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Hypertension	Cardiac disorders	All Patients	1	2	1
other	Headache	Nervous system disorders	All Patients	1	2	1
other	Cardiac ischemia / infarction	Cardiac disorders	All Patients	1	2	1
other	Parainfluenza	Infections and infestations	All Patients	1	2	1

Source ClinicalTrials.gov

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	All Patients
Total, other adverse events	All Patients	2	2
Total, serious adverse events	All Patients	0	2

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT00004143

GET https://conductscience.org/api/trials/v1/NCT00004143/registered

API reference For AI assistants