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Hematologic DiseasesPhase 2NCT00029380

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

A Phase 2 study testing Sangstat, Cyclophosphamide and Busulfan in people with Hematologic Diseases and Anemia, Sickle Cell, run by National Heart, Lung, and Blood Institute (NHLBI) at 15 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study will develop a national cord blood bank for siblings of patients with hemoglobinopathies and thalassemia. Read more about this study

Who is it for?
Ages 3 to 14 years
What is being tested?
Sangstat, Cyclophosphamide and Busulfan
Type of treatment
Surgery or procedure
How many people
30 participants
Where
15 sitesin 2 countries
Study dates
Jan 1999to Aug 2006
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 14 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Suitable UCB collection from an HLA-identical sibling
  • Sickle cell anemia (Hb SS or S beta thalassemia) with significant disease manifestations as defined by at least one of the following criteria:
  • A history of painful events defined as three or more painful events in the 2 years prior to enrollment. Pain may occur in typical sites associated with vaso-occlusive painful events and cannot be explained by causes other than sickle cell disease. The pain must last at least 4 hours and require treatment with either parenteral narcotics, an equianalgesic dose of oral narcotics (if pain is treated in a local facility where parenteral narcotics are not routinely used to treat painful events), or parenteral nonsteroidal anti-inflammatory drugs. Painful events managed at home will be considered only if there is documentation of the event in a clinical record that may be reviewed by an investigator.
  • Acute chest syndrome (ACS) with two or more episodes of ACS with the development of a new infiltrate on chest radiograph and/or having a perfusion defect demonstrable on a lung radioisotope scan
  • Any combination of painful events and episodes of ACS that total three events in the 2 years before transplantation
  • Any clinically significant neurologic event (stroke or hemorrhage) or any neurologic defect lasting more than 24 hours
Show all 15Show fewer
  • Abnormal cerebral MRI and abnormal cerebral MRA
  • An episode of dactylitis in the first year of life with significant anemia (Hbg less than 7 g/dL), or leukocytosis in the second year of life such that the risk of a severe adverse outcome before 18 years of age exceeds 54% (as defined by the cooperative study of sickle cell disease (CSSCD) infant cohort study)
  • History of positive trans-cranial Doppler studies (average greater than 200 cm/sec)
  • Beta thalassemia major with significant disease manifestations as defined by the following criteria: Beta thalassemia genotype consistent with clinical diagnosis of beta thalassemia major (could include patients with E-beta thalassemia genotype) and requiring eight or more red blood cell (RBC) transfusions a year and iron chelation therapy. Younger patients who are at risk of transfusional iron overload but who have not yet initiated iron chelation therapy will be eligible.
  • Adequate physical function as measured by the following criteria:
  • Cardiac: Asymptomatic or, if symptomatic, then left ventricular ejection fraction at rest must be greater than 40% and must improve with exercise, or shortening fraction greater than 26%
  • Hepatic: Less than 5 times the clinical baseline of AST and less than 2.5 times the clinical baseline mg/dL of total serum bilirubin (clinical baseline is determined from the mean of the four most recent test results)
  • Renal: Serum creatinine within normal range for age or if serum creatinine is outside normal range for age then renal function (creatinine clearance or GFR) greater than 50% of the lower limit of normal (LLN) for age
  • Pulmonary: Asymptomatic, or, if symptomatic, DLCO, FEV1, FEC (diffusion capacity) greater than 45% of predicted (corrected for hemoglobin); if unable to obtain PFT, oxygen saturation greater than 85% on room air

Who cannot join

None listed.

Trial locations and contacts

15 sites in 2 countries.

15 sites in 2 countries

United States14 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Medical University of South Carolina

    Charleston, South Carolina, 29403

  • Children's Memorial Hospital

    Chicago, Illinois, 60614

  • University of Texas Southwestern Medical Center - Dallas

    Dallas, Texas, 75235

  • Duke University Medical Center Children's Hospital

    Durham, North Carolina

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Nemours Children's Clinic

    Jacksonville, Florida, 32207

  • University of Miami Batchelor Children's Research Center

    Miami, Florida, 33136

  • Louisiana State University Children's Medical Center

    New Orleans, Louisiana

  • Children's Hospital Oakland

    Oakland, California, 94609

  • Children's Hospital, Oakland

    Oakland, California, 94609

  • Children's Hospital Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Texas Transplant Institute

    San Antonio, Texas, 78229

  • Children's National Medical Center

    Washington D.C., District of Columbia

Other trials in United States

Canada1 site

  • Hopital Ste-Justine

    Montreal, Quebec

Other trials in Canada

About this study

Official title: Sibling Donor Cord Blood Banking and Transplantation

This study will develop a national cord blood bank for siblings of patients with hemoglobinopathies and thalassemia.

Read the detailed description

BACKGROUND:

During the past decade, a number of advances have been made in the treatment of patients with sickle cell anemia and thalassemia. Among these advances is allogeneic bone marrow transplantation, which is the only current treatment that offers a potential for cure. In sickle cell anemia, transplantation has been performed in patients who have had advanced organ damage. In thalassemia, transplantation has been performed before having any evidence of iron-related tissue damage. Due to concerns over engraftment and graft versus host disease (GVHD), transplants for patients with hemoglobinopathies have been limited to situations in which a human leukocyte antigen (HLA) compatible donor existed. Unfortunately, an HLA-matched related donor is often not available. Umbilical cord blood (UCB), a recently recognized source of hematopoietic stem cells, has been used to successfully transplant bone marrow to over 500 patients. The potential advantage of cord blood over other donor sources of stem cells is the minimal risk of high-grade GVHD (even without complete HLA compatibility).

DESIGN NARRATIVE:

This study will establish a national sibling donor cord blood (SDCB) program, evaluate its use in a multi-center pilot study of transplantation, and develop a Web-based data management system to support these two projects. A multi-center pilot study was conducted on cord blood transplantation in children with either sickle cell disease or thalassemia. The investigators tested the hypothesis that a novel immunosuppressive conditioning regimen (fludarabine, cyclophosphamide, and busulfan) and post transplant therapy (mycophenolate mofetil and cyclosporine) would improve engraftment rates and prevent disease recurrence. The effect of SDCB transplantation on hematologic parameters and GVHD was monitored. Enrollment in the study was suspended on December 29, 2003. The protocol was revised, replacing the previous conditioning regimen of fludarabine, busulfan, and cyclophosphamide with a more conventional regimen of rabbit anti-thymocyte globulin (Sangstat), busulfan, and cyclophosphamide. The revised protocol is open for enrollment.

Medical subject headings
Hematologic Diseases, Anemia, Sickle Cell, beta-Thalassemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Hematologic parameters

  2. GVHD

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Jan 2002 (estimated)
  2. Study startJan 1999
  3. Primary completionAug 2006
  4. Study completionAug 2006

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00029380
Sponsor's protocol ID
141
Other IDs
U01HL061877
Status last verified
Sep 2008
First submitted
10 Jan 2002
Last updated
29 Jul 2016
Results posted
No

Publications

  • Reed W, Walters M, Lubin BH. Collection of sibling donor cord blood for children with thalassemia. J Pediatr Hematol Oncol. 2000 Nov-Dec;22(6):602-4. doi: 10.1097/00043426-200011000-00031.

    PubMed 11132238

  • Lubin BH, Eraklis M, Apicelli G. Umbilical cord blood banking. Adv Pediatr. 1999;46:383-408. No abstract available.

    PubMed 10645470

  • Woodard P, Lubin B, Walters CM. New approaches to hematopoietic cell transplantation for hematological diseases in children. Pediatr Clin North Am. 2002 Oct;49(5):989-1007. doi: 10.1016/s0031-3955(02)00026-3.

    PubMed 12430622

  • Reed W, Smith R, Dekovic F, Lee JY, Saba JD, Trachtenberg E, Epstein J, Haaz S, Walters MC, Lubin BH. Comprehensive banking of sibling donor cord blood for children with malignant and nonmalignant disease. Blood. 2003 Jan 1;101(1):351-7. doi: 10.1182/blood-2002-02-0394. Epub 2002 Aug 8.

    PubMed 12393579

  • Locatelli F, Rocha V, Reed W, Bernaudin F, Ertem M, Grafakos S, Brichard B, Li X, Nagler A, Giorgiani G, Haut PR, Brochstein JA, Nugent DJ, Blatt J, Woodard P, Kurtzberg J, Rubin CM, Miniero R, Lutz P, Raja T, Roberts I, Will AM, Yaniv I, Vermylen C, Tannoia N, Garnier F, Ionescu I, Walters MC, Lubin BH, Gluckman E; Eurocord Transplant Group. Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood. 2003 Mar 15;101(6):2137-43. doi: 10.1182/blood-2002-07-2090. Epub 2002 Nov 7.

    PubMed 12424197

  • Reed W, Walters M, Trachtenberg E, Smith R, Lubin BH. Sibling donor cord blood banking for children with sickle cell disease. Pediatr Pathol Mol Med. 2001 Mar-Apr;20(2):167-74.

    PubMed 12673840

  • Leonard A, Furstenau D, Abraham A, Darbari DS, Nickel RS, Limerick E, Fitzhugh C, Hsieh M, Tisdale JF. Reduction in vaso-occlusive events following stem cell transplantation in patients with sickle cell disease. Blood Adv. 2023 Jan 24;7(2):227-234. doi: 10.1182/bloodadvances.2022008137.

    PubMed 36240296

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Sangstat, Cyclophosphamide and Busulfan in people with Hematologic Diseases and Anemia, Sickle Cell, run by National Heart, Lung, and Blood Institute (NHLBI) at 15 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Hematologic parameters. The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00029380, which also lists 7 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2016; AzTrials last refreshed its copy on 8 Nov 2026.

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