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Anemia, Sickle CellPhase 1/2NCT01419704

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

A Phase 1/2 study testing Enriched Hematopoetic Stem Cell Infusion in people with Anemia, Sickle Cell and Complex and Transfusion-dependent Hemoglobinopathies, run by Talaris Therapeutics Inc. at 3 sites in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies. Read more about this study

Who is it for?
Ages Up to 45 years
What is being tested?
Enriched Hematopoetic Stem Cell Infusion
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
3 sitesUnited States
Study dates
May 2011to May 2016
Phase
Phase 1/2
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 Up to 45 years
  • All sexes
  • No healthy volunteers
  • Child, Adult
  1. Inclusion Criteria The following criteria are established to identify subjects with hemoglobinopathies, hematologic or bone marrow failure syndromes who have a high predicted morbidity and are at risk for early mortality:
  • Patients with alpha or beta thalassemia major.
  • Patients with Diamond-Blackfan anemia and other bone marrow failure syndromes, characterized by severe chronic anemia.
  • Patients with other complex and transfusion-dependent hemoglobinopathies, including sickle cell disease.
  • Patients with sickle disease who have one or more of the following:
  • Overt or silent stroke
  • Neurocognitive impairment
  • Pain crises 2 or more episodes per year for past year
  • One or more episodes of acute chest syndrome
  • Osteonecrosis involving 1 or more joints
  • Evidence of retinopathy
  • Priapism
  • Microalbuminuria or evidence of sickle cell nephropathy
  • Alloimmunization Subjects must also meet all of the following general inclusion criteria:
  • Subjects must have a related donor which can consist of Histocompatibility Leukocyte Antigen (HLA)-matched donor up to haploidentical match, mismatched for 1, 2 or 3 HLA-A, B or -DR loci.
  • Subjects must have adequate cardiopulmonary function as documented by echocardiogram or radionuclide scan. (Shortening fraction >26% or ejection fraction >40% or ≥ 80% of normal value for age).
  • Subjects must have adequate pulmonary function documented by Forced expiratory volume in 1 second (FEV1) of ≥ 50% of predicted for age and/or Diffusing capacity of the lung for carbon monoxide (DLCO) (corrected for hemoglobin) ≥50% of predicted for age for patients > 10 years of age.
  • Subjects must have adequate hepatic function as demonstrated by a serum albumin ≥ 3.0 mg/dL, and serum glutamic pyruvic transaminase (SGPT) or serum glutamic oxaloacetic transaminase (SGOT) less than or equal to 5 times the upper limit of normal. Liver biopsy or a liver MRI is necessary if the patient has received chronic transfusions for over a year and/or has a ferritin level of ≥ 1600.
  • Subjects must have adequate renal function as demonstrated by a serum creatinine less than or equal to 2 mg/dL. If serum creatinine is ≥ 2 mg/dL, then a creatinine clearance test or nuclear medicine GFR should document GFR of ≥ 50 ml/min/1.73 m2.
  • Subjects or legal guardians must give written informed consent.
  • Female patients of childbearing potential cannot be pregnant or lactating/breast-feeding and must be either surgically sterile, postmenopausal (no menses for the previous 12 months), or must be practicing an effective method of birth control as determined by the investigator (e.g., oral contraceptives, double barrier methods, hormonal injectable or implanted contraceptives, tubal ligation, or partner with vasectomy).
  • Less than or equal to 45 years of age.
  1. Exclusion Criteria
  • Patients with cirrhosis, extensive bridging hepatic fibrosis, or active hepatitis are excluded from enrollment.
  • Uncontrolled infection or severe concomitant diseases, which in the judgment of the Principal Investigator, indicate that the patient could not tolerate reduced intensity transplantation.
  • Severe impairment of functional performance as evidenced by a Karnofsky score <70% (patients ≥16 years old) or Lansky (children <16 years old) score <70%
  • Renal insufficiency (GFR <50 ml/min/1.73 m2).
  • Subjects with a positive human immunodeficiency virus (HIV) antibody test result.
  • Subjects who are pregnant, as indicated by a positive serum human chorionic gonadotrophin (HCG) test.
  • Subjects whose only donor is pregnant at the time of intended transplant.
  • Subjects of childbearing potential who are not practicing adequate contraception as defined by the investigator at the site.
  • Allogeneic hematopoietic stem cell transplant within the previous 1 year.
  • Subjects must not have had previous radiation therapy that would preclude total body irradiation (TBI) (as determined by a radiation therapist).
  • Jehovah's Witness unwilling to be transfused .
  • Uncontrolled hypersplenism.
  • Severe alloimmunization with inability to guarantee a supply of adequate packed red blood cell (PRBC) donors.
  • Subjects with thalassemia who are Lucarelli Class 3
  • Fanconi anemia.
  • Insufficient funds for the bone marrow processing costs

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Northwestern Memorial Hospital

    Chicago, Illinois, 60611

  • Duke University Medical Center

    Durham, North Carolina, 27705

  • University of Louisville

    Louisville, Kentucky, 40202

Other trials in United States

About this study

The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.

Read the detailed description

This proposal is a phase I/II feasibility study to demonstrate that mixed chimerism can be established with minimal risk in recipients with hemoglobinopathies treated with Campath-1H-based nonmyeloablative conditioning and graft engineering to reduce the risk of Graft Versus Host Disease (GVHD), but preserve engraftment of donor cells.

Being tested
Enriched Hematopoetic Stem Cell Infusion
Keywords
other hemoglobinopathies, chimerism, sickle cell, thalassemia, Marrow/Enriched Hematopoetic Stem Cell Transplant
Medical subject headings
Anemia, Sickle Cell, Thalassemia, Anemia, Diamond-Blackfan, Bone Marrow Failure Disorders, alpha-Thalassemia, beta-Thalassemia

What participants receive

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

  • Enriched Hematopoetic Stem Cell InfusionBiological

    Enriched Hematopoetic Stem Cell Infusion

Study groups in NCT01419704
Study groupRoleWhat they receive
Hemoglobinopathies diagnosed patientsExperimentalRecipients diagnosed with Hemoglobinopathies are treated with an enriched hematopoetic stem cell infusion from living donor bone marrow
Receives: Biological: Enriched Hematopoetic Stem Cell Infusion

What is being measured

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

  1. Proportion of Hemoglobin A and S

    Red blood cell contents by hemoglobin electrophoresis

    Measured: one month to three years

Secondary outcomes (1)

  1. Enriched Hematopoetic Stem Cell Engraftment

    Measured: One month to three years

Timeline

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

  1. First posted18 Aug 2011 (estimated)
  2. Study startMay 2011
  3. Primary completionMay 2016
  4. Study completionMay 2016

Why it stopped: Study completed at site, no active participants.

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01419704
Sponsor's protocol ID
ICT-13881-012011
Status last verified
Mar 2023
First submitted
16 Aug 2011
Last updated
3 Mar 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Enriched Hematopoetic Stem Cell Infusion in people with Anemia, Sickle Cell and Complex and Transfusion-dependent Hemoglobinopathies, run by Talaris Therapeutics Inc. at 3 sites in United States.

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

Who may be eligible to take part?

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: withdrawn, as of the sponsor's last update on 3 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 was withdrawn before enrolling participants, so there are no results.

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?

Proportion of Hemoglobin A and S (time frame: one month to three years). 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 NCT01419704.

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 3 Mar 2023.

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