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Spinal Cord InjuryPhase 1NCT01328860

Autologous Stem Cells for Spinal Cord Injury (SCI) in Children

A Phase 1 study testing bone marrow progenitor cells (BMPC) autologous transplantation in people with Spinal Cord Injury, run by Aryn Knight at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The purpose of this research study is: 1. To see if Bone Marrow Cell harvest and transplantation are safe in children with Spinal Cord Injury, and 2. To determine if late functional outcome is improved following Bone Marrow Cell transplantation in children with Spinal Cord Injury, using pre-transplantation spinal cord… Read more about this study

Who is it for?
Ages 1 year to 15 years
Conditions the study is for
Spinal Cord Injury
What is being tested?
bone marrow progenitor cells (BMPC) autologous transplantation
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
Apr 2011to Jun 2016
Phase
Phase 1
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 1 year to 15 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Between 1 year and 15 years of age on the day of study BMPC infusion.
  • Survived at least six months with PSCI, but are less than 4 years post injury (± 30 days), and have fixed neurologic deficits related to their injury at the time of enrollment.
  • Ability of child to understand and speak English.
  • Ability of child and caregiver to travel to Houston, Texas, and stay for at least 4 days, and to return for all Follow-up visits (patient is responsible for cost of travel and lodging while in Houston).
  • Pediatric patients with any type of spinal cord injury as long as their spinal cords are continuous on MRI evaluation. This includes paraplegic and quadriplegic patients with complete or incomplete spinal cord injuries. This includes patients with ASIA impairment scales from A to D. The clinical classification will be described by the level below which motor/sensory function is impaired, and the degree of that impairment. (i.e. C-5 if the deltoid muscle is intact but the biceps and other muscle groups below the C-5 level are weak [incomplete motor injury] or paralyzed [complete motor injury]. A similar evaluation of sensory function will be established clinically.

Who cannot join

  • Lack of informed consent.
  • Uncorrected coagulopathy during the baseline period defined as: INR > 1.4; PTT > 35 sec; PLT < 100,000.
  • Pre-injury history of seizure disorder and/or neurological impairment where the patient would not be able to participate in age appropriate pain rating scales.
  • A history of prior SCI or severe traumatic brain injury.
  • Known history of:
  • Recently diagnosed infection (within past 2 weeks) requiring treatment and/or medical intervention.
Show all 19Show fewer
  • Renal disease or altered renal function as defined by serum creatinine > 1.5 mg/dL.
  • Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and/or T. Bilirubin > 1.3 mg/dL.
  • Malignancy.
  • Immunosuppression as defined by WBC < 3 (10x3) at screening and/or baseline evaluation lab.
  • HIV.
  • Hepatitis B or C.
  • 8. Unhealed fractures or wounds including osteomyelitis.
  • 9. Pneumonia, or chronic lung disease requiring oxygen.
  • 10. An anatomically discontinuous spinal cord diagnosed by CT or MRI imaging.
  • 11. Positive urine pregnancy test (urine pregnancy test will be routinely performed on females of childbearing potential, age 11 or older.
  • 12. Participation in a concurrent intervention study.
  • 13. Desire for organ-donation in the event of death.
  • 14. Unwillingness or inability to stay for at least four days following BMPC infusion (should any problems arise following the infusion) and to return for a 30 day, and 6 month follow-up visit, and be available for 1 year, and 2 year follow-up phone calls.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Children's Memorial Hemann Hospital; University of Texas Health Science Center - Houston

    Houston, Texas, 77030

Other trials in United States

About this study

Official title: Safety of Autologous Stem Cell Treatment for Spinal Cord Injury in Children

The purpose of this research study is:

  1. To see if Bone Marrow Cell harvest and transplantation are safe in children with Spinal Cord Injury, and
  2. To determine if late functional outcome is improved following Bone Marrow Cell transplantation in children with Spinal Cord Injury, using pre-transplantation spinal cord function as the control.
Read the detailed description

Of the estimated 11,000 cases of acute spinal cord injury (SCI) which occur each year in North America, 5% involve children. The injury is divided into the primary mechanical event which causes the injury, and the secondary events which follow. Outcome for SCI depends of the severity of the primary injury (complete vs. incomplete) and the spinal cord level of the injury. Current therapy is designed only to minimize the secondary events of SCI and other trauma-associated injuries. Because the current therapy does nothing to reverse the primary insult, significant advances in reducing the disability associated with SCI are unlikely. Recent basic science and animal studies suggest that stem cell treatment can foster functional improvement after SCI by helping repair the primary injury and reducing the secondary injury.

Stem cells are "unspecialized" cells in the body that do not have a specific function yet (for example, they have not become "heart cells" or "brain cells" yet.) Stem cells are able to divide and develop into more mature, function-specific cells and take the place of those cells that die, are injured or can no longer function the way they are supposed to. Stem cells are being studied a lot because of this ability and there is the possibility that they may be used to take the place of cells that are no longer working in different parts of the body because of disease (as in cancer, diabetes, and heart disease). Stem cells can be found throughout the body, but they are most common in the bone marrow, the thick, spongy material inside the bones.

The primary objective of this study is to determine the safety of transplantation of the patient's own (autologous) Bone Marrow Progenitor Cells (BMPC) in children with SCI. The secondary objective is to determine if functional, physiological and anatomic outcome measures are improved after BMPC autologous transplantation in children with SCI.

Being tested
bone marrow progenitor cells (BMPC) autologous transplantation
Keywords
Autologous, Stem Cells, Spinal Cord Injury, Children
Medical subject headings
Spinal Cord Injuries

What participants receive

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

  • bone marrow progenitor cells (BMPC) autologous transplantationBiological

    Also called Autologous Stem Cells, Patient's own Stem Cells

    Not more than 5ml/kg bone marrow will be collected under anesthesia; after the bone marrow has been processed, about 6-8 hours after bone marrow aspiration, subjects will receive the cells via intra-venous (in the vein) infusion and will be monitored for 24 hours for any adverse events.

Study groups in NCT01328860
Study groupRoleWhat they receive
Biologic; Stem CellsExperimentalNot described
Receives: Biological: bone marrow progenitor cells (BMPC) autologous transplantation

What is being measured

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

  1. American Spinal Injury Association (ASIA) - Standard Neurological Classification of Spinal Cord Injury.

    ASIA classification assessment will take place pre-procedure, 1 day, 30 days, and 180 days post-procedure to define changes in functional outcome.

    Measured: 180 days

Secondary outcomes (1)

  1. Standard Neuropathic Pain Rating Scale

    Assessment for improvement, worsening or development of neuropathic pain with an age appropriate standard pain rating scale will be performed pre-procedure, and post-procedure days 1 - 14, and again 30 days, 180 days, 1 year, and 2 year post-procedure.

    Measured: 2 years

Timeline

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

  1. First posted5 Apr 2011 (estimated)
  2. Study startApr 2011
  3. Primary completionJun 2014
  4. Study completionJun 2016

Why it stopped: Principal Investigator relocated to Orlando, Florida.

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
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01328860
Sponsor's protocol ID
BBIND14281
Status last verified
Mar 2024
First submitted
1 Apr 2011
Last updated
20 Mar 2024
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing bone marrow progenitor cells (BMPC) autologous transplantation in people with Spinal Cord Injury, run by Aryn Knight at 1 site 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?

The sponsor lists 5 inclusion and 19 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: stopped early, as of the sponsor's last update on 20 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 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.

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 stopped early (reason given: Principal Investigator relocated to Orlando, Florida.). Results are not available in the source record.

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?

American Spinal Injury Association (ASIA) - Standard Neurological Classification of Spinal Cord Injury. (time frame: 180 days). 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 NCT01328860.

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 20 Mar 2024.

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