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Neuromyelitis OpticaPhase 1/2SCT-NMONCT01339455

Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optica

A Phase 1/2 study testing AHSCT in people with Neuromyelitis Optica, run by University of Calgary at 1 site in Canada.

Terminated

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

Official record
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Trial summary

Neuromyelitis Optica (NMO) is a demyelinating and degenerative disorder of the CNS affecting vision and spinal cord function. This disease is rare compared to Multiple Sclerosis (MS), but it is devastating and often leads to accumulating disability with a 5 year-mortality of approximately 30%. Read more about this study

Who is it for?
Ages 18 to 65 years
Conditions the study is for
Neuromyelitis Optica
What is being tested?
AHSCT
Could I receive a placebo?
No, everyone receives an active treatment
How many people
3 participants
Where
1 siteCanada
Study dates
Mar 2011to Mar 2017
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 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age between 18-65, inclusive
  • Diagnosis of NMO using Wingerchuk 2006 NMO Criteria
  • EDSS 0-6.5
  • Treatment with a minimum of one NMO therapy in past 12 months
  • One objective and documented relapse in the past 12 months and two relapse events in the past 24 months despite medical therapy
  • ECOG performance status 0-3
Show all 11Show fewer
  • Platelets ≥100 x 109/L
  • ALT ≤3 x ULN
  • Total bilirubin ≤2.0 x ULN, except in patients with Gilbert syndrome or in patients in whom the bilirubin rise is of non-hepatic origin
  • Serum creatinine <1.5 x ULN or creatinine clearance ≥50 cc/min
  • Patients must reside in Alberta, Canada for the duration of the transplant period of the trial

Who cannot join

  • Any illness that would jeopardize the ability of the patient to complete study protocol
  • Prior malignancy unless non-melanoma skin cancer, carcinoma in-situ of the cervix (CIN) or breast, or malignancy treated more than 5 years previously with no evidence of recurrent disease since initial treatment
  • Pregnant or lactating females. Women of childbearing potential must have a negative serum or urine β-hCG pregnancy test at screening
  • Inability or unwillingness to pursue effective means of birth control
  • FEV1/FVC < 50% of predicted
  • DLCO < 50% of predicted
Show all 13Show fewer
  • Resting LVEF < 50 %
  • Known hypersensitivity to mouse, rabbit, or E. Coli derived proteins, or to iron compounds/medications
  • Presence of metallic objects implanted in the body that would preclude the ability of the patient to safely have MRI exams
  • Unable or unwilling to provide written informed consent for participation
  • Active infection except asymptomatic bacteriuria
  • Any use of investigational therapies within 4 weeks prior to initiation of study treatment
  • Patients dependent on prednisone who cannot be successfully tapered to a maximum of 0.5mg/kg/d prior to mobilization therapy

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • Foothills Medical Centre, University of Calgary

    Calgary, Alberta, T2N 2T9

Other trials in Canada

About this study

Official title: Autologous Hematopoietic Stem Cell Transplant in Patients With Neuromyelitis Optica

Neuromyelitis Optica (NMO) is a demyelinating and degenerative disorder of the CNS affecting vision and spinal cord function. This disease is rare compared to Multiple Sclerosis (MS), but it is devastating and often leads to accumulating disability with a 5 year-mortality of approximately 30%. Survivors are typically left with severe morbidity secondary to blindness, quadriparesis and respiratory failure. No agent has been found to be highly effective in halting disease activity. Based on recent outcomes of stem cell transplant trials and reports in autoimmune diseases including MS, and based on the mechanisms of NMO, we anticipate that stem cell transplantation may provide lasting disease stability for NMO patients. The hypothesis of the present trial is that autologous hematopoetic stem cell transplantation in patients with NMO will provide lasting benefit in relapse prevention. Specifically, we anticipate a 50% reduction in the proportion of patients experiencing relapse over a three year period. We will be following patients for a total of five years after transplantation.

Read the detailed description

Patients who are deemed eligible will be enrolled and undergo a two stage transplant process followed by neurological assessments every 6 months for the following 5 years assessing EDSS, visual metrics, MRI, AQP-4 antibodies, MSFC and SF36.

Being tested
AHSCT
Keywords
Neuromyelitis Optica, NMO, Devic's Disease, Stem Cell Transplant
Medical subject headings
Neuromyelitis Optica

What participants receive

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

  • AHSCTProcedure or surgery

    AHSCT Procedure:

    1. Mobilization and Harvesting:
    • Cyclophosphamide
    • Rituximab
    • GSCF
    • Dexamethasone
    • Apheresis
    1. Conditioning and Infusion (3-4 weeks after Mobilization and Harvesting):
    • Cyclophosphamide
    • MESNA
    • Rabbit ATG
    • Rituximab
    • Methylprednisolone
    • Stem Cell infusion
    • GSCF
Study groups in NCT01339455
Study groupRoleWhat they receive
AHSCTExperimentalAll patients undergo autologous hematopoietic stem cell transplantation in a two stage process.
Receives: Procedure: AHSCT

What is being measured

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

  1. Proportion relapse-free at three years

    The proportion of surviving patients who are relapse-free at three years after transplant

    Measured: 3 years

Secondary outcomes (9)

  1. Proportion relapse-free at five years

    The proportion of surviving patients relapse-free at year five

    Measured: 5 years

  2. Relapse count

    Number of NMO relapse events

    Measured: Annually over 5 years

  3. Disability progression

    Time to progression of EDSS by one step

    Measured: Over 5 years

  4. Retinal nerve fiber layer (RFNL) status

    Change in RNFL by optical coherence tomography over trial

    Measured: 5 years

  5. 25 foot timed walk test

    Change in 25 ft timed walk test over trial

    Measured: 5 years

  6. PASAT

    Annual and change from baseline to end of trial in Paced Auditory Serial Addition Test to assess cognitive function.

    Measured: Annually over 5 years

  7. Hospitalization

    Number of hospitalizations, days in hospital over trial period

    Measured: Over 5 years

  8. Overall survival

    Survival over trial period

    Measured: Over 5 years

  9. Time to next relapse

    Time to next relapse after transplant

    Measured: Over 5 years

Timeline

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

  1. First posted20 Apr 2011 (estimated)
  2. Study startMar 2011
  3. Primary completionMar 2017
  4. Study completionMar 2017

Why it stopped: recruitment failure

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
3 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01339455
Sponsor's protocol ID
CHREB ID# 23282
Acronym
SCT-NMO
Status last verified
Apr 2018
First submitted
18 Apr 2011
Last updated
2 May 2018
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing AHSCT in people with Neuromyelitis Optica, run by University of Calgary at 1 site in Canada.

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 11 inclusion and 13 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 2 May 2018. 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 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 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 stopped early (reason given: recruitment failure). 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?

Proportion relapse-free at three years (time frame: 3 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 NCT01339455.

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 2 May 2018.

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