Stiff-Person SyndromePhase 1/2SPSNCT02282514
Stem Cell Transplantation for Stiff Person Syndrome (SPS)
A Phase 1/2 study testing Autologous Hematopoietic Stem Cells, Cyclophosphamide and Mesna in people with Stiff-Person Syndrome, run by Northwestern University at 1 site in United States.
Trial summary
Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment. When using a non-myeloablative regimen recovery occurs without infusion of stem cells and the stem cells are autologous. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Stiff-Person Syndrome
- What is being tested?
- Autologous Hematopoietic Stem Cells, Cyclophosphamide and Mesna
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2014to 30 Aug 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Diagnosis of Stiff-person Syndrome and
- Age between 18 and 60 years old
- Failure of medically tolerable doses (20-40 mg/day) of diazepam
- Failure of either intravenous immunoglobulin (IVIg) and or plasmapheresis
- Stiffness in the axial muscles, prominently in the abdominal and thoracolumbar paraspinal muscle leading to a fixed deformity (hyperlordosis)
- Superimposed painful spasms precipitated by unexpected noises, emotional stress, tactile stimuli
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- Confirmation of the continuous motor unit activity in agonist and antagonist muscles by electromyography when off diazepam and anti-spasmatic medications
- Absence of neurological or cognitive impairments that could explain the stiffness
- Inability to run or walk, or abnormal gait
- Diagnosis of a SPS variant- Progressive Encephalomyelitis with Rigidity and Myoclonus (PERM) defined as:
- Acute onset of painful rigidity and muscle spasms in the limbs and trunk
- Brainstem dysfunction (nystagmus, opsoclonus, ophthalmoparesis, deafness, dysarthria, dysphagia)
- Profound autonomic disturbance.
- Positive serology for GAD65 (or amphiphysin) autoantibodies, assessed by immunocytochemistry, western blot or radioimmunoassay (>1000 u/ml)
- MRI may show increased signal intensity throughout the spinal cord and the brainstem
- Diagnosis of a SPS variant - anti-GAD positive cerebellar ataxia
- Subacute or chronic onset of cerebellar symptoms-gait or limb ataxia, dysarthria, nystagmus
- Positive serology for GAD65 (or amphiphysin) autoantibodies, assessed by immunocytochemistry, western blot or radioimmunoassay (>1000 u/ml)
- Anti-GAD antibody in cerebrospinal fluid
- Abnormal MRI imaging of brainstem or cerebellum other than cerebellar atrophy
- Negative history of toxin or alcohol
- Absence of Vitamin B12 or Vitamin E deficiency
- Absence of positive HIV, syphilis or whipple disease
- Absence of consanguinity, positive family history for ataxia or positive genetic screen for spinocerebellar ataxia (SCA) 1, SCA 2, SCA 3, SCA 6, SCA 7 or SCA 8 mutation
Who cannot join
- Current or prior history of a malignancy or paraneoplastic syndrome
- Inability to sign and understand consent and be compliant with treatment
- Positive pregnancy test
- Inability to or comprehend irreversible sterility as a possible side effect
- Amphiphysin antibody positive
- Left ventricular ejection fraction (LVEF) < 45% or ischemic coronary artery disease on dobutamine stress echocardiogram
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- Diffusing capacity of the lungs for carbon monoxide (DLCO) < 60% predicted
- Serum creatinine > 2.0 mg/dl
- Bilirubin >2.0 mg/dl
- Platelet count < 100,000 / ul, white blood cell count (WBC) < 1,500 cells/mm3
- History of toxin or alcohol abuse
- History of Vitamin B12 or Vitamin E deficiency
- Positive HIV, syphilis, or whipple disease
- Consanguinity, positive family history for ataxia or positive genetic screen for SCA1, SCA2, SCA3, SCA6, SCA 7 or SCA8 mutation (if ataxia present)
- Absence of at least one SPS associated antibody such as anti-GAD, or gamma-aminobutyric acid (GABA)-A receptor associated protein, or synaptophysin, or gephyrin, or GABA-transaminase
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Non-myeloablative Hematopoietic Stem Cell Transplantation for Stiff Person Syndrome (SPS) and Anti-GAD Antibody Variants: Progressive Encephalomyelitis With Rigidity and Myoclonus (PERM), and Adult Onset Autoimmune Anti-GAD Positive Cerebellar Ataxia
Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment.
When using a non-myeloablative regimen recovery occurs without infusion of stem cells and the stem cells are autologous. While not necessary for recovery, stem cell infusion may shorten the interval of neutropenia and attendant complications. Thus in reality there is no transplant only an autologous supportive blood product.
Based on our encouraging results of non-myeloablative hematopoietic stem cell transplantation, for patients with multiple sclerosis and chronic inflammatory demyelinating polyneuropathy, the investigators will investigate the role of non-myeloablative hematopoietic stem cell transplantation for patients with SPS who require assistance to ambulate.
Read the detailed description
Pre-study Testing
- History and physical
- Electrocardiogram (EKG)
- Dobutamine stress echocardiogram
- High-resolution computed tomography of the chest (HRCT)
- Blood draw for laboratory tests- these tests will include a complete blood count, evaluating liver and kidney function, assessing immune system, tissue typing, and checking for certain germs that can cause infections, including a pregnancy test for females and prostate-specific antigen (PSA) for male as well as testing for HIV
- Pulmonary Function Test (PFT)
- Electromyography (EMG)
- Magnetic Resonance Imaging (MRI) of the Abdomen and Pelvis
- Magnetic Resonance Imaging (MRI) of the Spinal Cord
- Magnetic Resonance Imaging (MRI) of the Brain with Gadolinium (only if PERM of cerebellar ataxia)
- Colonoscopy
- Mammogram (if female)
- Timed ambulation
- Quality of Life Questionnaires [ Short Form (36) Health Survey (SF36) and Barthel Index]
- Chronic Pain Acceptance Questionnaire (CPAQ)
- Rankin Functional Scale
- Modified Ashworth Scale
- Purkinje Cell Cytoplasmic Antibody, Type 1 (PCA-1), Purkinje Cell Cytoplasmic Antibody, Type 2 (PCA-2) antibody (only if cerebellar ataxia)
- Spinocerebellar ataxia (SCA) 1, 2, 3, 4, 5, 6, 7, 8 genes (only if ataxia)
Study Treatment
Stem Cell Collection: Cyclophosphamide 2.0 gm/m2 will be given on day 0, G-CSF 5-10 mcg/kg/day subcutaneous (SQ) will start on day +5 and will continue until apheresis is discontinued. Apheresis will begin when the absolute neutrophil count (ANC) > 1.0 x 109/L and continue until >2.0 x 106 cluster of differentiation 34 (CD34)+ cells/kg patient weight are cryopreserved. A 10-15 liter apheresis will be performed unless stopped earlier for clinical judgment of toxicity (e.g., numbness, tetany). A maximum of four apheresis will be performed.
- Conditions
- Stiff-Person Syndrome
- Being tested
- Autologous Hematopoietic Stem Cells, Cyclophosphamide, Mesna, rATG, Methylprednisolone
- Keywords
- Autoimmune Diseases, Autologous Hematopoietic Stem Cell Transplantation
- Medical subject headings
- Stiff-Person Syndrome, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Hematopoietic Stem CellsBiological
The stem cells will be collected from patient's blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous Hematopoietic Stem Cell Transplantation is to re-infuse immature cells that can re-establish blood production and patient's immune system.
CyclophosphamideDrug
Also called Cytoxan, Neosar, Endoxan
An alkylating agent which causes prevention of cell division by forming adducts with DNA
MesnaDrug
Also called Mesnex
Medication used to decrease the risk of hemorrhagic cystitis prophylaxis
rATGDrug
Also called Thymoglobulin
A predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells
Also called Solu-Medrol
Steroid
G-CSFDrug
Also called Neupogen, Filgrastim, Granix, Zarxio
Granulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream
RituxanDrug
Also called Rituximab
A chimeric monoclonal antibody used in the treatment of B cell non-Hodgkin's lymphoma, B cell leukemia, and some autoimmune disorders
| Study group | Role | What they receive |
|---|---|---|
| Hematopoietic Stem Cell Transplantation | Experimental | The conditioning regimen will be 200 mg/kg of intravenous cyclophosphamide given in 4 equal fractions on days -5 through -2 with intravenous mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused intravenously before each dose of rATG. Autologous hematopoietic stem cells will be infused intravenously on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1. Receives: Biological: Autologous Hematopoietic Stem Cells; Drug: Cyclophosphamide; Drug: Mesna; Drug: rATG; Drug: Methylprednisolone; Drug: G-CSF; Drug: Rituxan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
Number of Participants who Did Not Experience Treatment-Related Mortality
Measured: Mean 3.6 years
Secondary outcomes (2)
Reduction of Muscle Relaxation Anti-spasmatic Medications
Decrease (50%) and complete discontinuation of muscle relaxation anti-spasmatic medications
Measured: Mean 3.6 years
Short-form 36 Quality of Life Questionnaire (SF-36 QOL)
Improvement is defined as a statistically significant change in SF-36 QOL score. The scale is 0-100. The lower the score the worse quality of life.
Measured: mean 3.6 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2014 (estimated)
- Study startOct 2014
- Primary completion19 Aug 2019
- Study completion30 Aug 2019
Why it stopped: Could not predict who would respond, relapse or go into remission
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Stiff-Person SyndromeStudies of the same condition, open and under way.
- More trials run by Northwestern UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Richard Burt, MD, Northwestern University
- Investigators
- Richard Burt, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02282514
- Sponsor's protocol ID
- DIAD.SPS.2014
- Acronym
- SPS
- Status last verified
- Jan 2021
- First submitted
- 30 Oct 2014
- Last updated
- 27 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Burt RK, Balabanov R, Han X, Quigley K, Arnautovic I, Helenowski I, Rose J, Siddique T. Autologous Hematopoietic Stem Cell Transplantation for Stiff-Person Spectrum Disorder: A Clinical Trial. Neurology. 2021 Feb 9;96(6):e817-e830. doi: 10.1212/WNL.0000000000011338. Epub 2020 Dec 14.
PubMed 33318163
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non-myeloablative Hematopoietic Stem Cell Transplantation for Stiff Person Syndrome (SPS) and Anti-GAD Antibody Variants: Progressive Encephalomyelitis With Rigidity and Myoclonus (PERM), and Adult Onset Autoimmune Anti-GAD Positive Cerebellar Ataxia
- ClinicalTrials.gov ID
- NCT02282514
- Acronym / sponsor ID
- SPS / DIAD.SPS.2014
- Conditions
- Stiff-Person Syndrome
- Keywords
- Autoimmune Diseases, Autologous Hematopoietic Stem Cell Transplantation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Could not predict who would respond, relapse or go into remission
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Study start
- Oct 2014
- Primary completion
- 19 Aug 2019 (actual)
- Study completion
- 30 Aug 2019 (actual)
- First posted
- 4 Nov 2014 (estimated)
- Results
- Posted 6 Jan 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment.
When using a non-myeloablative regimen recovery occurs without infusion of stem cells and the stem cells are autologous. While not necessary for recovery, stem cell infusion may shorten the interval of neutropenia and attendant complications. Thus in reality there is no transplant only an autologous supportive blood product.
Based on our encouraging results of non-myeloablative hematopoietic stem cell transplantation, for patients with multiple sclerosis and chronic inflammatory demyelinating polyneuropathy, the investigators will investigate the role of non-myeloablative hematopoietic stem cell transplantation for patients with SPS who require assistance to ambulate.
Detailed description
Pre-study Testing
- History and physical
- Electrocardiogram (EKG)
- Dobutamine stress echocardiogram
- High-resolution computed tomography of the chest (HRCT)
- Blood draw for laboratory tests- these tests will include a complete blood count, evaluating liver and kidney function, assessing immune system, tissue typing, and checking for certain germs that can cause infections, including a pregnancy test for females and prostate-specific antigen (PSA) for male as well as testing for HIV
- Pulmonary Function Test (PFT)
- Electromyography (EMG)
- Magnetic Resonance Imaging (MRI) of the Abdomen and Pelvis
- Magnetic Resonance Imaging (MRI) of the Spinal Cord
- Magnetic Resonance Imaging (MRI) of the Brain with Gadolinium (only if PERM of cerebellar ataxia)
- Colonoscopy
- Mammogram (if female)
- Timed ambulation
- Quality of Life Questionnaires [ Short Form (36) Health Survey (SF36) and Barthel Index]
- Chronic Pain Acceptance Questionnaire (CPAQ)
- Rankin Functional Scale
- Modified Ashworth Scale
- Purkinje Cell Cytoplasmic Antibody, Type 1 (PCA-1), Purkinje Cell Cytoplasmic Antibody, Type 2 (PCA-2) antibody (only if cerebellar ataxia)
- Spinocerebellar ataxia (SCA) 1, 2, 3, 4, 5, 6, 7, 8 genes (only if ataxia)
Study Treatment
Stem Cell Collection: Cyclophosphamide 2.0 gm/m2 will be given on day 0, G-CSF 5-10 mcg/kg/day subcutaneous (SQ) will start on day +5 and will continue until apheresis is discontinued. Apheresis will begin when the absolute neutrophil count (ANC) > 1.0 x 109/L and continue until >2.0 x 106 cluster of differentiation 34 (CD34)+ cells/kg patient weight are cryopreserved. A 10-15 liter apheresis will be performed unless stopped earlier for clinical judgment of toxicity (e.g., numbness, tetany). A maximum of four apheresis will be performed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hematopoietic Stem Cell Transplantation | Experimental | Biological: Autologous Hematopoietic Stem Cells, Drug: Cyclophosphamide, Drug: Mesna, Drug: rATG, Drug: Methylprednisolone, Drug: G-CSF, Drug: RituxanThe conditioning regimen will be 200 mg/kg of intravenous cyclophosphamide given in 4 equal fractions on days -5 through -2 with intravenous mesna. Rabbit antithymocyte globulin (rATG) (Thymoglobulin®) will be dosed at 0.5 mg/kg on day-5, 1.0 mg/kg on days -4 and -3, and then 1.5 mg/kg on days -2 and -1. Methylprednisolone 1000 mg will be infused intravenously before each dose of rATG. Autologous hematopoietic stem cells will be infused intravenously on day 0. A granulocyte-colony stimulating factor (G-CSF) 5-10 mcg/kg will be started on day + 5 and continued until neutrophil engraftment. Intravenous Rituxan (500mg) will be administered on days -6 and +1. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (24 listed)
- Diagnosis of Stiff-person Syndrome and
- Diagnosis of a SPS variant- Progressive Encephalomyelitis with Rigidity and Myoclonus (PERM) defined as:
- Acute onset of painful rigidity and muscle spasms in the limbs and trunk
- Diagnosis of a SPS variant - anti-GAD positive cerebellar ataxia
Key exclusion criteria (15 listed)
- Current or prior history of a malignancy or paraneoplastic syndrome
- Inability to sign and understand consent and be compliant with treatment
- Positive pregnancy test
- Inability to or comprehend irreversible sterility as a possible side effect
- Amphiphysin antibody positive
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Hematopoietic Stem CellsThe stem cells will be collected from patient's blood during mobilization. Then the patient will be given high dose chemotherapy in accordance with approved recommendations for use in conditioning regimens for stem cell transplant in autoimmune diseases. Autologous Hematopoietic Stem Cell Transplantation is to re-infuse immature cells that can re-establish blood production and patient's immune system. | Biological | Hematopoietic Stem Cell Transplantation |
| CyclophosphamideAlso: Cytoxan, Neosar, EndoxanAn alkylating agent which causes prevention of cell division by forming adducts with DNA | Drug | Hematopoietic Stem Cell Transplantation |
| MesnaAlso: MesnexMedication used to decrease the risk of hemorrhagic cystitis prophylaxis | Drug | Hematopoietic Stem Cell Transplantation |
| rATGAlso: ThymoglobulinA predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells | Drug | Hematopoietic Stem Cell Transplantation |
| MethylprednisoloneAlso: Solu-MedrolSteroid | Drug | Hematopoietic Stem Cell Transplantation |
| G-CSFAlso: Neupogen, Filgrastim, Granix, ZarxioGranulocyte-colony stimulating factor; a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream | Drug | Hematopoietic Stem Cell Transplantation |
| RituxanAlso: RituximabA chimeric monoclonal antibody used in the treatment of B cell non-Hodgkin's lymphoma, B cell leukemia, and some autoimmune disorders | Drug | Hematopoietic Stem Cell Transplantation |
Primary outcome
Overall Survival
Time frame: Mean 3.6 years
Number of Participants who Did Not Experience Treatment-Related Mortality
2 secondary outcomes
Reduction of Muscle Relaxation Anti-spasmatic Medications
Time frame: Mean 3.6 years
Short-form 36 Quality of Life Questionnaire (SF-36 QOL)
Time frame: mean 3.6 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 33318163Burt RK, Balabanov R, Han X, Quigley K, Arnautovic I, Helenowski I, Rose J, Siddique T. Autologous Hematopoietic Stem Cell Transplantation for Stiff-Person Spectrum Disorder: A Clinical Trial. Neurology. 2021 Feb 9;96(6):e817-e830. doi: 10.1212/WNL.0000000000011338. Epub 2020 Dec 14.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard Burt, MD principal investigator
Northwestern University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Autologous Hematopoietic Stem Cells, Cyclophosphamide and Mesna in people with Stiff-Person Syndrome, run by Northwestern University 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 24 inclusion and 15 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 27 Jan 2021. 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 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?
Yes. Results are posted in the registry (first posted 6 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Overall Survival (time frame: Mean 3.6 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 NCT02282514, which also lists 1 linked publication.
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 27 Jan 2021.