Retinal DiseasePhase 1NCT00278486
Hematopoietic Stem Cell Transplantation in Autoimmune-Related Retinopathy(ARRON)
A Phase 1 study testing Hematopoietic stem cell transplantation. in people with Retinal Disease, run by Richard Burt, MD at 1 site in United States.
Trial summary
ARRON is a disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the tissue in the retina and/or optic nerve. In addition, the disease may affect the nerves in the ear or other parts of the body . Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Retinal Disease
- What is being tested?
- Hematopoietic stem cell transplantation.
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2004to Apr 2012
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Age between 18-60.
- Diagnosis of ARRON syndrome. Diagnostic criteria described below.
- Unexplained visual loss over weeks to months. The visual loss includes both visual acuity and field loss define as follows:
- Visual acuity: 20/40 or less
- OR
- Visual field: perimetric mean deviation -5b
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- Positive antibody to retina or optic nerve.
- OR
- A response to immunosuppressive drugs or immune modulators (response is defined by improvement of vision or decrease the rate of decline of visual loss).
- Absence of malignancy {negative physical examination, gastrointestinal endoscopies, mammography and gynecologic examination (for female), and serum PSA measurement (for male) within a year}.
- Negative MRI of brain.
- The patient has failed at least 3 months of corticosteroids (prednisone 0.5mg/kg to start), IVIG and at least one other immunosuppressive drug such as methotrexate, Imuran, cyclosporine, etc. Failure is defined by decline of visual acuity (by standard Snellen acuity clinical testing) or visual field (by Humphrey Automated Machine with the 30-2 program or using Kinetic Visual Fields on the Goldman Perimeter)
Who cannot join
- Absence of light perception lasting more than 6 months
- Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
- Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis.
- Positive pregnancy test.
- Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an IUD (intrauterine device); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam.
- Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
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- FEV1/FVC < 60% of predicted after bronchodilator therapy (if necessary).
- DLCO < 50% of predicted.
- Active ischemic heart disease and/or those who have had a myocardial infarction within 6 months.
- Resting LVEF < 40 %.
- Bilirubin > 2.0 mg/dl
- Serum creatinine > 2.0 mg/dl.
- 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.
- Diagnosis of primary progressive MS.
- Platelet count < 100,00/ul
- Psychiatric illness, mental deficiency or cognitive dysfunction making compliance with treatment or informed consent impossible.
- Active infection except asymptomatic bacteruria.
Trial locations and contacts
1 site in 1 country.
United States1 site
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, 60611
About this study
Official title: Immune Ablation and Hematopoietic Stem Cell Transplantation in Patients With Autoimmune-Related Retinopathy and Optic Neuropathy (ARRON) Syndrome (Not Associated With Cancer)
ARRON is a disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the tissue in the retina and/or optic nerve. In addition, the disease may affect the nerves in the ear or other parts of the body . The affected nerves fail to respond, or respond only weakly, to stimuli causing numbing, tingling, pain, and progressive muscle weakness. If the nerves to the ear are affected, reduced hearing or deafness may result. The likelihood of progression of your disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide and rabbit ATG (drugs which reduce the function of the immune system) followed by return of previously collected blood stem cells will stop the progression of ARRON syndrome. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the cyclophosphamide and rabbit ATG is to destroy the cells in the immune system which are thought to be causing this disease. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and to produce a normal immune system that will no longer attack the body.
- Conditions
- Retinal Disease
- Being tested
- Hematopoietic stem cell transplantation.
- Medical subject headings
- Retinal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoietic stem cell transplantation.Biological
Autologous hematopoietic stem cell transplantation will be performed.
| Study group | Role | What they receive |
|---|---|---|
| stem cell transplantation | Experimental | Not described Receives: Biological: Hematopoietic stem cell transplantation. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Standard Snellen acuity clinical testing and improvement visual fields is done by using Humphrey Automated Machine with 30-2 program or using Kinetic Visual Fields on the Goldman Perimeter)
Measured: 5 years after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2006 (estimated)
- Study startAug 2004
- Primary completionApr 2012
- Study completionApr 2012
Why it stopped: First subject over five years after the transplant. The second subject is not in compliance with follow ups. We do not plan to enroll more subjects.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinal DiseaseStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Richard Burt, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Richard Burt, MD, Northwestern University
- Investigators
- Richard Burt, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00278486
- Sponsor's protocol ID
- DI ARRON 04
- Status last verified
- Apr 2013
- First submitted
- 15 Jan 2006
- Last updated
- 8 Apr 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Immune Ablation and Hematopoietic Stem Cell Transplantation in Patients With Autoimmune-Related Retinopathy and Optic Neuropathy (ARRON) Syndrome (Not Associated With Cancer)
- ClinicalTrials.gov ID
- NCT00278486
- Acronym / sponsor ID
- DI ARRON 04
- Conditions
- Retinal Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: First subject over five years after the transplant. The second subject is not in compliance with follow ups. We do not plan to enroll more subjects.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Richard Burt, MDUniversity, hospital or other
- Study start
- Aug 2004
- Primary completion
- Apr 2012 (actual)
- Study completion
- Apr 2012 (actual)
- First posted
- 18 Jan 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
ARRON is a disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the tissue in the retina and/or optic nerve. In addition, the disease may affect the nerves in the ear or other parts of the body . The affected nerves fail to respond, or respond only weakly, to stimuli causing numbing, tingling, pain, and progressive muscle weakness. If the nerves to the ear are affected, reduced hearing or deafness may result. The likelihood of progression of your disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide and rabbit ATG (drugs which reduce the function of the immune system) followed by return of previously collected blood stem cells will stop the progression of ARRON syndrome. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the cyclophosphamide and rabbit ATG is to destroy the cells in the immune system which are thought to be causing this disease. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and to produce a normal immune system that will no longer attack the body.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| stem cell transplantation | Experimental | Biological: Hematopoietic stem cell transplantation. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (12 listed)
- Age between 18-60.
- Diagnosis of ARRON syndrome. Diagnostic criteria described below.
- Unexplained visual loss over weeks to months. The visual loss includes both visual acuity and field loss define as follows:
- OR
- OR
Key exclusion criteria (18 listed)
- Absence of light perception lasting more than 6 months
- Any illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive chemotherapy.
- Prior history of malignancy except localized basal cell, squamous skin cancer or carcinoma in situ of the cervix. Other malignancies for which the patient is judged to be cured, such as head and neck cancer, or breast cancer will be considered on an individual basis.
- Positive pregnancy test.
- Inability or unwillingness to pursue effective means of birth control. Effective birth control is defined as 1) refraining from all acts of vaginal intercourse (ABSTINENCE); 2) consistent use of birth control pills; 3) injectable birth control methods (Depo-provera, Norplant); 4) tubal sterilization or male partner who has undergone vasectomy; 5) placement of an IUD (intrauterine device); or 6) use, with every act of intercourse, of diaphragm with contraceptive jelly and/or condoms with contraceptive foam.
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 |
|---|---|---|
| Hematopoietic stem cell transplantation.Autologous hematopoietic stem cell transplantation will be performed. | Biological | stem cell transplantation |
Primary outcome
Standard Snellen acuity clinical testing and improvement visual fields is done by using Humphrey Automated Machine with 30-2 program or using Kinetic Visual Fields on the Goldman Perimeter)
Time frame: 5 years after transplant
Results and safety
The study was stopped early (reason given: First subject over five years after the transplant. The second subject is not in compliance with follow ups. We do not plan to enroll more subjects.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2013.
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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 study testing Hematopoietic stem cell transplantation. in people with Retinal Disease, run by Richard Burt, MD 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 12 inclusion and 18 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 8 Apr 2013. 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: First subject over five years after the transplant. The second subject is not in compliance with follow ups. We do not plan to enroll more subjects.). 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?
Standard Snellen acuity clinical testing and improvement visual fields is done by using Humphrey Automated Machine with 30-2 program or using Kinetic Visual Fields on the Goldman Perimeter) (time frame: 5 years after transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Apr 2013.