Phase 3 study to test the effects of SA237 in patients with NMO&NMOSD
Research type
Research Study
Full title
A MULTICENTRE, RANDOMISED, ADDITION TO BASELINE TREATMENT, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE 3 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF SA237 IN PATIENTS WITH NEUROMYELITIS OPTICA (NMO) AND NMO SPECTRUM DISORDER (NMOSD)
IRAS ID
140701
Contact name
Jacqueline Palace
Contact email
Sponsor organisation
Chugai Pharmaceutical Co., Ltd.
Eudract number
2013-003752-21
Clinicaltrials.gov Identifier
Research summary
Summary of Researh
Neuromyelitis optica (NMO) and NMO spectrum disorder (NMOSD) are diseases of the immune system which result in damage to nerve coverings. Both NMO and NMOSD are characterised by relapses (attacks) that can lead to various disabilities such as visual impairment (including blindness) and paralysis of arms and legs.
There are currently no licensed treatments available specifically for the prevention of relapses in NMO/NMOSD. Currently NMO/NMOSD is treated by suppressing the immune system with drugs such as azathioprine, mycophenolate mofetil or corticosteroids, but these drugs are not approved for these diseases and remain unproven.
The study drug SA237 is designed to be more targeted than these drugs and to inhibit specific components of the immune system which are thought to be important in the biology of NMO/NMOSD. The study is being done to test how effective and safe SA237 is in combination with each of the above treatments for NMO and NMOSD.
SA237 is an antibody drug in liquid form that is injected under the skin (subcutaneously) every 2 to 4 weeks. SA237 is a protein that gets attached to a specific target (the interleukin-6 receptor) and prevents it from interacting with the protein (interleukin-6) it normally attaches to. This study will test whether SA237’s ability to block this association will prevent the attacks that occur in NMO/NMOSD.
This is a multicentre randomised, double-blind study which will take place across 7 countries. Approximately 70 patients will be recruited worldwide, of which 35 will receive the study drug on top of their current therapy and 35 patients will receive a placebo (an injection solution containing no drug) on top of their current therapy.
This study is sponsored by Chugai Pharmaceutical Co., Ltd. It is anticipated that participants will remain in the study for up to 2.5 years.Summary of Results
This was a clinical study for satralizumab. Roche, the sponsor of this study and manufacturer of satralizumab, consider it of great importance to share the study results and with interested members of the public. Chugai Pharmaceutical Co., Ltd. a member of the Roche group, designed the study, provided the study drug and placebo, and analysed the data.This is a summary of the final results of the SAkuraSky study.
When and where was the study done, and what has happened since?
The were 83 people who took part in this study. It took about 52 months for the entire study to finish, after starting in February 2014. The study took place at 34 locations in 11 countries: France, Germany, Hungary, Italy, Japan, Poland, Spain, Taiwan, Ukraine, United Kingdom, and the United States. When the study ended in June 2018, Chugai and Roche reviewed all of the data and created a report of the final results.Why was the study needed and who took part?
Neuromyelitis optica spectrum disorder (NMOSD) is a rare disease that affects the spinal cord and the nerves of the eyes (optic nerves). NMOSD is a broad term that includes people who have been diagnosed with neuromyelitis optica (NMO).NMOSD can cause a wide range of symptoms that vary from person to person. People with NMOSD may feel weak, lose vision in one or both eyes, and have nerve pain or muscle spasms. NMOSD also causes some people to feel sick or to have problems with their bladder or bowels. People with NMOSD often have feelings of fatigue (tiredness).
People with NMOSD can have times where their symptoms get much worse. When this happens it is called an ‘attack’. If it happens more than once it is called a ‘relapse’. Attacks and relapses should be avoided as much as possible. This is because they can cause permanent damage to the body. It is very important for doctors to help reduce their patient’s risk of having a relapse. NMOSD is a complicated disease, and researchers are still trying to find out the exact causes. Up until very recently, people with NMOSD had to take medicines that are normally given to patients with other, more common diseases.
A new, experimental drug that could help people with NMOSD has been developed. The new drug is called satralizumab. The purpose of this clinical study was to find out if satralizumab helps to reduce the risk of relapse in people with NMOSD. The study also looked at whether participants had any side effects during the study.
People were examined by a doctor and chosen to be in the study if they:
• Had been diagnosed with NMO or NMOSD.
• Were aged between 12 and 74 years old.
• Had at least two relapses in the last two years.
• Had not changed their NMO or NMOSD medicine in the last eight weeks.What kind of study was this?
This study was a Phase 3, randomized clinical trial. Phase 1 and Phase 2 trials are used to test new drugs on small numbers of people. In Phase 3 trials, new drugs are tested in larger numbers of patients. Satralizumab has already been tested in a small number of people in Phase 1 and Phase 2 trials. This trial tested satralizumab in a larger number of patients with NMOSD.In this study, satralizumab was compared with placebo. Placebo means a treatment that looks the same as the real drug but does not contain any medicine. All patients in the study carried on taking their usual NMO or NMOSD medicine.
The study was randomized. This means that a computer was used to decide who was given the drug and who was given placebo. The decision was completely random, like tossing a coin. Half of the participants were given satralizumab, and the other half were given placebo.
No one was told if their medicine was the real drug or placebo. Also, the doctor who gave patients their medicine did not know if it was the real drug or placebo. This is called a ‘double-blind’ study. This was done to make sure that the study results were not biased in any way.
What happened during the study?
Participants took the treatment until they had a relapse, or until 26 participants in total had relapses.The treatments that participants received were:
Satralizumab 120 mg, as an injection just under the skin:
- Once every two weeks for the first four weeks of the study.
- Once every four weeks for the rest of the study.
Placebo, as an injection just under the skin:
- Once every two weeks for the first four weeks of the study.
- Once every four weeks for the rest of the study.Participants could take their usual NMO or NMOSD medicine during the study.
If doctors thought someone might be having a relapse, they performed a number of medical checks. These checks helped to make sure that other health problems were not being wrongly reported as NMOSD relapses.
What were the overall results of the study?
Participants who took satralizumab were less likely to have a relapse than those who took placebo. Satralizumab did not seem to make participants feel less pain or less tired in normal life. Satralizumab did not cause any serious side effects.The following is a summary of the medical questions asked in the study, and the answers to those questions.
Question 1: Are patients with NMOSD less likely to have a relapse if they take satralizumab as well as their usual medicine?
Yes. Patients who took satralizumab were less likely to have a relapse than patients who took placebo. Eight patients who took satralizumab had a relapse during the study. 18 patients who took placebo had a relapse during the study.
The study was completed as planned, and there were no issues that caused it to finish early.
Question 2: Do patients with NMOSD have less pain or fatigue in their normal life if they take satralizumab?
Overall, participants said that satralizumab did not affect their pain or fatigue.
Pain
Participants were asked to rate their pain at the start of the study. They were then asked to rate their pain again 24 weeks later. People rated their pain by placing a mark on a 100-millimetre line. One end of the line represented ‘no pain’. The other end represented ‘pain as bad as it could be’.In general, participants did not rate their pain differently the second time around. This means that taking satralizumab did not make people feel less pain at the end of 24 weeks.
Fatigue (tiredness)
Overall, satralizumab did not make participants feel less fatigue. People took a fatigue survey at the start of the study. They then took the same survey again 24 weeks later. Each survey was given a score out of 52. Higher scores meant less fatigue. In total, participants did not have changes in how fatigued they felt. This means that taking satralizumab or placebo did not make people feel less fatigue.What side effects did patients have?
Side effects are medical problems that happen during a study. Doctors checked to see if satralizumab affected the number of medical problems participants had.When someone has a health problem during a study, it can be hard to tell exactly what caused it. Sometimes the health problem is a side effect of one of the treatments. Other times the health problem can be caused by a patient’s long-term disease, or by a new illness.
Did any participants have serious side effects?
A side effect is considered ‘serious’ if it is life-threatening, requires hospital care, or causes lasting problems.Seven people (17%) had serious medical problems while taking satralizumab. Nine people (21%) had serious medical problems while taking placebo. Three people taking satralizumab left the study early due to medical problems. Five people taking placebo left the study early due to medical problems.
There were no deaths and no serious allergic reactions.
What were the most common medical problems that participants experienced in this study?
More than 9 out of 10 participants experienced a medical problem at some point during the study. Participants taking placebo had the same number of medical problems as those taking satralizumab. The most common medical problems were common cold, upper respiratory tract infection, headache and urinary tract infection.How has this study helped patients and researchers?
One drawback of this study was that there were not many participants. NMOSD is a rare condition, and finding the right people to take part in studies can be difficult. When there are not many people in a study, it can be harder to assess if something happened by chance or because of the drug.No single clinical study can give a complete understanding of the risks and benefits of a drug, so researchers look at the results of many studies to understand which drugs work and how they work. It take lots of participants in many studies all around the world to advance medical science.
Where can I learn more about this study?
The full title of this study is Efficacy and Safety Study as Add-on Therapy of Satralizumab (SA237) to Treat NMO and NMOSD.You can find more information about this study at:
https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agbRehJ-2Fi4xyo44sEgJVCl5BcbW9wgc64X2JjNxW-2BqMpAMPgCjLWVqTBa9UOWhtRg-2FBw-3D-3Dhetj_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YI5iYdJebGbnHkzndhttffrZ7qRzNBMFZJ9fHo7lRT39qshtVDO9IEPAXEnbhCgJePBwxDlMrokgko2zREjoE-2BPAJazJCl7j2Rm5YeuAJ3cjfnYuqQxM3E5TWsi1lqvCXbhS3U4jMpR6IZ2On71idlZfmWqKtMGJFzK5dd5pi9zEw-3D-3D&data=05%7C01%7Capprovals%40hra.nhs.uk%7Cdae9b6f35f844b0712ed08da84f512f4%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C637968485362723364%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=tYGhbiswaC6RacndKFxqLE%2Bw%2B4xqlkG0o2kUPn%2Bb3S4%3D&reserved=0Trial Identifier: NCT02028884
REC name
South Central - Oxford A Research Ethics Committee
REC reference
14/SC/0170
Date of REC Opinion
19 May 2014
REC opinion
Further Information Favourable Opinion