Source: https://www.pioneeringhealthcare.com/sma/clinical-trial-sma-type-2-3/
Timestamp: 2018-10-15 22:10:37
Document Index: 428319388

Matched Legal Cases: ['art 1', 'art 2', 'art 1', 'art 2', 'art 1', 'art 2', 'art 2', 'art 2', 'art 1', 'art 2', 'art 1', 'art 1', 'art 2', 'art 2', 'art 1', 'art 2']

Clinical trial for SMA Type 2 & 3 - SMA - Pioneering Healthcare
SUNFISH & JEWELFISH trials for SMA Type 2 & 3
What is the purpose of our SUNFISH & JEWELFISH clinical trials?
We aim to learn whether an investigational medicine called risdiplam (also known as RO7034067) is safe and effective in people with Type 2 and Type 3 SMA.
The SUNFISH clinical trial examines our investigational medicine for an age group between 2 to 25 years.
Another clinical trial, JEWELFISH is for people who have previously participated in an SMA patient study with SMN2 targeting therapies RG7800 or nusinersen. For more information about JEWELFISH, click here.
about an SMA trial
The SUNFISH clinical trial
The study doctor conducting the clinical trial must first assess whether a person is eligible to participate in a clinical trial. This assessment is based on specific medical grounds.
The SUNFISH trial has two parts
Part 1 will allow us to determine the most appropriate dose of risdiplam to be further investigated in Part 2. Part 1 includes a small number of people, so it will take place at study sites in a few countries.
Part 2 will assess safety and efficacy of risdiplam at the dose selected in Part 1. As Part 2 includes a much larger number of people, more sites and countries will participate in Part 2.
How many people will be enrolled?
Part 2 will include 168 participants.
Part 1: 12 weeks of treatment followed by an open-label extension phase. In the open-label extension, everyone receives risdiplam.
Part 2: People receiving placebo will be switched to risdiplam after 12 months of treatment. After 24 months of treatment every patient will be offered to receive the study drug in an open label extension phase*.
Yes, the SUNFISH trial is a randomised, double-blinded, placebo controlled trial, so you may be given placebo.
In Part 1 for every 2 people receiving risdiplam, one person will receive placebo, i.e. the ratio is 2:1. Everyone who is receiving placebo in Part 1 will switch to risdiplam for the rest of the clinical trial, after approximately 12 weeks of treatment.
In Part 2, for every 2 people receiving risdiplam, one person will receive placebo i.e. the ratio is 2:1*. Everyone who is receiving placebo in Part 2 will switch to risdiplam for the rest of the clinical trial, after they have completed 12 months of therapy*.
1. Be between 2 and 25 years of age at enrollment
2. Genetically confirmed diagnosis of either SMA Type 2 or Type 3
3. Part 1: Type 2 or 3 SMA ambulant or non-ambulant
Part 2: Type 2 or 3 SMA non-ambulant (can’t walk more than 10 meters (32 feet) without assistance) but able to sit independently with revised upper limb module (RULM) entry item A greater than or equal to 2
4. Able to travel to the study site several times over a 2 year period
6. Have not received gene or cell therapy, SMN2 antisense oligonucleotide therapy or any SMN2
splicing modifier
9. No surgery for scoliosis or hip fixation within 1 year before screening or planned within the next 18 months
10. Have not had an eye disease within the past year
11. Be able to allow eye examinations to be performed
I am interested in joining SUNFISH – what should I do?
Please contact your treating physician to discuss the clinical trial.
SUNFISH ON
Share this link with your physician to
SUNFISH trial for SMA Type 2 & 3
Locate hospitals that are participating in the SUNFISH trial using our Find My Trial tool.
My country is not listed in www.clinicaltrials.gov and is not found using FIND MY TRIAL. How can I take part in SUNFISH?
* The updated study protocol is pending final approval from the regulatory authorities.
View SUNFISH Sites
The study is for people who have previously participated in an SMA patient study with SMN2 targeting therapies RG7800 or nusinersen.
The JEWELFISH clinical trial examines our investigational medicine for an age group between 12 to 60 years of age.
The clinical trial will include approximately 24 patients.
The clinical trial runs for 24 months of treatment followed by 12 months follow up period, all patients will receive risdiplam. There is no placebo in this clinical trial.
1. Be between 12 and 60 years of age at enrollment
2. Be medically diagnosed with either SMA Type 2 or Type 3
3. Have previously participated in a clinical trial with a SMN2 targeting antisense oligonucleotide (e.g. nusinursen) or SMN2 splicing modifier other than risdiplam.
JEWELFISH trial for SMA Type 2 & 3