FIREFISH trial for SMA Type 1

What is the purpose of the FIREFISH clinical trial?

We aim to learn whether an investigational medicine called risdiplam (also known as RO7034067) is safe and effective in babies aged 1-7 months with Type 1 SMA.

The investigational treatment is taken once daily in liquid form by mouth or feeding tube.

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 FIREFISH 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 babies, 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 this part includes a larger number of babies, more sites and countries will take part in Part 2.

How many participants will be enrolled?

Part 1 is completed.
Part 2 will include 40 participants.

How long is treatment given for?

In Part 1: After completing the exploratory dose finding phase, babies will continue to receive risdiplam in an open-label extension phase.
In Part 2: 24 months of treatment (analysis of efficacy is at 12 months of treatment).
There is no placebo in the FIREFISH trial.

What are the eligibility criteria to take part?

1. 1-7 months of age at enrollment
2. Genetically confirmed diagnosis of type 1 SMA
3. Able to travel to the study site several times over a 2 year period
4. Not currently enrolled in another clinical trial
5. Have not received gene or cell therapy, SMN2 antisense oligonucleotide therapy or any
SMN2 splicing modifier
6. Be able to take a daily oral medication either by mouth or by feeding tube
7. Have two survival motor neuron 2 (SMN2) gene copies
8. Body weight greater than or equal to third percentile for age, using appropriate country-
specific guidelines
9. Receive adequate nutrition and hydration (with or without gastrostomy) at the time of screening, in the opinion of the investigator
10. Have not had an eye disease within the past 6 months
11. Not requiring invasive ventilation or tracheostomy
Please refer to for inclusion and exclusion criteria.

I am interested in joining FIREFISH – what should I do?

Please contact your treating physician to discuss the clinical trial.

Your local patient group may also be able to provide you with additional information.
You can also contact us if you need more information

Where is my nearest study centre?

Locate hospitals that are participating in the FIREFISH trial using our Find My Trial tool.

My country is not currently listed in and is not found using FIND MY TRIAL. How can I take part in FIREFISH?

Participating in a trial in another country will be at the discretion of the investigator and is decided on an individual basis.

We can put you in contact with people who can answer your questions about Roche clinical trials in another country.

You can also regularly check back at and Find My Trial for newly opened sites

Who will pay the costs associated with being part of the trial?

Roche will cover the cost of study medication, visits, assessments and reasonable costs of travel.