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Small Molecule
RO7204239 + Risdiplam for Spinal Muscular Atrophy (MANATEE Trial)
Phase 2 & 3
Recruiting
Research Sponsored by Hoffmann-La Roche
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Participants who are ambulant, where ambulant is defined as able to walk/run unassisted (i.e., without the use of assistive devices such as canes, walking sticks, crutches, walkers, person/hand-held assistance, braces, orthoses, over the malleoli insoles or any other type of support) 10 meters in </= 30 as measured by the Timed 10-Meter Walk/Run Test [10MWRT] seconds at screening
Participants who are 2 to 10 years of age inclusive at screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 4.5 years
Awards & highlights
MANATEE Trial Summary
This trial is testing a new drug combination to see if it is safe and effective in improving muscle function in children with Spinal Muscular Dystrophy.
Who is the study for?
This trial is for individuals aged 2-25 with confirmed genetic diagnosis of 5q-autosomal recessive Spinal Muscular Atrophy (SMA). Participants must be able to walk or sit unassisted, as applicable, and have not had certain treatments or surgeries recently. They should not have severe heart issues, skin conditions at injection sites, major recent illnesses, or require daytime ventilation.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of RO7204239 combined with Risdiplam in SMA patients. Part 1 determines the proper dose among ambulant/non-ambulant children; Part 2 assesses its impact on a broader age range. The goal is to improve muscle function by increasing SMN protein production and inhibiting myostatin.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include reactions at the injection site due to RO7204239 and general drug-related risks such as allergic reactions or impacts on liver function which will be monitored throughout the trial.
MANATEE Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can walk or run 10 meters in 30 seconds or less without help.
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I am between 2 and 10 years old.
Select...
I have a genetic diagnosis of 5q-SMA.
Select...
I have a genetic diagnosis of 5q-SMA.
Select...
I am between 2 and 25 years old, depending on the study part.
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I can walk or run 10 meters unassisted in less than 30 seconds.
MANATEE Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 4.5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 4.5 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Area under the curve (AUC) of RO7204239
Change from baseline in Revised Hammersmith Scale (RHS) total score
Change from baseline in serum concentration of free latent myostatin
+8 moreSecondary outcome measures
AUC of RO7204239
AUC of risdiplam
Change from baseline in MFM-32 total score
+13 moreSide effects data
From 2023 Phase 2 trial • 231 Patients • NCT0290868522%
Nasopharyngitis
15%
Upper respiratory tract infection
13%
Vomiting
13%
Pyrexia
10%
Headache
10%
Cough
9%
Diarrhoea
8%
Gastroenteritis
7%
Pneumonia
6%
Respiratory tract infection
6%
Abdominal pain
5%
Bronchitis
5%
Pharyngitis
5%
Rash
4%
Ear pain
3%
Influenza like illness
3%
Ear infection
3%
Influenza
3%
Constipation
3%
Nausea
3%
Limb injury
3%
Arthralgia
3%
Back pain
3%
Oropharyngeal pain
3%
Rhinorrhoea
3%
Eczema
2%
Contusion
2%
Gastrointestinal infection
2%
Urinary tract infection
2%
Gastritis
2%
Sinusitis
2%
Arthropod bite
2%
Decreased appetite
2%
Pain in extremity
2%
Dry skin
2%
Pruritus
2%
Erythema
1%
Herpes zoster
1%
Pneumonia mycoplasmal
1%
Post procedural infection
1%
Dehydration
1%
Asthenia
1%
Hypersensitivity
1%
Conjunctivitis
1%
Cystitis
1%
Groin infection
1%
Rhinitis
1%
Scarlet fever
1%
Tonsillitis
1%
Device related infection
1%
Encephalitis
1%
Infective thrombosis
1%
Pyelonephritis
1%
Viral upper respiratory tract infection
1%
Brain contusion
1%
Partial seizures
1%
Haematuria
1%
Nephrolithiasis
1%
Atelectasis
1%
Pneumonitis aspiration
1%
Pneumothorax
1%
Tachycardia
1%
Ocular hyperaemia
1%
Abdominal pain upper
1%
Aphthous ulcer
1%
Neck pain
1%
Dizziness
1%
Amenorrhoea
1%
Dysmenorrhoea
1%
Nasal congestion
1%
Rhinitis allergic
1%
Asthma
1%
Epistaxis
1%
Productive cough
1%
Acne
1%
Alopecia
1%
Blister
1%
Dermatitis
1%
Seborrhoeic dermatitis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part 2 OLT: Risdiplam/Risdiplam
Part 2 OLT: Placebo/Risdiplam
Part 2 OLE: Risdiplam
Part 1 Group B: Children (0.25 mg/kg Risdiplam)
Part 1 Group A: Adolescents and Adults (3 mg Risdiplam)
Part 1 Group A: Adolescents and Adults (5 mg Risdiplam)
Part 1 Group B: Children (Placebo-Control Period Pooled)
Part 1 Group A: Adolescents and Adults (Placebo-Control Period Pooled)
Part 1 Group B: Children (0.02 mg/kg Risdiplam)
Part 1 Group B: Children (0.05 mg/kg Risdiplam)
Part 1 Group B: Children (0.15 mg/kg Risdiplam)
Part 1 Group A: OLE
Part 1 Group B: OLE
Part 2 Placebo-Controlled: Risdiplam
Part 2 Placebo-Controlled: Placebo
MANATEE Trial Design
2Treatment groups
Experimental Treatment
Active Control
Group I: RO7204239 + RisdiplamExperimental Treatment2 Interventions
Participants who have not previously been treated with risdiplam will receive risdiplam for at least 8 weeks prior to randomization into a treatment group (Part 1 only). Participants that have been treated with risdiplam for at least 8 continuous weeks immediately prior to joining the study may be immediately randomized to combination therapy, or join the study run-in period (the period between screening and randomization to a treatment group) where they will continue to receive risdiplam monotherapy until randomization.
Participants enrolled in Part 1 will receive RO7204239 (low or high dose) + risdiplam for 24 weeks, followed by RO7204239 + risdiplam for 72 weeks.
Participants enrolled in Part 2 will receive risdiplam for 8 weeks and then treatment with RO7204239 + risdiplam for 72 weeks.
Once the treatment period has completed (Part 1 or Part 2), participants will have the option of treatment with RO7204239 + risdiplam for 2 additional years.
Group II: Placebo + RisdiplamActive Control2 Interventions
Participants who have not previously been treated with risdiplam will receive risdiplam for at least 8 weeks prior to randomization into a treatment group (Part 1 only). Participants that have been treated with risdiplam for at least 8 continuous weeks immediately prior to joining the study may be immediately randomized to combination therapy, or join the study run-in period (the period between screening and randomization to a treatment group) where they will continue to receive risdiplam monotherapy until randomization.
Participants enrolled in Part 1 will receive placebo (low or high dose-matched) + risdiplam for 24 weeks, followed by RO7204239 + risdiplam for 72 weeks.
Participants enrolled in Part 2 will receive risdiplam for 8 weeks and then treatment with placebo + risdiplam for 72 weeks.
Once the treatment period has completed (Part 1 or Part 2), participants will have the option of treatment with RO7204239 + risdiplam for 2 additional years.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Risdiplam
2016
Completed Phase 2
~420
Find a Location
Who is running the clinical trial?
Hoffmann-La RocheLead Sponsor
2,432 Previous Clinical Trials
1,089,964 Total Patients Enrolled
Clinical TrialsStudy DirectorHoffmann-La Roche
2,201 Previous Clinical Trials
888,681 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have not taken any drugs that are cleared by the kidneys in the last 2 weeks.I have not taken specific medications in the last 90 days.I have hereditary fructose intolerance.You have a known allergy to RO7204239 or risdiplam, or any ingredients in the medication.I can walk or run 10 meters in 30 seconds or less without help.I have a history of cancer.I am between 2 and 10 years old.I do not have any major issues with my digestive, kidney, liver, hormone, or heart systems.I have a genetic diagnosis of 5q-SMA.I have been treated with anti-myostatin therapies.I haven't had scoliosis or hip surgery in the last 6 months and don't plan to in the next 9 or 21 months.I have SMA and have stopped certain treatments for at least 90 days, with normal health checks.I need a machine to help me breathe during the day.I've had SMA treatments but meet all the specific timing and health requirements for this trial.I have had cell therapy in the past.I have a genetic diagnosis of 5q-SMA.You have had a severe allergic reaction that needed strong medication to support your heart.I have had a condition where my lymphocytes multiply unusually.I have not had a major illness in the last month.I have not had any surgery, broken bones, or serious injuries to my arms or legs in the last 3 months.I am between 2 and 25 years old, depending on the study part.Your lab tests show important health problems at the time of screening.I haven't had any cancer except for skin cancer in the last 5 years.You do not live in the United States.I do not have serious heart issues that could make the trial unsafe for me.I have no skin issues on my abdomen that would affect injection site monitoring.You have a severe curvature in your spine or scoliosis that would make it hard to do the tests in the study.I was hospitalized for a lung problem in the last 2 months or expect to be soon.I can walk or run 10 meters unassisted in less than 30 seconds.I can sit up as required by the study's specific criteria.My doctor says my spinal muscular atrophy symptoms are noticeable.I do not have body stiffness that would affect body scans or movement tests.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo + Risdiplam
- Group 2: RO7204239 + Risdiplam
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Who else is applying?
How old are they?
< 18
18 - 65
What site did they apply to?
Columbia University Medical Center
Uniwersytecki Szpital Kliniczny w Poznaniu; Od. Kliniczny Neurologii Dzieci i M?odziezy
Boston Childrens Hospital
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
What state do they live in?
California
Why did patients apply to this trial?
I am fascinated by turtles. I'm hoping that the myostatin inhibitor can enhance my muscle strength in addition to my existing Risdiplam treatment.
PatientReceived no prior treatments
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