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A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy (MANATEE)

Primary Purpose

Spinal Muscular Atrophy (SMA)

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
RO7204239
Placebo
Risdiplam
Sponsored by
Hoffmann-La Roche
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinal Muscular Atrophy (SMA)

Eligibility Criteria

2 Years - 25 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Participants who are 2 to 10 years of age inclusive at screening
  • Participants who have a confirmed genetic diagnosis of 5q-autosomal recessive SMA
  • Symptomatic SMA disease, as per investigator's clinical judgement
  • 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 have received previous SMA disease-modifying therapies may be included provided that: Onasemnogene abeparvovec was received at least 90 days prior to screening. Participants should be tapered off steroids prior to receiving risdiplam. In addition, participants should have normal levels of liver function tests, coagulatory parameters, platelets, and troponin-I at 90 days after administration of onasemnogene abeparvovec or at least 1 month after tapering off corticosteroids, whichever comes later; Nusinersen last dose was received at least 90 days prior to screening; Risdiplam is switched to the investigational medicinal product (IMP) provided by the site

Exclusion Criteria:

  • Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening or 5 half-lives of the drug whichever is longer, with the exception of those who have completed a risdiplam study, or participated in a nusinersen or onasemnogene abeparvovec study
  • Receiving or have received previous administration of anti-myostatin therapies
  • For Part 1 participants aged 5 to 10 years only: contraindications for MRI scans including difficulties maintaining a prolonged supine position, or any other clinical history or examination finding that would pose a potential hazard in combination with MRI
  • Any history of cell therapy
  • Hospitalization for a pulmonary event within the last 2 months or planned hospitalization at the time of screening
  • Past surgery for scoliosis or hip fixation in the 6 months preceding screening or planned within the next 9 months (Part 1) or 21 months (Part 2)
  • Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases considered to be clinically significant
  • Clinically significant ECG abnormalities at screening from average of triplicate measurement, abnormal findings at echocardiography, or cardiovascular disease indicating a safety risk for participants at the time of screening
  • Any major illness within 1 month before screening
  • Received any multidrug and toxin extrusion (MATE1/2K) substrates within 2 weeks before screening
  • Hereditary fructose intolerance
  • Used any of the following medications within 90 days prior to screening: riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, probenecid, acetyl cholinesterase inhibitors, agents that could potentially increase or decrease muscle strength, and agents with known or presumed histone deacetylase (HDAC) inhibitory effect
  • Clinically significant abnormalities in laboratory test results at the time of screening
  • Ascertained or presumptive hypersensitivity to RO7204239 or risdiplam, or to the constituents of its formulations
  • Clinically relevant history of anaphylactic reaction requiring inotropic support
  • Any abnormal skin conditions, pigmentation or lesions in the area intended for SC injection (abdomen) and that would prevent visualization of potential injection site reactions to RO7204239
  • Immobilization, surgical procedures, fracture, or trauma to the upper or lower limbs within 90 days prior to screening

Sites / Locations

  • Boston Childrens Hospital
  • Columbia University Medical CenterRecruiting
  • UZ GentRecruiting
  • Chr de La CitadelleRecruiting
  • Clinical Hospital Centre ZagrebRecruiting
  • Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria InfantileRecruiting
  • IRCCS Istituto Giannina Gaslini; U.O.S.D. Centro di Miologia e Patologie NeurodegenerativeRecruiting
  • Fondazione IRCCS Istituto Neurologico ?Carlo Besta?; UO di Neurologia dello SviluppoRecruiting
  • Universitair Medisch Centrum UtrechtRecruiting
  • Uniwersyteckie Centrum Kliniczne; Klinika Neurologii RozwojowejRecruiting
  • Uniwersytecki Szpital Kliniczny w Poznaniu; Od. Kliniczny Neurologii Dzieci i M?odziezyRecruiting
  • Klinika Neurologii I Wydzialu Lekarskiego WUM w WarszawieRecruiting
  • Great Ormond Street Hospital For Children; NeurologyRecruiting
  • John Radcliffe HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

RO7204239 + Risdiplam

Placebo + Risdiplam

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Part 1 - Percentage of participants with adverse events (AEs)
Part 1 - Incidence of relevant echocardiographic parameter z scores > 2
Part 1 - Serum concentration of RO7204239
Part 1 - Time to maximum serum concentration (Cmax) of RO7204239
Part 1 - Area under the curve (AUC) of RO7204239
Part 1 - Trough concentration (Ctrough) of RO7204239
Part 1 - Plasma concentration of risdiplam
Part 1 - Plasma concentration of risdiplam metabolite (M1)
Part 1 - Cmax of risdiplam
Part 1 - AUC of risdiplam
Part 1 - Ctrough of risdiplam
Part 1 - Incidence of anti-drug antibodies (ADAs)
Part 1 - Change from baseline in serum concentration of total myostatin
Part 1 - Change from baseline in serum concentration of free latent myostatin
Part 1 - Change from baseline in serum concentration of mature myostatin
Part 1 - Percent change from baseline in the contractile area of skeletal muscle in the dominant thigh muscles as assessed by magnetic resonance imaging (MRI) in participants aged at least 5 years
Part 1 - Percent change from baseline in the contractile area of skeletal muscle in the dominant calf muscles as assessed by MRI in participants aged at least 5 years
Part 2 - Change from baseline in Revised Hammersmith Scale (RHS) total score

Secondary Outcome Measures

Part 2 - Change from baseline in Motor Function Measure (MFM) Domain 1 + Domain 2 (D1 + D2) score
Part 2 - Change from baseline in MFM-32 total score
Part 2 - Change from baseline in time taken to rise from the floor as measured by RHS Item 25
Part 2 - Change from baseline in time taken to walk/run 10 meters as measured by RHS Item 19
Part 2 - Percent change from baseline in lean mass as assessed by full body dual energy X-ray absorptiometry (DXA) scan in participants aged at least 5 years
Part 2 - Percentage of participants with adverse events (AEs)
Part 2 - Serum concentration of RO7204239
Part 2 - Cmax of RO7204239
Part 2 - AUC of RO7204239
Part 2 - Ctrough of RO7204239
Part 2 - Plasma concentration of risdiplam
Part 2 - Plasma concentration of risdiplam metabolite (M1)
Part 2 - Cmax of risdiplam
Part 2 - AUC of risdiplam
Part 2 - Ctrough of risdiplam
Part 2 - Incidence of ADAs

Full Information

First Posted
November 1, 2021
Last Updated
October 23, 2023
Sponsor
Hoffmann-La Roche
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1. Study Identification

Unique Protocol Identification Number
NCT05115110
Brief Title
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Acronym
MANATEE
Official Title
A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 2, 2022 (Actual)
Primary Completion Date
June 27, 2026 (Anticipated)
Study Completion Date
June 27, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hoffmann-La Roche

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an investigational anti-myostatin antibody that is designed to target myostatin. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. RO7204239 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and clinical outcomes for people living with SMA. This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in patients with spinal muscular atrophy (SMA). The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either ambulant (aged 2-10 years) or non-ambulant (aged 5-10 years) within separate cohorts, and Part 2 is the pivotal part in SMA patients aged 2-25 years that are ambulant.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinal Muscular Atrophy (SMA)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
259 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
RO7204239 + Risdiplam
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo + Risdiplam
Arm Type
Active Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
RO7204239
Intervention Description
RO7204239 will administered every 4 weeks (Q4W) by subcutaneous (SC) injection into the abdomen. RO7204239 will be investigated at low- and high-dose in Part 1.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered Q4W by SC injection into the abdomen.
Intervention Type
Drug
Intervention Name(s)
Risdiplam
Other Intervention Name(s)
RO7034067, Evrysdi
Intervention Description
Risdiplam will be administered orally once daily (QD) for the duration of the study.
Primary Outcome Measure Information:
Title
Part 1 - Percentage of participants with adverse events (AEs)
Time Frame
Up to 4.5 years
Title
Part 1 - Incidence of relevant echocardiographic parameter z scores > 2
Time Frame
Up to 4.5 years
Title
Part 1 - Serum concentration of RO7204239
Time Frame
Through Week 96
Title
Part 1 - Time to maximum serum concentration (Cmax) of RO7204239
Time Frame
Through Week 96
Title
Part 1 - Area under the curve (AUC) of RO7204239
Time Frame
Through Week 96
Title
Part 1 - Trough concentration (Ctrough) of RO7204239
Time Frame
Through Week 96
Title
Part 1 - Plasma concentration of risdiplam
Time Frame
Week 21
Title
Part 1 - Plasma concentration of risdiplam metabolite (M1)
Time Frame
Week 21
Title
Part 1 - Cmax of risdiplam
Time Frame
Week 21
Title
Part 1 - AUC of risdiplam
Time Frame
Week 21
Title
Part 1 - Ctrough of risdiplam
Time Frame
Week 21
Title
Part 1 - Incidence of anti-drug antibodies (ADAs)
Time Frame
Through Week 96
Title
Part 1 - Change from baseline in serum concentration of total myostatin
Time Frame
Through Week 85
Title
Part 1 - Change from baseline in serum concentration of free latent myostatin
Time Frame
Through Week 85
Title
Part 1 - Change from baseline in serum concentration of mature myostatin
Time Frame
Through Week 85
Title
Part 1 - Percent change from baseline in the contractile area of skeletal muscle in the dominant thigh muscles as assessed by magnetic resonance imaging (MRI) in participants aged at least 5 years
Time Frame
Week 24 of combination treatment
Title
Part 1 - Percent change from baseline in the contractile area of skeletal muscle in the dominant calf muscles as assessed by MRI in participants aged at least 5 years
Time Frame
Week 24 of combination treatment
Title
Part 2 - Change from baseline in Revised Hammersmith Scale (RHS) total score
Time Frame
Week 72 of combination treatment (study Week 80)
Secondary Outcome Measure Information:
Title
Part 2 - Change from baseline in Motor Function Measure (MFM) Domain 1 + Domain 2 (D1 + D2) score
Time Frame
Week 72 of combination treatment (study Week 80)
Title
Part 2 - Change from baseline in MFM-32 total score
Time Frame
Week 72 of combination treatment (study Week 80)
Title
Part 2 - Change from baseline in time taken to rise from the floor as measured by RHS Item 25
Time Frame
Week 72 of combination treatment (study Week 80)
Title
Part 2 - Change from baseline in time taken to walk/run 10 meters as measured by RHS Item 19
Time Frame
Week 72 of combination treatment (study Week 80)
Title
Part 2 - Percent change from baseline in lean mass as assessed by full body dual energy X-ray absorptiometry (DXA) scan in participants aged at least 5 years
Time Frame
Week 72 of combination treatment (study Week 80)
Title
Part 2 - Percentage of participants with adverse events (AEs)
Time Frame
Up to 4.5 years
Title
Part 2 - Serum concentration of RO7204239
Time Frame
Through Week 80
Title
Part 2 - Cmax of RO7204239
Time Frame
Through Week 80
Title
Part 2 - AUC of RO7204239
Time Frame
Through Week 80
Title
Part 2 - Ctrough of RO7204239
Time Frame
Through Week 80
Title
Part 2 - Plasma concentration of risdiplam
Time Frame
Week 32
Title
Part 2 - Plasma concentration of risdiplam metabolite (M1)
Time Frame
Week 32
Title
Part 2 - Cmax of risdiplam
Time Frame
Week 32
Title
Part 2 - AUC of risdiplam
Time Frame
Week 32
Title
Part 2 - Ctrough of risdiplam
Time Frame
Week 32
Title
Part 2 - Incidence of ADAs
Time Frame
Through Week 80

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age at screening: Part 1 Cohorts A (ambulant participants), B (ambulant participants), and D (non-ambulant participants): 5-10 years, inclusive; Part 1 Cohort C (ambulant participants): 2-4 years, inclusive; Part 2 (ambulant participants): 2-25 years, inclusive Participants who have a confirmed genetic diagnosis of 5q-autosomal recessive SMA Symptomatic SMA disease, as per investigator's clinical judgement Participants who have received previous SMA disease-modifying therapies may be included provided that: Onasemnogene abeparvovec was received at least 90 days prior to screening. Participants should be tapered off steroids prior to receiving risdiplam. In addition, participants should have normal levels of liver function tests, coagulatory parameters, platelets, and troponin-I at 90 days after administration of onasemnogene abeparvovec or at least 1 month after tapering off corticosteroids, whichever comes later; Nusinersen last dose was received at least 90 days prior to screening; Risdiplam is switched to the investigational medicinal product (IMP) provided by the site Inclusion Criteria for Part 1 Cohorts A, B, and C and Part 2 only: 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 seconds as measures by the Timed 10-Meter Walk/Run Test [10MWRT] at screening Inclusion Criteria for Part 1 Cohort D only: Participants who are able to sit, defined by: A score of 3 on Item 9 of the MFM32 (sitting without upper limb support while maintaining contact between the two hands for 5 seconds); A score of at least 2 on Item 10 of the MFM32 (while seated, leaning forward to touch a tennis ball and sitting back again, either with or without upper limb support) Participants who are able to raise a standardized plastic cup with a 200g weight in it to the mouth, using both hands if necessary, defined by a score of 3 on the entry item of the Revised Upper Limb Module (RULM) Exclusion Criteria: Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening or 5 half-lives of the drug whichever is longer, with the exception of those who have completed a risdiplam study, or participated in a nusinersen or onasemnogene abeparvovec study Receiving or have received previous administration of anti-myostatin therapies Any history of cell therapy Hospitalization for a pulmonary event within the last 2 months or planned hospitalization at the time of screening Past surgery for scoliosis or hip fixation in the 6 months preceding screening or planned within the next 9 months (Part 1) or 21 months (Part 2) Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases considered to be clinically significant Clinically significant ECG abnormalities at screening from average of triplicate measurement, abnormal findings at echocardiography, or cardiovascular disease indicating a safety risk for participants at the time of screening Any major illness within 1 month before screening Received any multidrug and toxin extrusion (MATE1/2K) substrates within 2 weeks before screening Hereditary fructose intolerance Used any of the following medications within 90 days prior to screening: riluzole, valproic acid, hydroxyurea, sodium phenylbutyrate, butyrate derivatives, creatine, carnitine, growth hormone, anabolic steroids, probenecid, acetyl cholinesterase inhibitors, agents that could potentially increase or decrease muscle strength, and agents with known or presumed histone deacetylase (HDAC) inhibitory effect Clinically significant abnormalities in laboratory test results at the time of screening Ascertained or presumptive hypersensitivity to RO7204239 or risdiplam, or to the constituents of its formulations Clinically relevant history of anaphylactic reaction requiring inotropic support Any abnormal skin conditions, pigmentation or lesions in the area intended for SC injection (abdomen) and that would prevent visualization of potential injection site reactions to RO7204239 Immobilization, surgical procedures, fracture, or trauma to the upper or lower limbs within 90 days prior to screening Exclusion Criteria for Part 1 Cohorts A and B only: Participants with contraindications for MRI scan (including, but not restricted to, claustrophobia, pacemaker, artificial heart valves, cochlear implants, presence of foreign metal objects in heart or body, including spinal rods, intracranial vascular clips, insulin pumps, etc.), difficulties maintaining a prolonged supine position, or any other clinical history or examination finding that would pose a potential hazard in combination with MRI Exclusion Criteria for Part 1 Cohort D only: Participants who are unable to adopt the correct position to endure adequate quality of DXA scan acquisition, as determined by the DXA scan technologist Participants who have contractures at screening that would interfere with DXA scan acquisition or functional assessments, as confirmed by the DXA scan technologist and clinical evaluator For participants able to take steps only: Able to walk 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 seconds as measured by the timed 10MWRT at screening Participants who have severe scoliosis (curvature > 40°) at screening based on the participant's most recent X-ray as performed per standard of care or scoliosis that would interfere with functional assessments, as confirmed by the clinical evaluator. An X-ray is not required if it is not clinically indicated (e.g., in participants with mild scoliosis) Participants who require invasive ventilation, tracheostomy, or the use of noninvasive ventilation (e.g., bilevel positive airway pressure) during the daytime
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Reference Study ID Number: BN42644 https://forpatients.roche.com/
Phone
888-662-6728
Email
global-roche-genentech-trials@gene.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Hoffmann-La Roche
Official's Role
Study Director
Facility Information:
Facility Name
Boston Childrens Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Name
UZ Gent
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Individual Site Status
Recruiting
Facility Name
Chr de La Citadelle
City
Liège
ZIP/Postal Code
4000
Country
Belgium
Individual Site Status
Recruiting
Facility Name
Clinical Hospital Centre Zagreb
City
Zagreb
ZIP/Postal Code
10000
Country
Croatia
Individual Site Status
Recruiting
Facility Name
Policlinico Agostino Gemelli; Dipartimento di Neuropsichiatria Infantile
City
Roma
State/Province
Lazio
ZIP/Postal Code
00168
Country
Italy
Individual Site Status
Recruiting
Facility Name
IRCCS Istituto Giannina Gaslini; U.O.S.D. Centro di Miologia e Patologie Neurodegenerative
City
Genova
State/Province
Liguria
ZIP/Postal Code
16147
Country
Italy
Individual Site Status
Recruiting
Facility Name
Fondazione IRCCS Istituto Neurologico ?Carlo Besta?; UO di Neurologia dello Sviluppo
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20133
Country
Italy
Individual Site Status
Recruiting
Facility Name
Universitair Medisch Centrum Utrecht
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Uniwersyteckie Centrum Kliniczne; Klinika Neurologii Rozwojowej
City
Gda?sk
ZIP/Postal Code
80-952
Country
Poland
Individual Site Status
Recruiting
Facility Name
Uniwersytecki Szpital Kliniczny w Poznaniu; Od. Kliniczny Neurologii Dzieci i M?odziezy
City
Pozna?
ZIP/Postal Code
60-355
Country
Poland
Individual Site Status
Recruiting
Facility Name
Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie
City
Warszawa
ZIP/Postal Code
02-097
Country
Poland
Individual Site Status
Recruiting
Facility Name
Great Ormond Street Hospital For Children; Neurology
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
John Radcliffe Hospital
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).

Learn more about this trial

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

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