Clinical trial • Phase III • Neurology|Rare Disease

Risdiplam for Spinal muscular atrophy

Phase III trial of Risdiplam for Spinal muscular atrophy.

Overview

Trial Therapeutic Area
Neurology|Rare Disease
Trial Disease
Spinal muscular atrophy
Trial Stage
Phase III
Drug Modality
Monoclonal antibody|Small molecule
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
23-07-2024
First CTIS Authorization Date
26-08-2024

Trial design

Randomised, placebo in combination with risdiplam (ro7034067) — dose and schedule not specified in the public record-controlled Phase III trial in Belgium, Croatia, Italy and others.

Randomised
Yes
Comparator
Placebo in combination with risdiplam (RO7034067) — dose and schedule not specified in the public record
Target Sample Size
42
Trial Duration For Participant
560

Eligibility

Recruits 42 paediatric patients.

Vulnerable Population
The trial explicitly includes vulnerable populations (children and adolescents). Multiple assent forms and age‑specific ICFs are provided (assent forms for ages 3-6, 7-11, 12-17; caregiver and parent/legal representative ICFs). Consent is obtained from parents/legal representatives for minors; age‑appropriate assent is requested from participating children per the available assent documents. Additional caregiver information and optional addenda (e.g., video recording) are available.

Inclusion criteria

  • {"criterion_text":"- 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\n- Participants who have a confirmed genetic diagnosis of 5q-autosomal recessive SMA\n- 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\n- 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\n- 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)\n- Inclusion Criteria for Part 1 Cohort D only: 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

  • {"criterion_text":"- 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\n- Receiving or have received previous administration of anti-myostatin therapies\n- Exclusion Criteria for Part 1 Cohort D only: 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)\n- 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\n- Exclusion Criteria for Part 1 Cohort D only: Participants who are unable to adopt the correct position to ensure adequate quality of DXA scan acquisition, as determined by the DXA scan technologist\n- Exclusion Criteria for Part 1 Cohort D only: 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"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 1. Incidence, severity, and causal relationship of adverse events (AEs), with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v5.0)","definition_or_measurement_approach":"Incidence, severity and causal relationship assessed; severity determined according to NCI CTCAE v5.0"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 2. Change from baseline in vital signs, physical findings, ECG, echocardiogram, relevant echocardiographic parameters, and clinical laboratory results","definition_or_measurement_approach":"Change from baseline assessments across clinical examinations, ECG, echocardiogram and lab results"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 3. Incidence of local and systemic injection reactions","definition_or_measurement_approach":"Recording of local and systemic injection reactions (incidence)"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 4. Incidence of abnormal laboratory findings","definition_or_measurement_approach":"Incidence of clinically relevant abnormal laboratory findings compared to baseline"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 5. Incidence of abnormal ECG parameters","definition_or_measurement_approach":"Incidence of ECG parameter abnormalities compared to baseline"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 6. Incidence of relevant echocardiographic parameter z scores > 2","definition_or_measurement_approach":"Incidence of echocardiographic parameter z-scores greater than 2"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 7. Incidence of abnormal vital signs","definition_or_measurement_approach":"Incidence of abnormal vital sign measurements compared to baseline"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 8. Serum concentration of RO7204239 at specified timepoints","definition_or_measurement_approach":"RO7204239 serum PK measured at specified timepoints"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 9. Maximum observed concentration (Cmax) of RO7204239 at specified timepoints","definition_or_measurement_approach":"PK parameter Cmax for RO7204239 at specified timepoints"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 10. Area under the concentration-time curve (AUC) of RO7204239 at specified timepoints","definition_or_measurement_approach":"PK parameter AUC for RO7204239 at specified timepoints"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 11.Trough concentration (Ctrough) of RO7204239 at specified timepoints","definition_or_measurement_approach":"PK parameter Ctrough for RO7204239 at specified timepoints"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 12. Plasma concentration of risdiplam (and its metabolite M1) at specified timepoints","definition_or_measurement_approach":"Plasma PK of risdiplam and metabolite M1 at specified timepoints"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 13. Cmax of risdiplam (and its metabolite M1) at specified timepoints","definition_or_measurement_approach":"PK parameter Cmax for risdiplam and M1"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 14. AUC of risdiplam (and its metabolite M1) at specified timepoints","definition_or_measurement_approach":"PK parameter AUC for risdiplam and M1"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 15. Ctrough of risdiplam (and its metabolite M1) at specified timepoints","definition_or_measurement_approach":"PK parameter Ctrough for risdiplam and M1"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 16. Prevalence of anti-drug antibodies (ADAs) against RO7204239 at baseline and incidence of ADAs during the study","definition_or_measurement_approach":"Assessment of ADA prevalence at baseline and incidence during study using immunogenicity assays"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 17. Change from baseline in serum concentrations of total and free latent myostatin, and mature myostatin","definition_or_measurement_approach":"Serum biomarker concentrations (total/free latent and mature myostatin) compared to baseline"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 18. Percentage change from baseline in the contractile area of skeletal muscle in the dominant thigh as assessed by MRI (participants >=5 years) at Week 24 of combination treatment","definition_or_measurement_approach":"MRI assessment of contractile muscle area in dominant thigh; percent change from baseline at Week 24"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 19. Percentage change from baseline in the contractile area of skeletal muscle in the dominant calf as assessed by MRI (participants >=5 years) at Week 24 of combination treatment","definition_or_measurement_approach":"MRI assessment of contractile muscle area in dominant calf; percent change from baseline at Week 24"}
  • {"endpoint_text":"- Part 1 Ambulant Cohorts A-C: 20. Relationship between PK and PD endpoints, ADA status, safety, or efficacy","definition_or_measurement_approach":"Correlation analyses between PK/PD, ADA status and safety/efficacy outcomes"}
  • {"endpoint_text":"- Part 2: 21. Change from baseline in the Revised Hammersmith Scale (RHS) total score at Week 72 of combination treatment (Study Week 80)","definition_or_measurement_approach":"Change from baseline in RHS total score measured at Week 72 (Study Week 80)"}

Secondary endpoints

  • {"endpoint_text":"- Part 2: 1. Change from baseline in Motor Function Measure (MFM) Domain 1 + Domain 2 (D1 + D2) score at Week 72 of combination treatment (Study Week 80)","definition_or_measurement_approach":"Change from baseline in MFM D1+D2 score at Week 72"}
  • {"endpoint_text":"- Part 2: 2. Change from baseline in MFM-32 item total score at Week 72 of combination treatment (Study Week 80)","definition_or_measurement_approach":"Change from baseline in MFM-32 total item score at Week 72"}
  • {"endpoint_text":"- Part 2: 3. Change from baseline in the time taken to rise from floor as measured by RHS Item 25 at Week 72 of combination treatment (Study Week 80)","definition_or_measurement_approach":"Timed assessment (RHS Item 25) change from baseline at Week 72"}
  • {"endpoint_text":"- Part 2: 4. Change from baseline in the time taken to walk/run 10 meters as measured by RHS Item 19 at Week 72 of combination treatment (Study Week 80)","definition_or_measurement_approach":"Timed 10MWRT (RHS Item 19) change from baseline at Week 72"}
  • {"endpoint_text":"- Part 2: 5. Percent change from baseline in lean mass as assessed by full-body DXA scan (participants >=5 years) at Week 72 of combination treatment (Study Week 80)","definition_or_measurement_approach":"Full-body DXA percent change in lean mass from baseline at Week 72"}
  • {"endpoint_text":"- Part 2: 6. Incidence, severity, and causal relationship of AEs, with severity determined according to NCI CTCAE v5.0","definition_or_measurement_approach":"AE monitoring with severity graded per NCI CTCAE v5.0"}
  • {"endpoint_text":"- Part 2: 7. Change from baseline in vital signs, physical findings, ECG, echocardiogram, relevant echocardiographic parameters and clinical laboratory results","definition_or_measurement_approach":"Change from baseline across clinical and laboratory safety assessments"}
  • {"endpoint_text":"- Part 2: 8. Incidence of local and systemic injection reactions","definition_or_measurement_approach":"Recording incidence of injection reactions"}
  • {"endpoint_text":"- Part 2: 9. Incidence of abnormal laboratory findings","definition_or_measurement_approach":"Incidence of clinically relevant abnormal lab results"}
  • {"endpoint_text":"- Part 2: 10. Incidence of abnormal ECG parameters","definition_or_measurement_approach":"Incidence of ECG abnormalities"}
  • {"endpoint_text":"- Part 2: 11. Incidence of relevant echocardiographic parameter z scores >2","definition_or_measurement_approach":"Incidence of echocardiographic z-scores > 2"}
  • {"endpoint_text":"- Part 2: 12. Incidence of abnormal vital signs","definition_or_measurement_approach":"Incidence of abnormal vital sign measurements"}
  • {"endpoint_text":"- Part 2: 13. Serum concentration of RO7204239 at specified timepoints","definition_or_measurement_approach":"RO7204239 serum PK at specified timepoints"}
  • {"endpoint_text":"- Part 2: 14. Cmax of RO7204239 at specified timepoints","definition_or_measurement_approach":"Cmax measurement for RO7204239"}
  • {"endpoint_text":"- Part 2: 15. AUC of RO7204239 at specified timepoints","definition_or_measurement_approach":"AUC PK parameter for RO7204239"}
  • {"endpoint_text":"- Part 2: 16. Ctrough of RO7204239 at specified timepoints","definition_or_measurement_approach":"Ctrough PK parameter for RO7204239"}
  • {"endpoint_text":"- Part 2: 17. Plasma concentration of risdiplam (and its metabolite M1) at specified timepoints","definition_or_measurement_approach":"Plasma PK of risdiplam and M1"}
  • {"endpoint_text":"- Part 2: 18. Cmax of risdiplam (and its metabolite M1) at specified timepoints","definition_or_measurement_approach":"Cmax for risdiplam and M1"}
  • {"endpoint_text":"- Part 2: 19. AUC of risdiplam (and its metabolite M1) at specified timepoints","definition_or_measurement_approach":"AUC for risdiplam and M1"}
  • {"endpoint_text":"- Part 2: 20. Ctrough of risdiplam (and its metabolite M1) at specified timepoints","definition_or_measurement_approach":"Ctrough for risdiplam and M1"}
  • {"endpoint_text":"- Part 2: 21. Prevalence of ADAs at baseline and incidence of ADAs during the study; Impact of ADA on PK, PD, safety and efficacy parameters","definition_or_measurement_approach":"ADA prevalence and incidence; analyses of ADA impact on PK/PD/safety/efficacy"}

Recruitment

Planned Sample Size
42
Recruitment Window Months
81
Consent Approach
Informed consent is obtained from adult participants and from the parent(s)/legal representative(s) for minor participants. Age-appropriate assent forms are provided for children (assent forms and ICFs for ages 3-6, 7-11, 12-17 are listed). Caregiver information and caregiver ICFs are available where applicable. Multiple language versions of ICF/assent documents are provided (e.g., EN, DE, FR, NL, IT, PL and others) and optional addenda (e.g., video recording addendum) are included.

Methods

  • Patient Recruitment by Publicis Healthcare Communications Group Limited (role listed as 'Patient Recruitment' in third-party duties)

Geography

Total Number Of Sites
26
Total Number Of Participants
114

Belgium

Earliest CTIS Part Ii Submission Date
08-08-2024
Latest Decision Or Authorization Date
28-08-2024
Processing Time Days
20
Number Of Sites
2
Number Of Participants
11

Sites

Site Name
Universitair Ziekenhuis Gent
Department Name
Neuromuscular Reference Center
Contact Person Name
Nicolas Deconinck
Contact Person Email
elke.devos@uzgent.be
Site Name
Centre Hospitalier Regional De La Citadelle
Department Name
Neurology
Contact Person Name
Laurent Servais

Croatia

Earliest CTIS Part Ii Submission Date
08-08-2024
Latest Decision Or Authorization Date
02-09-2024
Processing Time Days
25
Number Of Sites
1
Number Of Participants
12

Sites

Site Name
KBC Zagreb
Department Name
Department of Pediatrics, Division for Neuropediatrics
Contact Person Name
Ivan Lehman
Contact Person Email
ilehman@kbc-zagreb.hr

Italy

Earliest CTIS Part Ii Submission Date
08-08-2024
Latest Decision Or Authorization Date
30-09-2024
Processing Time Days
53
Number Of Sites
6
Number Of Participants
21

Sites

Site Name
Centro Clinico Nemo
Department Name
Centro clinico NEMO (NEuroMuscular Omnicentre)
Contact Person Name
Michela Coccia
Site Name
Bambino Gesu Childrens Hospital
Department Name
UO Malattie Neuromuscolari e Neurodegenerative
Contact Person Name
Adele D'Amico
Contact Person Email
adele2.damico@opbg.net
Site Name
Giannina Gaslini Institute For Scientific Hospitalization And Care
Department Name
UOSD Centro di Miologia Traslazionale e Sperimentale
Contact Person Name
Claudio Bruno
Contact Person Email
claudiobruno@gaslini.org
Site Name
Centro Clinico Nemo
Department Name
Centro clinico NEMO (NEuroMuscular Omnicentre)
Contact Person Name
Valeria Sansone
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
UOC Neuropsichiatria Infantile
Contact Person Name
Eugenio Maria Mercuri
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
UOC Neurologia dello Sviluppo
Contact Person Name
Riccardo Masson

Spain

Earliest CTIS Part Ii Submission Date
08-08-2024
Latest Decision Or Authorization Date
26-08-2024
Processing Time Days
18
Number Of Sites
4
Number Of Participants
25

Sites

Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurología Pediátrica
Contact Person Name
Inmaculada Pitarch Castellano
Contact Person Email
Inmapitarch@hotmail.com
Site Name
Hospital Universitario La Paz
Department Name
Neurología Pediátrica
Contact Person Name
María del Mar García Romero
Contact Person Email
yambee@hotmail.com
Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurología Pediátrica
Contact Person Name
David Gómez Andrés
Site Name
Sant Joan De Deu Barcelona Hospital
Department Name
Neurología Pediátrica
Contact Person Name
Andrés Nascimento Osorio

Netherlands

Earliest CTIS Part Ii Submission Date
08-08-2024
Latest Decision Or Authorization Date
26-08-2024
Processing Time Days
18
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Universitair Medisch Centrum Utrecht
Department Name
Neuromuscular Diseases
Contact Person Name
Ludo van der Pol
Contact Person Email
smaonderzoek@umcutrecht.nl

Germany

Earliest CTIS Part Ii Submission Date
29-08-2024
Latest Decision Or Authorization Date
09-09-2024
Processing Time Days
11
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Kinderheilkunde
Contact Person Name
Andrea Gangfuß
Contact Person Email
andrea.gangfuss@uk-essen.de

France

Earliest CTIS Part Ii Submission Date
08-08-2024
Latest Decision Or Authorization Date
02-09-2024
Processing Time Days
25
Number Of Sites
4
Number Of Participants
10

Sites

Site Name
Raymond Poincare Hospital
Department Name
Neurologie et Réanimation pédiatriques
Contact Person Name
Marta GOMEZ GARCIA DE LA BANDA
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
Service de Neuropédiatrie
Contact Person Name
ULRIKE WALTHER LOUVIER
Contact Person Email
u-louvier@chu-montpellier.fr
Site Name
Hospices Civils De Lyon
Department Name
Service Central de Rééducation pédiatrique L’Escale
Contact Person Name
Carole Vuillerot
Contact Person Email
carole.vuillerot@chu-lyon.fr
Site Name
Trousseau Hospital
Department Name
Institut I-Motion
Contact Person Name
Silvana De Lucia

Poland

Earliest CTIS Part Ii Submission Date
08-08-2024
Latest Decision Or Authorization Date
05-10-2024
Processing Time Days
58
Number Of Sites
5
Number Of Participants
19

Sites

Site Name
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
Department Name
Klinika Neurologii I Wydzialu Lekarskiego WUM
Contact Person Name
Anna Kostera-Pruszczyk
Contact Person Email
kn.csk@wum.edu.pl
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Klinika Neurologii Rozwojowej
Contact Person Name
Maria Mazurkiewicz-Beldzinska
Contact Person Email
neurologiarozwojowa@uck.gda.pl
Site Name
Uniwersytecki Szpital Kliniczny W Poznaniu
Department Name
Oddział Kliniczny Neurologii Dzieci i Młodzieży
Contact Person Name
Barbara Steinborn
Contact Person Email
nwrsk2@ump.edu.pl
Site Name
Instytut Centrum Zdrowia Matki Polki
Department Name
Klinika Neurologii Rozwojowej i Epileptologii
Contact Person Name
Lukasz Przyslo
Contact Person Email
sek49@iczmp.edu.pl
Site Name
Instytut Pomnik Centrum Zdrowia Dziecka
Department Name
Klinika Neurologii i Epileptologii
Contact Person Name
Katarzyna Kotulska-Jóźwiak
Contact Person Email
oddzial.neurologia@ipczd.pl

Portugal

Earliest CTIS Part Ii Submission Date
28-08-2024
Latest Decision Or Authorization Date
28-08-2024
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Hospital De Santa Maria E.P.E.
Department Name
Pediatria
Contact Person Name
Joana Coelho
Contact Person Email
ped.ensaioscaml@gmail.com
Site Name
Unidade Local De Saude De Sao Jose E.P.E.
Department Name
Neuropediatria
Contact Person Name
Sandra Jacinto

Sponsor

Primary sponsor

Full Name
F. Hoffmann-La Roche AG
Organisation Type
Pharmaceutical company
Country Of Registered Address
Switzerland

Contract research organisations

Name
Icon Development Solutions LLC
Responsibilities
code: 4
Name
IQVIA Limited
Responsibilities
Monitoring; EKG Provider (roles listed across third-party entries)
Name
Signant Health Global LLC
Responsibilities
Rater Management, Scale Management and Equipment Fulfillment; eCOA/rater services
Name
MARKEN Germany GmbH
Responsibilities
Clinical Supply Management Services
Name
Greenphire LLC
Responsibilities
Drug Management
Name
SGS Analytics Switzerland AG
Responsibilities
Laboratory/analytical services (code: 4)

Third parties

  • {"country":"United States","full_name":"Icon Development Solutions LLC","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"code: 4","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Germany","full_name":"MicroCoat Biotechnologie GmbH","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Blueprint Genetics Inc.","duties_or_roles":"code: 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Signant Health Global LLC","duties_or_roles":"for Rater Management, Scale Management and Equipment Fulfillment","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"SGS Analytics Switzerland AG","duties_or_roles":"code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"France","full_name":"Sysnav","duties_or_roles":"Provision of Syde Devices","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Q2q Communications Limited","duties_or_roles":"Meeting Organizer","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Drug Management","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Monitoring","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Publicis Healthcare Communications Group Limited","duties_or_roles":"Patient Recruitment","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Invicro LLC","duties_or_roles":"Imaging Services (MRI, DEXA)","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"MARKEN Germany GmbH","duties_or_roles":"Clinical Supply Management Services","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Chillibean Limited","duties_or_roles":"Video Capture, Upload, Storage, De-Identification","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
RO 720-4239
Modality
Monoclonal antibody
Investigational Product Name
RO 703-4067 (risdiplam)
Active Substance
Risdiplam
Modality
Small molecule
Orphan Designation
Yes
Combination Treatment
Yes

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