Trial · NCT03032172

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

NCT03032172, Phase 2, Completed, Hoffmann-La Roche; results first posted 2025-10-01

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy. Trials, record NCT03032172. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03032172

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
NCT number: NCT03032172
Status: Completed
Phases: Phase 2
Enrollment: 174 participants, actual
Lead sponsor: Hoffmann-La Roche
Conditions: Spinal Muscular Atrophy
Interventions: Risdiplam
First posted: 2017-01-26
Last updated: 2025-10-01
Other registries' IDs: eudract:2016-004184-39
Results first posted: 2025-10-01

Source ClinicalTrials.gov

Registered outcomes12

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Number of Participants With Adverse Events (AEs)	Baseline up to 5 years	An AE was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product, regardless of causal attribution. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
Primary	Number of Participants Who Discontinued Treatment Due to AEs	Baseline up to 5 years	An AE was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product, regardless of causal attribution. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Participants who discontinued treatment due to AEs are reported here.
Primary	Number of Participants With Shift in Puberty Status From Baseline	Baseline, Week 52, 104, 156, 208 and 260	Tanner staging of sexual development is a scale used to assess physical maturation as children transition through adolescence into adulthood. Scale defines physical development based on the following characteristics: pubic hair, penis, and testes development in boys; and pubic hair and breast development in girls. It consists of 5 stages, Stage I (prepubertal) to Stage V (mature adult). Participants under 9 years at screening began Tanner staging assessments at 1st visit following their 9th birthday. Tanner data are presented in three categories: Normal (expected stage of puberty based on participant's age at post-baseline visit), Delayed (pubertal development is behind expectations for age at post-baseline visit), \& Missing (participant did not attend scheduled visit). Tanner staging assessments were scheduled for participants aged 9-17 years but were also conducted in some older participants, up to age 22. Shift in puberty status from baseline to each week has been represented here.
Primary	Number of Participants With Protocol-defined Neurological Conditions (NC)	Baseline up to Week 260	Neurological examination was performed by asking questions to the participants and/or their caregiver, as well as observing the participants' behavior in general and while performing certain tasks. Questions and tasks were adapted to the age and motor ability of the participant. For very young participants, observing reaction to a sound, speech development, shifting attention to a newly introduced toy, observing the participant interact with the parent/caregiver \& for older participants examination of social interaction (school, friends, activities, job as appropriate), memory (e.g., with short word recall), reasoning \& language, drawing, etc. Participants with neurological conditions besides those expected with SMA and those expected with SMA are reported.
Primary	Number of Participants With Emergence or Worsening of Symptoms as Assessed Using Columbia Suicide Severity Rating Scale (C-SSRS)	Up to approximately 5 years	C-SSRS was used to assess the lifetime suicidality of a participant (baseline) as well as any new instances of suicidality (since last visit). The interview prompts recollection of suicidal ideation (SI), including the intensity of the ideation, behavior and attempts with actual/potential lethality. A modified and reduced version (pediatric version) was used for children (aged 6-11 years). Categories have binary responses (yes/no) \& include: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active SI with Any Methods (Not Plan) without Intent to Act; Active SI with Some Intent to Act, without Specific Plan; Active SI with Specific Plan \& Intent, Preparatory Acts \& Behavior; Aborted Attempt; Interrupted Attempt; Actual Attempt (non-fatal); Completed Suicide. SI or behavior is indicated by a "yes" answer to any of the listed categories. A score of 0 is assigned if no suicide risk is present. A score of 1 or higher indicates SI or behavior.
Primary	Anthropometric Examination: Change From Baseline in Weight	Baseline up to Week 260	Weight was measured at baseline, Weeks 65, 91, and every 13 weeks thereafter for participants 2-17 years and at every visit for participants \<2 years. Per protocol, height was not measured at Day 7, Weeks 8, 17, 35, and thus not collected for all participants. No participants in the RO6885247 arm attended the Weeks 65, 169, 195, and 247 visit; none in the AVXS-101 arm attended the Week 247 visit. Symptom-directed height/weight assessments were done at clinically indicated visits as needed.
Primary	Anthropometric Examination: Change From Baseline in Height	Baseline up to Week 260	Height of all participants able to stand was measured while standing using a stadiometer, with at least 3 independent measurements, which were averaged. Participants unable to stand during the measurement, height was derived from measurement of ulna length. For very young children, height was measured with child in a lying position using an inflexible length board with fixed headboard \& movable footboard. Height was measured: baseline, Weeks 13,39,52,78 \& 104 for participants 2-17 years; Weeks 52 \& 104 for \>17 years \& every visit for participants \<2 years. After Week 104, measurements occurred every 26 weeks. Per protocol, height was not measured at Weeks 2,4,17,26,35,43,61,65,87,91,96,117,143,169,195,221 \&247 \& thus not collected for all participants. No participants \<17 years in RO6885247, Olesoxime, or AVXS-101 arms attended the Week 260 visit \& no participants in RO6885247 arm attended Week 39. Symptom-directed height assessments were done at clinically indicated visits as needed.
Primary	Anthropometric Examination: Change From Baseline in Head Circumference	Baseline up to Week 208	Head circumference for participants aged \< 5 years was measured to the nearest 0.1 cm using a flexible, non-stretchable tape. Head circumference was measured around the widest part of the head from the most prominent point on the back of the head (occiput) to the most prominent part of the forehead between the eyebrows. The measurement was repeated three times, and the largest measurement was recorded. Head circumference was assessed for participants \< 5 years of age. Since all participants in the RO6885247 and Olesoxime arms were \> 5 years, data were not collected for these arms. Head circumference was not collected at Weeks 35 and 61 (AVXS-101 arm) and Weeks 87, 182, and 208 (Nusinersen arm) because the protocol only required this measurement for participants \< 5 years of age. At these timepoints, neither of the participants \< 5 years attended the visit, and other participants may have been \> 5 years of age.
Primary	Maximum Plasma Concentration (Cmax) of Risdiplam	Predose on Weeks 2, 4, 13, 26, 39, 52, 65, 91 and post-dose on Weeks 1, 4, 13, 52, 91 and 104	As pre-specified in the protocol, the PK data were not to be compared between participants who received different prior treatments (RO6885247, nusinersen, olesoxime and AVXS-101) received before entering this study, but to analyze the PK parameters of risdiplam. Hence, PK data have been presented in a single arm group irrespective of prior therapies received by the participants in previous studies.
Primary	Area Under the Concentration-Time Curve (AUC0-24h) of Risdiplam	24 hours Postdose at Year 2 Visit	As pre-specified in the protocol, the PK data were not to be compared between participants who received different prior treatments (RO6885247, nusinersen, olesoxime and AVXS-101) received before entering this study, but to analyze the PK parameters of risdiplam. Hence, PK data have been presented in a single arm group irrespective of prior therapies received by the participants in previous studies.

1 to 10 of 12Source ClinicalTrials.gov

See all 12 registered outcomes

Outcomes12

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants With Adverse Events (AEs)	Baseline up to 5 years	SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants Who Discontinued Treatment Due to AEs	Baseline up to 5 years	SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Shift in Puberty Status From Baseline	Baseline, Week 52, 104, 156, 208 and 260	SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Overall number analyzed is the number of participants with data available for analysis. No participants from the AVXS-101 arm were assessed for Tanner staging since all participants in this arm were under 9 years of age.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Protocol-defined Neurological Conditions (NC)	Baseline up to Week 260	SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Number of Participants With Emergence or Worsening of Symptoms as Assessed Using Columbia Suicide Severity Rating Scale (C-SSRS)	Up to approximately 5 years	SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Participants with SI, suicidal behavior, and self-injurious behavior without suicidal intent are reported for this outcome measure. Overall number analyzed are the number of participants with at least one post-baseline assessment.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Anthropometric Examination: Change From Baseline in Weight	Baseline up to Week 260	SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint.	kilograms (kg)	MEAN
PRIMARY	Anthropometric Examination: Change From Baseline in Height	Baseline up to Week 260	SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint.	centimeters (cm)	MEAN
PRIMARY	Anthropometric Examination: Change From Baseline in Head Circumference	Baseline up to Week 208	SE population included all participants who received at least one dose of risdiplam, whether prematurely withdrawn from the study or not. Overall number analyzed is the number of participants with data available for analysis. Number analyzed is the number of participants with data available for analysis at the specified timepoint.	cm	MEAN
PRIMARY	Maximum Plasma Concentration (Cmax) of Risdiplam	Predose on Weeks 2, 4, 13, 26, 39, 52, 65, 91 and post-dose on Weeks 1, 4, 13, 52, 91 and 104	PK population included all participants with at least one timepoint with a measurable drug concentration. Overall number analyzed is the number of participants with data available for analysis.	nanograms per milliliter (ng/mL)	MEDIAN
PRIMARY	Area Under the Concentration-Time Curve (AUC0-24h) of Risdiplam	24 hours Postdose at Year 2 Visit	PK population included all participants with at least one timepoint with a measurable drug concentration. Overall number analyzed is the number of participants with data available for analysis.	nanogramshour/milliliter (ngh/mL)	MEDIAN

1 to 10 of 12Source ClinicalTrials.gov

See all 12 outcomes

Results by group469

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Number of Participants With Adverse Events (AEs)	RO6885247		12	Participants
Number of Participants With Adverse Events (AEs)	Nusinersen		76	Participants
Number of Participants With Adverse Events (AEs)	Olesoxime		70	Participants
Number of Participants With Adverse Events (AEs)	AVXS-101		14	Participants
Number of Participants Who Discontinued Treatment Due to AEs	RO6885247		0	Participants
Number of Participants Who Discontinued Treatment Due to AEs	Nusinersen		1	Participants
Number of Participants Who Discontinued Treatment Due to AEs	Olesoxime		0	Participants
Number of Participants Who Discontinued Treatment Due to AEs	AVXS-101		0	Participants
Number of Participants With Shift in Puberty Status From Baseline	RO6885247	Baseline Status Normal: Week 52 Status Normal	0	Participants
Number of Participants With Shift in Puberty Status From Baseline	Nusinersen	Baseline Status Normal: Week 52 Status Normal	0	Participants

1 to 10 of 469Source ClinicalTrials.gov

See all 469 results by group

Participant flow56

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Treatment Period	STARTED	Treatment Phase: RO6885247	13
Treatment Period	STARTED	Treatment Phase: Nusinersen	76
Treatment Period	STARTED	Treatment Phase: Olesoxime	71
Treatment Period	STARTED	Treatment Phase: AVXS-101	14
Treatment Period	STARTED	OLE Phase: RO6885247	0
Treatment Period	STARTED	OLE Phase: Nusinersen	0
Treatment Period	STARTED	OLE Phase: Olesoxime	0
Treatment Period	STARTED	OLE Phase: AVXS-101	0
Treatment Period	Safety-evaluable (SE) Population	Treatment Phase: RO6885247	13
Treatment Period	Safety-evaluable (SE) Population	Treatment Phase: Nusinersen	76

1 to 10 of 56Source ClinicalTrials.gov

See all 56 participant flow

Adverse events896

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Anaemia	Blood and lymphatic system disorders	RO6885247	1	13	1
other	Anaemia	Blood and lymphatic system disorders	Nusinersen	0	76	0
other	Anaemia	Blood and lymphatic system disorders	Olesoxime	8	70	10
other	Anaemia	Blood and lymphatic system disorders	AVXS-101	0	14	0
other	Iron deficiency anaemia	Blood and lymphatic system disorders	RO6885247	1	13	1
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Nusinersen	0	76	0
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Olesoxime	1	70	1
other	Iron deficiency anaemia	Blood and lymphatic system disorders	AVXS-101	0	14	0
other	Lymphadenopathy	Blood and lymphatic system disorders	RO6885247	0	13	0
other	Lymphadenopathy	Blood and lymphatic system disorders	Nusinersen	0	76	0

1 to 10 of 896Source ClinicalTrials.gov

See all 896 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	RO6885247	0	13
Total, all-cause mortality	Nusinersen	0	76
Total, all-cause mortality	Olesoxime	0	70
Total, all-cause mortality	AVXS-101	2	14
Total, other adverse events	RO6885247	12	13
Total, other adverse events	Nusinersen	76	76
Total, other adverse events	Olesoxime	69	70
Total, other adverse events	AVXS-101	14	14
Total, serious adverse events	RO6885247	3	13
Total, serious adverse events	Nusinersen	25	76

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
37148485	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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Read this trial from software.

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