Trial · NCT03342053

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)

NCT03342053, Phase 2, Completed, Hoffmann-La Roche; results first posted 2021-01-19

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139). Trials, record NCT03342053. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03342053

On this page

Trial

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

Title: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)
NCT number: NCT03342053
Status: Completed
Phases: Phase 2
Enrollment: 46 participants, actual
Lead sponsor: Hoffmann-La Roche
Conditions: Huntington's Disease
Interventions: RO7234292 (RG6042)
First posted: 2017-11-14
Last updated: 2022-03-24
Results first posted: 2021-01-19

Source ClinicalTrials.gov

Registered outcomes8

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

List	Outcome	Time frame	Description
Primary	Percentage of Participants With Adverse Events	From baseline up to 18 months	Adverse Events include Adverse Events that started at or after Date/Time of First Exposure to Treatment and procedure-related Adverse Events occurring before the start of treatment.
Secondary	RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	From baseline to Day 421
Secondary	CSF mHTT Protein Concentration Logarithmic Value Change in Geometric Mean (95%CI) From Baseline	From Baseline to Day 421	The results of the planned analysis related to mHTT protein levels in CSF are reported
Secondary	Mean Percentage Change in Ventricular Volume Boundary Shift Integral From Baseline to 15 Months	Baseline up to 15 months
Secondary	Mean Percentage Change in Caudate Volume Boundary Shift Integral From Baseline to 15 Months	Baseline up to 15 months
Secondary	Mean Percentage Change in Whole Brain Volume Boundary Shift Integral From Baseline to 15 Months	Baseline up to 15 months
Secondary	EEG Parameters: Mean Change From Baseline to 15 Months in Absolute Power [8-12Hz]	Baseline to 15 Months
Secondary	Mean Change From Baseline in Huntington's Disease Cognitive Assessment Battery Composite Score	Baseline to 15 Months	HD Cognitive Assessment Battery (HD-CAB) was developed to assess cognitive dysfunction in late premanifest and early manifest HD patients. HD-CAB combines scores from six cognitive tests: SDMT, Self-Paced Tapping, Emotional Recognition, CANTAB One Touch Stocking, Hopkins Verbal Learning Test - Revised, and Trail-Making Test. A multi-component score is derived by transforming the subject's score on each cognitive test to a z-score. Using z-scores permits the combination of test scores with different scales. Unlike other measures that use an external reference population to create z-scores, HD-CAB uses the baseline data of the study. Individually, for each of the six cognitive tests, the study baseline mean is subtracted from the subject's score, and this value is divided by the study baseline standard deviation. The six z-scores are averaged to produce the HD-CAB score. A positive change from baseline indicates improvement in cognitive function; a negative change indicates worsening.

Source ClinicalTrials.gov

Outcomes8

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percentage of Participants With Adverse Events	From baseline up to 18 months	Safety population comprising all participants that were randomized and received at least one dose of RO7234292.	Percentage of participants	NUMBER
SECONDARY	RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	From baseline to Day 421	CSF Trough Concentrations were reported following study drug administration. Data for Bi-monthly arm was not collected on days 57, 113, 169, 225, 281, 337, 393. Here Number Analyzed represents number of participants from whom samples were collected and analyzed.	ng/mL	GEOMETRIC_MEAN
SECONDARY	CSF mHTT Protein Concentration Logarithmic Value Change in Geometric Mean (95%CI) From Baseline	From Baseline to Day 421	ITT Population. The data for Bi-monthly arm was not collected on days 113, 169, 225, 281, 337, 393. Here Number Analyzed represents number of participants from whom samples were collected and analyzed	Log (10) fmol/L	GEOMETRIC_MEAN
SECONDARY	Mean Percentage Change in Ventricular Volume Boundary Shift Integral From Baseline to 15 Months	Baseline up to 15 months	Here Number of Participants Analyzed represents number of participants from whom data were collected and analyzed. Only data that passed the QC imaging process were included in the analysis.	Percentage change	MEAN
SECONDARY	Mean Percentage Change in Caudate Volume Boundary Shift Integral From Baseline to 15 Months	Baseline up to 15 months	Here Number of Participants Analyzed represents number of participants from whom data were collected and analyzed.	Percentage change	MEAN
SECONDARY	Mean Percentage Change in Whole Brain Volume Boundary Shift Integral From Baseline to 15 Months	Baseline up to 15 months	Here Number of Participants Analyzed represents number of participants from whom data were collected and analyzed.	Percentage change	MEAN
SECONDARY	EEG Parameters: Mean Change From Baseline to 15 Months in Absolute Power [8-12Hz]	Baseline to 15 Months		log10(mircrovolts^2)	MEAN
SECONDARY	Mean Change From Baseline in Huntington's Disease Cognitive Assessment Battery Composite Score	Baseline to 15 Months		z-score	MEAN

Source ClinicalTrials.gov

Results by group59

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

Outcome	Group	Category	Value	Spread	Units
Percentage of Participants With Adverse Events	RO7234292 Monthly		100		Percentage of participants
Percentage of Participants With Adverse Events	RO7234292 Bimonthly		95.7		Percentage of participants
RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	RO7234292 Monthly	Day 29	2.59	Full Range 1.31 to 12.7	ng/mL
RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	RO7234292 Bimonthly	Day 29	2.52	Full Range 0.817 to 7.85	ng/mL
RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	RO7234292 Monthly	Day 57	3.47	Full Range 1.43 to 19.2	ng/mL
RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	RO7234292 Monthly	Day 85	3.70	Full Range 1.38 to 7.34	ng/mL
RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	RO7234292 Bimonthly	Day 85	1.39	Full Range 0.164 to 5.74	ng/mL
RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	RO7234292 Monthly	Day 113	3.97	Full Range 1.96 to 16.4	ng/mL
RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	RO7234292 Monthly	Day 141	4.57	Full Range 1.72 to 13	ng/mL
RO7234292 CSF Trough Concentrations by Study Day Prior to Monthly and Bimonthly IT Administration of 120 mg RO7234292 (Primary Analysis)	RO7234292 Bimonthly	Day 141	1.35	Full Range 0.396 to 3.69	ng/mL

1 to 10 of 59Source ClinicalTrials.gov

See all 59 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	RO7234292 Monthly	23
Overall Study	STARTED	RO7234292 Bimonthly	23
Overall Study	COMPLETED	RO7234292 Monthly	21
Overall Study	COMPLETED	RO7234292 Bimonthly	22
Overall Study	NOT COMPLETED	RO7234292 Monthly	2
Overall Study	NOT COMPLETED	RO7234292 Bimonthly	1

Source ClinicalTrials.gov

Adverse events140

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	Vertigo	Ear and labyrinth disorders	RO7234292 Monthly	2	23	2
other	Vertigo	Ear and labyrinth disorders	RO7234292 Bimonthly	0	23	0
other	Diarrhoea	Gastrointestinal disorders	RO7234292 Monthly	0	23	0
other	Diarrhoea	Gastrointestinal disorders	RO7234292 Bimonthly	5	23	6
other	Nausea	Gastrointestinal disorders	RO7234292 Monthly	3	23	3
other	Nausea	Gastrointestinal disorders	RO7234292 Bimonthly	3	23	5
other	Vomiting	Gastrointestinal disorders	RO7234292 Monthly	2	23	2
other	Vomiting	Gastrointestinal disorders	RO7234292 Bimonthly	2	23	2
other	Fatigue	General disorders	RO7234292 Monthly	3	23	3
other	Fatigue	General disorders	RO7234292 Bimonthly	1	23	1

1 to 10 of 140Source ClinicalTrials.gov

See all 140 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	RO7234292 Monthly	1	23
Total, all-cause mortality	RO7234292 Bimonthly	0	23
Total, other adverse events	RO7234292 Monthly	22	23
Total, other adverse events	RO7234292 Bimonthly	22	23
Total, serious adverse events	RO7234292 Monthly	4	23
Total, serious adverse events	RO7234292 Bimonthly	3	23

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

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 comparisons2

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

Comparison	Source
RO7234292 Monthly vs RO7234292 Bimonthly: deaths from any cause	Trial comparisons
RO7234292 Monthly vs RO7234292 Bimonthly: serious adverse events	Trial comparisons

Tests3

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

Test	Named as	Source
Trail Making Test A	Secondary outcome	ConductCognition
Trail Making Test B	Secondary outcome	ConductCognition
Verbal Learning	Secondary outcome	ConductCognition

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.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03342053

GET https://conductscience.org/api/trials/v1/NCT03342053/results?page=2

API reference For AI assistants