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Healthy VolunteersPhase 1NCT03040635

A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants

A Phase 1 study testing Risdiplam in people with Healthy Volunteers, run by Hoffmann-La Roche at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a randomized, placebo-controlled study to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of single oral doses of Risdiplam in healthy Japanese participants. Read more about this study

Who is it for?
Ages 18 to 60 yearsHealthy volunteers can take part
Conditions the study is for
Healthy Volunteers
What is being tested?
Risdiplam
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
24 participants
Where
1 siteUnited States
Study dates
22 Mar 2017to 2 Oct 2017
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 60 years
  • All sexes
  • Healthy volunteers accepted
  • Adult

Who may be able to join

  • Participants of Japanese origin, with Japanese parents (for the provisional Caucasian cohort, participants must be male or female Caucasians with 4 Caucasian grand-parents)
  • Healthy participants. Healthy status is defined by the absence of evidence of any active or chronic disease following a detailed medical and surgical history, a complete physical examination including ophthalmological examination, vital signs, 12-lead electrocardiogram (ECG), hematology, blood chemistry, serology and urinalysis
  • A body mass index (BMI) between 18 to 30 kilograms per square meter (kg/m^2) inclusive
  • Male participants must agree to use a barrier method of contraception from dosing until completion of the study and for 4 months thereafter
  • Female participants must be either surgically sterile or post-menopausal

Who cannot join

  • Medical history of cardiovascular disease, renal disease, liver disease, digestive system disease, blood dyscrasia, immunologic disease, diseases of the nervous system, endocrine disease, metabolic disease, lung disease, or with anamnesis and obstacles in kidney, liver, or cardiopulmonary function
  • Participants with any clinically significant eye pathology
  • Laboratory test (hematology, biochemistry, physical examination or vital signs) values outside the institutional normal range and rated as clinically significant abnormal at screening
  • Any clinically significant abnormalities in ECG at screening
  • Inherited long QT syndrome or known family history of arrhythmia
  • Systolic blood pressure (SBP) higher than 140 millimeters of mercury (mmHg) or below 90 mmHg, and/or diastolic blood pressure (DBP) higher than 90 mmHg or below 50 mmHg in the supine position, as assessed at screening
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  • Positive result for human immunodeficiency virus (HIV) antigen and antibody, hepatitis B surface antigen (HBsAg) or hepatitis C virus (HCV) antibody at screening
  • Donation or loss of blood over 500 milliliters (mL) within three months prior to screening
  • History of smoking more than 10 cigarettes a day. Participants who have quit smoking or who have reduced their daily cigarette smoking to 10 or less for more than one month prior to screening are allowed
  • Daily consumption of food or drink containing a large amount of methylxanthine (caffeine, theophylline, theobromine)
  • Present or past history of substance addiction, dependence or abuse, such as abuse of drugs or alcohol
  • Concomitant or previous participation in any clinical trial either within 90 days or 5 half-lives of the investigational drug, whichever is longer, before administration of study drug in this study
  • Use of prescribed medications which have systemic exposure within one week before enrolment
  • Use of any concomitant drug during the study (including over-the-counter medication and medications used in dentistry)
  • Inability to meet the study requirements in the opinion of the Investigator

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Collaborative Neuroscience Network, Inc.

    Garden Grove, California, 92845

Other trials in United States

About this study

Official title: An Investigator/Subject Blind, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Doses of RO7034067 in Healthy Japanese Subjects

This is a randomized, placebo-controlled study to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of single oral doses of Risdiplam in healthy Japanese participants.

Being tested
Risdiplam, Placebo

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RisdiplamDrug

    Also called RO7034067

    Single oral doses of Risdiplam will be administered on Day 1.

  • PlaceboDrug

    Single oral doses of Risdiplam matching placebo will be administered on Day 1.

Study groups in NCT03040635
Study groupRoleWhat they receive
Risdiplam '2' Milligrams (mg)ExperimentalA single dose of 2 mg Risdiplam will be administered to all participants randomized to this arm under fasted conditions as an oral drinking solution on Day 1.
Receives: Drug: Risdiplam
Risdiplam '6' mgExperimentalA single dose of 6 mg Risdiplam will be administered to all participants randomized to this arm under fasted conditions as an oral drinking solution on Day 1.
Receives: Drug: Risdiplam
Risdiplam '12' mgExperimentalA single dose of 12 mg Risdiplam will be administered to all participants randomized to this arm under fasted conditions as an oral drinking solution on Day 1.
Receives: Drug: Risdiplam
PlaceboPlacebo comparatorA single dose of placebo will be administered to all participants randomized to this arm under fasted conditions as an oral drinking solution on Day 1.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum Observed Plasma Concentration (Cmax) of Risdiplam

    Measured: Pre-dose (0 hour [hr]), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

  2. Time to Maximum Plasma Concentration (Tmax) of Risdiplam

    Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

  3. Area Under the Plasma Concentration-Time Curve up to the Last Measurable Concentration (AUClast) of Risdiplam

    Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

  4. Area Under the Plasma Concentration-Time Curve Extrapolated to Infinity (AUC0-inf) of Risdiplam

    Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

  5. Area Under the Plasma Concentration-Time Curve up to Time t (AUC0-t) of Risdiplam

    Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

  6. Apparent Terminal Half-Life (t1/2) of Risdiplam

    Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

  7. Apparent Oral Clearance (CL/F) of Risdiplam

    Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

  8. Apparent Oral Volume of Distribution (Vz/F) of Risdiplam

    Measured: Pre-dose (0 hr), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1

  9. Cumulative Amount Excreted Unchanged into Urine (Ae) of Risdiplam

    Measured: Pre-dose (0 hr), and 5 urine samples at different time-ranges postdose (0-6 hr, 6-12 hr, 12-24 hr, 24-48 hr, and 48-72 hr)

  10. Renal Clearance (CLR) of Risdiplam

    Measured: Pre-dose (0 hr), and 5 urine samples at different time-ranges postdose (0-6 hr, 6-12 hr, 12-24 hr, 24-48 hr, and 48-72 hr)

  11. Fraction of Dose Excreted Unchanged Renally (Fe) of Risdiplam

    Measured: Pre-dose (0 hr), and 5 urine samples at different time-ranges postdose (0-6 hr, 6-12 hr, 12-24 hr, 24-48 hr, and 48-72 hr)

  12. Percentage of Participants with Adverse Events (AEs)

    Measured: Up to approximately 7 weeks

Secondary outcomes (3)

  1. Change from Baseline in Splicing Modifications of Survival of Motor Neuron (SMN) Messenger Ribonucleic Acids (mRNAs), Including SMN1, SMN2 Full Length (FL), and SMNdelta7 mRNA in Blood In Vivo

    Measured: Days -1, 1, 2, 3, 4, 6

  2. Change from Baseline in mRNA Levels of Paired Box (PAX) Genes in Blood

    Measured: Days -1, 1, 2, 3, 4, 6

  3. Change from Baseline in SMN Protein Levels in Blood

    Measured: Day -1, follow-up visit (Day 21)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Feb 2017 (estimated)
  2. Study start22 Mar 2017
  3. Primary completion2 Oct 2017
  4. Study completion2 Oct 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
24 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03040635
Sponsor's protocol ID
NP39625
Status last verified
Oct 2018
First submitted
1 Feb 2017
Last updated
4 Oct 2018
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Risdiplam in people with Healthy Volunteers, run by Hoffmann-La Roche at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Observed Plasma Concentration (Cmax) of Risdiplam (time frame: Pre-dose (0 hour [hr]), 0.5, 1, 2, 3, 4, 4.5, 5, 6, 8, 10, 12, 24, 36, 48, 72, 120, 168, 264 hr postdose from Day 1). The study lists 12 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03040635.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Oct 2018; AzTrials last refreshed its copy on 7 Nov 2026.

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