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Trial record 1 of 1 for:    Jewelfish
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A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy (Jewelfish)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03032172
Recruitment Status : Active, not recruiting
First Posted : January 26, 2017
Last Update Posted : April 24, 2023
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Description
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Brief Summary:
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Drug: Risdiplam Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 174 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients With Spinal Muscular Atrophy
Actual Study Start Date : March 3, 2017
Estimated Primary Completion Date : December 27, 2024
Estimated Study Completion Date : December 27, 2024

Resource links provided by the National Library of Medicine

Drug Information available for: Risdiplam

Arms and Interventions
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Arm Intervention/treatment
Experimental: Risdiplam
Participants will receive multiple doses of risdiplam orally once daily for 24 months. After 24-month treatment, participants will be offered the opportunity to enter the open-label extension (OLE) phase.
 Drug: Risdiplam
Risdiplam will be administered orally once daily.
Other Name: RO7034067


Outcome Measures
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Primary Outcome Measures :
  1. Percentage of Participants With Adverse Events (AEs) and Serious AEs (SAEs) with Severity Determined According to National Cancer Institute Common Terminology Criteria for Adverse Events Scale, V 4.0 [ Time Frame: Baseline up to 5 years ]
  2. Percentage of Participants With Emergence or Worsening of Symptoms As Assessed Using Columbia Suicide Severity Rating Scale (C-SSRS) (Adult Version for Adults and Adolescents, Pediatric Version for Patients Aged 6-11 Years) [ Time Frame: Baseline up to 5 years ]
  3. Percentage of Participants With Protocol Defined Clinically Significant Changes in Ophthalmological Assessments [ Time Frame: Baseline up to 5 years ]
  4. Percentage of Participants With Protocol Defined Clinically Significant Changes in Neurological Assessments [ Time Frame: Baseline up to 5 years ]
  5. Tanner Staging Among all Participants Aged From 9 to 17 Years [ Time Frame: Baseline up to 5 years ]
  6. Mean Plasma Concentration of Risdiplam [ Time Frame: Up to 2 years ]
  7. Maximum Plasma Concentration (Cmax) of Risdiplam [ Time Frame: Up to 2 years ]
  8. Area Under the Plasma Concentration Versus Curve (AUC) of Risdiplam [ Time Frame: Up to 2 years ]
  9. Concentration of Risdiplam at the End of Dosing Interval (Ctrough) [ Time Frame: Up to 2 years ]
  10. Mean Plasma Concentration of Risdiplam Metabolite [ Time Frame: Up to 2 years ]
  11. Cmax of Risdiplam Metabolite [ Time Frame: Up to 2 years ]
  12. AUC of Risdiplam Metabolite [ Time Frame: Up to 2 years ]
  13. Ctrough of Risdiplam Metabolite [ Time Frame: Up to 2 years ]

Secondary Outcome Measures :
  1. SMN messenger Ribonucleic Acid (mRNA) Level in Blood [ Time Frame: Up to 2 years ]
  2. SMN Protein Levels in Blood [ Time Frame: Up to 2 years ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 60 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed diagnosis of 5q-autosomal recessive SMA
  • Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previous treatment with any of the following: 1.) Nusinersen (defined as having received >= 4 doses of nusinersen, provided that the last dose was received >= 90 days prior to screening) or 2.) Olesoxime (provided that the last dose was received <= 12 months and >= 90 days prior to screening) or 3.) AVXS-101 (provided that the time of treatment was >= 12 months prior to screening)
  • Adequately recovered from any acute illness at the time of screening and considered well enough to participate in the opinion of the Investigator
  • For women of childbearing potential: negative blood pregnancy test at screening, agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, and agreement to refrain from donating eggs for at least 28 days after the final dose of study drug
  • For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures and agreement to refrain from donating sperm
  • For participants aged 2 years or younger at screening: 1.) Parent or caregiver of participant is willing to consider nasogastric, naso-jejunal or gastrostomy tube placement, as recommended by the Investigator, during the study to maintain safe hydration, nutrition and treatment delivery; 2.) Parent or caregiver of participant is willing to consider the use of non-invasive ventilation, as recommended by the Investigator during the study

Exclusion Criteria:

  • Inability to meet study requirements
  • Concomitant participation in any investigational drug or device study
  • With the exception of studies of olesoxime, AVXS-101, or nusinersen: 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
  • Any history of gene or cell therapy, with the exception of AVXS-101
  • Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular system diseases as considered to be clinically significant by the Investigator
  • Inadequate venous or capillary blood access for the study procedures, in the opinion of the Investigator
  • For patients aged < 2 years, hospitalization for a pulmonary event within 2 months prior to screening and pulmonary function not fully recovered at the time of screening
  • Lactating women
  • Suspicion of regular consumption of drugs of abuse
  • For adults and adolescents only, positive urine test for drugs of abuse or alcohol at screening or Day -1 visit
  • Presence of clinically significant electrocardiogram (ECG) abnormalities before study drug administration from average of triplicate measurement or cardiovascular disease
  • History of malignancy if not considered cured
  • For participants aged > 6 years, significant risk for suicidal behavior, in the opinion of the Investigator as assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS)
  • Any major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration
  • Recently initiated treatment for spinal muscular atrophy (within <6 weeks prior to enrollment) with oral salbutamol or another beta 2-adrenergic agonist taken orally
  • Any prior use of chloroquine, hydroxychloroquine, retigabin, vigabatrin or thioridazine, is not allowed
  • Ascertained or presumptive hypersensitivity (e.g., anaphylactic reaction) to risdiplam or to the constituents of its formulation
  • Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would, in the opinion of the Investigator, pose an unacceptable risk to the participant in this study
  • Recent history (less than one year) of ophthalmological diseases
  • Any prior use of an inhibitor or inducer of FMO1 or FMO3 taken within 2 weeks (or within 5 elimination half-lives, whichever is longer) prior to dosing
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03032172


Locations
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Sponsors and Collaborators
Hoffmann-La Roche
Investigators
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Study Director: Clinical Trials Hoffmann-La Roche
More Information
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Additional Information:

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Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03032172    
Other Study ID Numbers: BP39054
2016-004184-39 ( EudraCT Number )
First Posted: January 26, 2017    Key Record Dates
Last Update Posted: April 24, 2023
Last Verified: April 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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/members/ourmembers/). 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).

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Spinal Cord Diseases
 Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Risdiplam
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs