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Clinical Trials/NL-OMON49730
NL-OMON49730RecruitingPhase 2

An open-label study to investigate the safety, tolerability, and Pharmacokinetics/Pharmacodynamics of risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophy - JEWELFISH

Hoffmann-La Roche0 sites10 target enrollmentStarted: TBDLast updated:
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Conditions

Trial Snapshot

Phase
Phase 2
Status
Recruiting
Enrollment
10
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional

Eligibility Criteria

Ages
2 to 99 (—)

Inclusion Criteria

  • •1. Males and females 6 months to 60 years of age inclusive (at screening)
  • •2. Confirmed diagnosis of 5q-autosomal recessive SMA, including:
  • •- Genetic confirmation of homozygous deletion or heterozygosity predictive of
  • •loss of function of the SMN1 gene.
  • •- Clinical history, signs, or symptoms attributable to SMA.
  • •3. Previous enrollment in Study BP29420 (Moonfish) with the splicing modifier
  • •RO6885247 or previous treatment with any of the following:
  • •- Nusinersen (defined as having received >=4 doses of nusinersen, provided that
  • •the last dose was received >= 90 days prior to screening)
  • •- Olesoxime (provided that the last dose was received <= 18 months and >= 90 days
  • •prior to screening)
  • •- AVXS-101 (provided that the time of treatment was >= 12 months prior to
  • •4. Able and willing to provide written informed consent and to comply with the
  • •study protocol according to International Conference on Harmonization (ICH) and
  • •local regulations. Alternatively, a legally authorized representative must be
  • •able to give consent for the patient according to ICH and local regulations and
  • •assent must be given whenever possible.
  • •5. Adequately recovered from any acute illness at the time of screening and
  • •considered well enough to participate in the opinion of the Investigator.
  • •6. 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, as defined below:
  • •- Women must remain abstinent (refrain from heterosexual intercourse) or use
  • •two adequate methods of contraception, including at least one method with a
  • •failure rate of < 1% per year, during the treatment period and for at least 28
  • •days after the final dose of study drug. Women must refrain from donating eggs
  • •during this same period.
  • •- A woman is considered to be of childbearing potential if she is
  • •postmenarcheal, has not reached a postmenopausal state (>= 12 continuous months
  • •of amenorrhea with no identified cause other than menopause), and has not
  • •undergone surgical sterilization (removal of ovaries and/or uterus). The
  • •definition of childbearing potential may be adapted for alignment with local
  • •guidelines or regulations.
  • •- Examples of contraceptive methods with a failure rate of < 1% per year
  • •include bilateral tubal ligation, male sterilization, established and proper
  • •use of hormonal contraceptives that inhibit ovulation, hormone-releasing
  • •intrauterine devices, and copper intrauterine devices.
  • •- A vasectomy is a highly effective birth control method provided that the
  • •partner is the sole sexual partner of the woman of child bearing potential
  • •trial participant, and provided the vasectomized partner has received medical
  • •assessment of the surgical success.
  • •- The reliability of sexual abstinence should be evaluated in relation to the
  • •duration of the clinical trial and the preferred and usual lifestyle of the
  • •patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or
  • •post-ovulation methods) and withdrawal are not acceptable methods of
  • •contraception. If required per local guidelines or regulations, locally
  • •recognized acceptable methods of contraception and information about the
  • •reliability of abstinence will be described in the local Informed Co

Exclusion Criteria

  • •1. Inability to meet study requirements.
  • •2. Concomitant participation in any investigational drug or device study.
  • •3. 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.
  • •4. Any history of gene or cell therapy, with the exception of AVXS-101.
  • •5. Unstable gastrointestinal, renal, hepatic, endocrine, or cardiovascular
  • •system diseases as considered to be clinically significant by the Investigator.
  • •6. Inadequate venous or capillary blood access for the study procedures, in the
  • •opinion of the Investigator.
  • •7. 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.
  • •8. Lactating women.
  • •9. Suspicion of regular consumption of drugs of abuse.
  • •10. For adults and adolescents only, i.e., aged > 12 years, positive urine test
  • •for drugs of abuse or alcohol at screening or Day -1 visit.
  • •11. Cardiovascular, blood pressure, and heart rate:
  • •- Adults: Sustained resting systolic blood pressure (SBP) > 140 mmHg or < 80
  • •mmHg, and/or diastolic blood pressure (DBP) > 90 mmHg or <40 mmHg; a resting
  • •heart rate < 45 bpm or > 100 bpm if considered to be clinically significant by
  • •the Investigator.
  • •- Adolescents (12*17 years of age): SBP and/or DBP outside the 95th percentile
  • •for age; resting heart rate < 50 bpm or > 100 bpm if considered to be
  • •clinically significant by the Investigator.
  • •- Children (6*11 years of age): SBP and/or DBP outside the 95th percentile for
  • •age; resting heart rate < 60 bpm or > 120 bpm, if considered to be clinically
  • •significant by the Investigator.
  • •- Children (2*5 years of age): SBP and/or DBP outside the 95th percentile for
  • •age; resting heart rate < 70 bpm or > 140 bpm if considered to be clinically
  • •significant by the Investigator.
  • •- Children (6 months to < 2 years of age): SBP and/or DBP outside the 95th
  • •percentile for age; resting heart rate <70 bpm or > 170 bpm, if considered to
  • •be clinically significant by the Investigator.
  • •12. Presence of clinically significant ECG abnormalities before study drug
  • •administration (e.g., second or third degree AV block, confirmed QTcF >460 msec
  • •for patients aged >= 10 years, or QTcB > 460 ms for children up to age 10 years
  • •(Bazett*s correction is more appropriate in young children) from the average of
  • •triplicate measurements, or cardiovascular disease (e.g., cardiac
  • •insufficiency, coronary artery disease, cardiomyopathy, congestive heart
  • •failure, family history of congenital long QT syndrome, family history of
  • •sudden death) indicating a safety risk for the patient as determined by the
  • •Investigator.
  • •13. History of malignancy if not considered cured.
  • •14. For patients 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).
  • •15. Any major illness within 1 month before the screening examination or any
  • •febrile illness within 1 week prior to screening and up to first dose
  • •administration
  • +3 more not shown

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