A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (Firefish)

Description

Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic, pharmacodynamics, and efficacy of RO7034067 in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate RO7034067 for 24-months at the dose selected in Part 1.

Study Start Date

December, 24 2016

Estimated Completion Date

July 2020

Interventions

  • Drug: RO7034067

Study ID

Hoffmann-La Roche -- BP39056

Status

Recruiting

Trial ID

NCT02913482

Study Type

Interventional

Trial Phase

Phase 2

Enrollment Quota

48

Sponsor

Hoffmann-La Roche

Inclusion Criteria

  • Clinical history, signs or symptoms attributable to Type 1 SMA with onset after 28 days but prior to the age of 3 months
  • Gestational age of 37 to 42 weeks
  • Confirmed diagnosis of 5q-autosomal recessive SMA
  • Participants has two survival motor neuron 2 (SMN2) gene copies, as confirmed by central testing
  • Body weight greater than or equal to (>=) third percentile for age, using appropriate country-specific guidelines
  • Receiving adequate nutrition and hydration (with or without gastrostomy) at the time of screening, in the opinion of the Investigator
  • Adequately recovered from any acute illness at the time of screening and considered well-enough to participate in the opinion of the Investigator

Exclusion Criteria

  • Concomitant or previous participation in any investigational drug or device study within 90 days prior to screening or 5 half-lives, whichever is longer
  • Concomitant or previous participation in a SMN2 targeting antisense oligonucleotide or SMN2 splicing modifier or gene therapy study
  • Any history of cell therapy
  • Hospitalization for pulmonary event within the last 2 months, or planned at the time of screening
  • Presence of clinically relevant electrocardiogram (ECG) abnormalities before study drug administration
  • Unstable gastrointestinal, renal, hepatic, endocrine or cardiovascular system diseases
  • Participants requiring invasive ventilation or tracheostomy
  • Participants requiring awake non-invasive ventilation or with awake hypoxemia (arterial oxygen saturation less than [<] 95 percent [%]) with or without ventilator support
  • Participants with a history of respiratory failure or severe pneumonia, and have not fully recovered their pulmonary function at the time of screening
  • Multiple or fixed contractures and/or hip subluxation or dislocation at birth
  • Presence of non-SMA related concurrent syndromes or diseases
  • 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
  • Any inhibitor of cytochrome P450 (CYP) 3A4 and/or any Organic Cation Transporter 2 (OCT-2) and multidrug and toxin extrusion (MATE) substrates taken within 2 weeks and/or any inducer of CYP3A4 taken within 4 weeks (or within 5-times the elimination half-life, whichever is longer) prior to dosing or participants (and the mother, if breastfeeding the infant) taking any nutrients known to modulate CYP3A activity and any known flavin containing monooxygenase (FMO) 1 or FMO3 inhibitors or substrates
  • Prior use, anticipated need for hydroxychloroquine, vigabatrin, retigabine, or any other drug known to cause retinal toxicity during the study
  • Recent history (less than 6 months) of ophthalmic disease that would interfere with the conduct of the study as assessed by an ophthalmologist

Gender

All

Ages

1 Month to 7 Months

Accepts Healthy Volunteers

No

Study Locations and Contact Information (7)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles None None None
Columbia University Medical Center The Neurological Institute of New York - New York, New York 181.2 miles None None None
Columbia University Medical Center The Neurological Institute of New York - New York, New York 181.2 miles None None None
Columbia University Medical Center The Neurological Institute of New York - New York, New York 181.2 miles None None None
Columbia University Medical Center The Neurological Institute of New York - New York, New York 181.2 miles None None None
Columbia University Medical Center The Neurological Institute of New York - New York, New York 181.2 miles None None None
Stanford University Medical Center - Palo Alto, California 2,694.6 miles None None None

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