Title: Clinical Study of Spinal Muscular Atrophy (PNCR SMA study)
Protocol ID: 31140
NCT: 00443066
PI: Dr. John Day
Study coordinator: Shirley Paulose, 650-724-3792, spaulose@stanford.edu
Sponsor: Columbia University, Collaborator: Spinal Muscular Atrophy Foundation
Purpose: To study the natural history of Spinal Muscular Atrophy to help with clinical trials in future.
Status: Recruiting in clinic
Title: A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients with Spinal Muscular Atrophy (SMA)
Protocol ID: 35918
NCT: 02644668
Sponsor: Cytokinetics, Inc.
PI: Dr. Carolina Tesi Rocha
Study coordinator: Shirley Paulose, 650-724-3792, spaulose@stanford.edu
Pupose: To study the pharmacodynamics, safety, tolerability and pharmacokinetics of the study medication, CK-2127107, in patients with SMA
Status: Recruiting
Recruitment Criteria Notes: Hammersmith score 10-54. Ambulatory patients, once having achieved a standing position independently, must be able to complete at least one lap in the 6-minute walk test (at least 50 meters) within 5 minutes without assistance. For non-ambulatory patients (defined as individuals who are unable to stay in a standing position independently and unable to take any steps with personal assistance or assistive devices; effectively requiring a wheelchair for all mobility needs) must be able to tolerate an upright sitting position, with support, continuously for 3 hours
Title: An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 (aka SHINE)
Protocol ID: 36172
NCT: 02594124
Sponsor: Ionis Pharmaceuticals, Inc.
PI: Dr. John Day
Study coordinator: Shirley Paulose, 650-724-3792, spaulose@stanford.edu
Purpose: To evaluate the long-term safety and tolerability, and to examine the cerebrospinal fluid pharmacokinetics of ISIS 396443 administered intrathecally to patients with SMA who previously participated in investigational studies of ISIS 396443.
Status: Active, but closed to enrollment
Title: Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non-ambulatory Patients with Spinal Muscular Atrophy (aka STRONG)
Protocol: 38893
NCT: 03381729
Sponsor: Avexis Pharmaceuticals, Inc.
PI: Dr. John Day
Study Coordinator: Shirley Paulose, 650-724-3792, spaulose@stanford.edu
Purpose: It is a gene therapy study to compare doses of the investigational product for a group of subjects with Spinal Muscular Atrophy
Status: Open for recruitment
Recruitment Criteria Notes: Children under 60 months with SMA, onset at less than 12 months of age.
Title: A Two Part Seamless, Open-Label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of R07034067 in Infants With Type 1 Spinal Muscular Atrophy (aka FIREFISH)
Protocol: 40452
NCT: 02913482
Sponsor: Roche Pharmaceuticals, Inc.
PI: Dr. John Day
Study Coordinator: Lesly Welsh, 650-497-3079, lwelsh@stanford.edu
Purpose: it is to assess an investigational product for spinal muscular atrophy Type 1
Status: Open for recruitment
Recruitment Criteria Notes: Children less than 7 months with SMA symptom onset between 1 and 3 months.
Title: AVXS-101-CL-303: Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN 2 Copies Phase I, Open-Label, Dose Comparison Study of AVXS-101 by Intravenous Infusion (STRIVE) GENE TRANSFER.
Protocol: 40902
NCT: N/A
Sponsor: AveXis Pharmaceuticals, Inc.
PI: Dr. John Day
Study Coordinator: Shirley Paulose, 650-724-3792, spaulose@stanford.edu
Purpose: It is a gene therapy study in subjects with Spinal Muscular Atrophy Type 1.
Status: Will be open for recruitment soon.
Recruitment Criteria Notes: Children with SMA and 1-2 copies of SMN2 aged less than 6 months.
Title: Phase 2, Two Part Seamless Multi-Center Randomized Placebo-Controlled, Double-Blind Study To Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of R07034067 in Type 2 and 3 SMA (SUNFISH)
Protocol: 44102
NCT: 02908685
Sponsor: Roche
PI: Dr. John Day
Study Coordinator: Lesly Welsh, 650-497-3079, lwelsh@stanford.edu
Purpose:
PART 1: To evaluate the safety, tolerability, PK and PD of RO7034067
in patients with Type 2 and Type 3 (ambulant or non-ambulant) spinal
muscular atrophy (SMA), and to select the dose for Part 2 of the
study.
PART 2: To evaluate efficacy of RO7034067 compared to placebo in
terms of motor function in Type 2 and non-ambulant Type 3 SMA
patients, as assessed by the change from baseline in the total score
of the motor function measure (MFM) at 12 months.
Status: IRB in progress
Title: A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 (aka SPRINT)
Protocol:
NCT:
Sponsor: AveXis
PI: Dr. John Day
Study Coordinator: Carolyn McLaughlin, 650-206-3178, cjmclaug@stanford.edu
Purpose:
Status: IRB in progress
