Clinical trials: Anti-myostatin
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- ONYX (NCT05626855)
- Testing
- Apitegromab
- Where
- 49 sites in United States, Belgium and 7 more
- Volunteers
- 238 people
- Sponsor
- Scholar Rock, Inc.
Spinal Muscular Atrophy, SMA
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones…
- Conditions
- Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3
- Trial name
- OPAL (NCT07047144)
- Testing
- Apitegromab, Nusinersen, Risdiplam
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 52 people
- Sponsor
- Scholar Rock, Inc.
Advanced Cancer
A Dose-Escalation Study in Participants With Advanced Cancer
This study is a multicenter, open-label, dose-escalation phase 1 study of intravenous (IV) LY2495655 in participants with advanced cancer.
- Trial name
- NCT01524224
- Testing
- LY2495655
- Where
- 7 sites in United States
- Volunteers
- 29 people
- Sponsor
- Eli Lilly and Company
End Stage Renal Disease, Kidney Disease
Effects of PINTA 745 in End Stage Renal Disease (ESRD) Patients Who Require Hemodialysis and Have Protein Energy Wasting
This is a pilot study to assess the safety, pharmacokinetics and effectiveness of PINTA 745 or placebo in treating protein energy wasting (PEW) in patients receiving maintenance hemodialysis (MHD).
- Conditions
- End Stage Renal Disease, Kidney Disease, Protein Energy Wasting
- Trial name
- NCT01958970
- Testing
- PINTA 745
- Where
- 6 sites in United States
- Volunteers
- 51 people
- Sponsor
- Pinta Biotherapeutics
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.