tadefgrobep alfa for Oppenheim Disease

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Oppenheim Disease+5 More
tadefgrobep alfa - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This trial will study if adding taldefgrobep alfa to current treatments for spinal muscular atrophy (SMA) is effective and safe.

Eligible Conditions
  • Oppenheim Disease
  • Spinal Muscular Atrophy (SMA)
  • SMA

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 0 Secondary · Reporting Duration: Baseline to Week 48

Baseline to Week 48
Efficacy of taldefgrobep alfa compared to placebo in change in the 32 item Motor Function Measure (MFM-32) total score

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

2 Treatment Groups

taldefgrobep alfa
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

225 Total Participants · 2 Treatment Groups

Primary Treatment: tadefgrobep alfa · Has Placebo Group · Phase 3

taldefgrobep alfa
Drug
Experimental Group · 1 Intervention: tadefgrobep alfa · Intervention Types: Drug
PlaceboPlaceboComparator Group · 2 Interventions: tadefgrobep alfa, Placebo · Intervention Types: Drug, Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: baseline to week 48

Who is running the clinical trial?

Biohaven Pharmaceuticals, Inc.Lead Sponsor
46 Previous Clinical Trials
31,964 Total Patients Enrolled
Lindsey Lair, MDStudy DirectorBiohaven Pharmaceuticals, Inc.

Eligibility Criteria

Age < 65 · All Participants · 4 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have spinal muscular atrophy (SMA) and you have a positive genetic test for the SMA gene.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: November 15th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

How old are they?
18 - 65100.0%
What site did they apply to?
Rare Disease Research100.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%