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Corticosteroid

Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) (LELANTOS-1 Trial)

Phase 3
Waitlist Available
Research Sponsored by FibroGen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, week 52
Awards & highlights
Pivotal Trial

Summary

This trial tests pamrevlumab with steroids in patients aged 12+ who have severe Duchenne muscular dystrophy and can't walk. The drug aims to reduce muscle damage by blocking a harmful protein.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52
Secondary study objectives
Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI)
Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry
Change From Baseline in Percent Predicted Peak Expiratory Flow (ppPEF) at Week 52, Assessed by Spirometry
+1 more

Side effects data

From 2017 Phase 2 trial • 160 Patients • NCT01890265
30%
Respiratory tract infection
28%
Cough
26%
Dyspnoea
20%
Fatigue
20%
Idiopathic pulmonary fibrosis
20%
Urinary tract infection
18%
Nasopharyngitis
16%
Diarrhoea
16%
Sinusitis
14%
Nausea
10%
Arthralgia
10%
Back pain
8%
Pain
8%
Headache
8%
Upper-airway cough syndrome
8%
Abdominal pain upper
8%
Chest pain
8%
Oedema peripheral
8%
Dizziness
8%
Insomnia
6%
Sleep apnoea syndrome
6%
Myalgia
6%
Chest discomfort
6%
Heart sounds abnormal
6%
Decreased appetite
6%
Musculoskeletal pain
6%
Anxiety
6%
Pulmonary hypertension
6%
Sinus congestion
6%
Flushing
6%
Hypertension
4%
Bronchitis
4%
Constipation
4%
Interstitial lung disease
4%
Pulmonary embolism
4%
Contusion
2%
Musculoskeletal chest pain
2%
Autoimmune haemolytic anaemia
2%
Immune thrombocytopenic purpura
2%
Non-cardiac chest pain
2%
Acute respiratory failure
2%
Respiratory failure
2%
Angina pectoris
2%
Sepsis
2%
Femoral neck fracture
2%
Humerus fracture
2%
Squamous cell carcinoma of the tongue
2%
Peripheral ischaemia
2%
Throat irritation
100%
80%
60%
40%
20%
0%
Study treatment Arm
Pamrevlumab
Placebo
Sub-Study: Pamrevlumab+Pirfenidone
Sub-Study: Placebo+Pirfenidone
Sub-Study: Pamrevlumab+Nintedanib
Sub-Study: Placebo+Nintedanib

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: PamrevlumabExperimental Treatment2 Interventions
Pamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Group II: PlaceboPlacebo Group2 Interventions
Matching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Pamrevlumab
2013
Completed Phase 3
~210
Corticosteroids
2003
Completed Phase 4
~8270

Find a Location

Who is running the clinical trial?

FibroGenLead Sponsor
58 Previous Clinical Trials
15,245 Total Patients Enrolled

Media Library

Corticosteroids (Corticosteroid) Clinical Trial Eligibility Overview. Trial Name: NCT04371666 — Phase 3
Duchenne Muscular Dystrophy Research Study Groups: Placebo, Pamrevlumab
Duchenne Muscular Dystrophy Clinical Trial 2023: Corticosteroids Highlights & Side Effects. Trial Name: NCT04371666 — Phase 3
Corticosteroids (Corticosteroid) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04371666 — Phase 3
~18 spots leftby Dec 2025