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Enzyme Replacement Therapy

Avalglucosidase Alfa for Pompe Disease (NEO-EXT Trial)

Phase 2
Waitlist Available
Research Sponsored by Genzyme, a Sanofi Company
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 (day 1) and weeks 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 52, 78, 104, 130, 156, 182, 208, 234, 260, 286, 312, 338, 364, 390, 416 and 442
Awards & highlights

NEO-EXT Trial Summary

This trial is studying the long-term safety and how well avalglucosidase alfa works in treating patients with Pompe disease.

Eligible Conditions
  • Pompe Disease

NEO-EXT Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (day 1) and weeks 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 52, 78, 104, 130, 156, 182, 208, 234, 260, 286, 312, 338, 364, 390, 416 and 442
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 1) and weeks 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 52, 78, 104, 130, 156, 182, 208, 234, 260, 286, 312, 338, 364, 390, 416 and 442 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Apparent Total Body Clearance Steady-State (CLss) of Avalglucosidase Alfa
Apparent Volume of Distribution Steady-State (Vss) of Avalglucosidase Alfa
Area Under the Plasma Concentration Versus Time Curve From Time Zero to the Real Time (AUClast) of Avalglucosidase Alfa
+16 more
Secondary outcome measures
Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442
Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442
Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442
+4 more

Side effects data

From 2022 Phase 2 trial • 19 Patients • NCT02032524
100%
Muscle Spasms
100%
Diarrhoea
75%
Ear Pain
75%
Dizziness
75%
Dyspnoea
75%
Myalgia
75%
Pain In Extremity
75%
Nausea
75%
Rash
75%
Contusion
75%
Fall
75%
Headache
50%
Neuralgia
50%
Cough
50%
Rhinorrhoea
50%
Vomiting
50%
Nasopharyngitis
50%
Oropharyngeal Pain
50%
Pyrexia
50%
Urinary Tract Infection
50%
Hypertension
50%
Ear Infection
50%
Oral Herpes
50%
Insomnia
50%
Palpitations
50%
Upper Respiratory Tract Infection
50%
Gastrooesophageal Reflux Disease
50%
Fatigue
50%
Post-Traumatic Pain
50%
Arthralgia
50%
Back Pain
25%
Sinusitis
25%
Furuncle
25%
Cataract
25%
Abdominal Distension
25%
Anaemia
25%
Eye Pruritus
25%
Application Site Reaction
25%
Accidental Overdose
25%
Bone Contusion
25%
Dry Eye
25%
Muscular Weakness
25%
Irritable Bowel Syndrome
25%
Epistaxis
25%
Sleep Apnoea Syndrome
25%
Hyperhidrosis
25%
Hot Flush
25%
Abdominal Discomfort
25%
Dyspepsia
25%
Covid-19
25%
Influenza
25%
Scratch
25%
Flank Pain
25%
Arteritis
25%
Eczema
25%
Erythema
25%
Infusion Site Extravasation
25%
Procedural Pain
25%
Pulmonary Contusion
25%
Body Temperature Increased
25%
Dysaesthesia
25%
Thermal Burn
25%
Traumatic Haematoma
25%
Peripheral Artery Stenosis
25%
Viral Upper Respiratory Tract Infection
25%
Lymphadenopathy
25%
Nasal Congestion
25%
Flushing
25%
Gastroenteritis
25%
Skin Laceration
25%
Ear Discomfort
25%
Peripheral Swelling
25%
Haemoglobin Decreased
25%
Rectal Haemorrhage
25%
Chest Discomfort
25%
Myocardial Ischaemia
25%
Lymphopenia
25%
Swelling Of Eyelid
25%
Herpes Zoster
25%
Infusion Site Pain
25%
Infusion Site Rash
25%
Asthenia
25%
Infusion Site Oedema
25%
Catheter Site Erythema
25%
Dysphagia
25%
Lower Respiratory Tract Infection
25%
Non-Cardiac Chest Pain
25%
Seasonal Allergy
25%
Post Procedural Infection
25%
Muscle Strain
25%
Pruritus
25%
Speech Disorder
25%
Infusion Related Reaction
25%
Jaw Clicking
25%
Tendonitis
25%
Melanocytic Naevus
25%
Syncope
25%
Rosacea
25%
Haematoma
25%
Hypoaesthesia
25%
Cervical Radiculopathy
25%
Sciatica
25%
Skin Lesion
25%
Sinus Congestion
25%
Respiratory Distress
25%
Ischaemic Stroke
25%
Anxiety
25%
Intraocular Pressure Increased
25%
Tooth Abscess
25%
Tooth Infection
25%
Weight Decreased
25%
Abdominal Pain
25%
Constipation
25%
Viral Tonsillitis
25%
Arthropod Bite
25%
Paraesthesia
25%
Iliotibial Band Syndrome
25%
Gout
25%
Oral Mucosal Blistering
25%
Peptic Ulcer
25%
Salivary Hypersecretion
25%
Toothache
25%
Chest Pain
25%
Chills
25%
Facial Pain
25%
Feeling Hot
25%
Injection Site Swelling
25%
Infusion Site Swelling
25%
Depression
25%
Pulmonary Congestion
25%
Choking
25%
Hypoventilation
25%
Somnolence
100%
80%
60%
40%
20%
0%
Study treatment Arm
Group 1: Avalglucosidase Alfa 5 mg/kg
Group 1: Avalglucosidase Alfa 20 mg/kg
Group 2: Avalglucosidase Alfa 5 mg/kg
Group 1: Avalglucosidase Alfa 10 mg/kg
Group 2: Avalglucosidase Alfa 10 mg/kg
Group 2: Avalglucosidase Alfa 20 mg/kg

NEO-EXT Trial Design

1Treatment groups
Experimental Treatment
Group I: Avalglucosidase AlfaExperimental Treatment1 Intervention
administered intravenously every 2 weeks
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Avalglucosidase Alfa
2014
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

Genzyme, a Sanofi CompanyLead Sponsor
524 Previous Clinical Trials
85,610 Total Patients Enrolled
Clinical Sciences & OperationsStudy DirectorSanofi
862 Previous Clinical Trials
2,019,819 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Avalglucosidase Alfa Extension Study
2014. "Avalglucosidase Alfa Extension Study". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02032524.
~2 spots leftby Apr 2025
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