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Gene Therapy

Subjects who received AT-GTX-501 gene transfer for Batten Disease

N/A
Waitlist Available
Research Sponsored by Amicus Therapeutics
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 up to 3 years
Awards & highlights
No Placebo-Only Group

Summary

This is a long-term safety and efficacy study in subjects with CLN6 Batten disease who previously received a single intrathecal administration of AT-GTX-501.

Eligible Conditions
  • Batten Disease
  • Neuronal Ceroid Lipofuscinosis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Long-term safety assessment based on Adverse Events (AEs)
Secondary study objectives
Hamburg Scale

Side effects data

From 2021 Phase 1 & 2 trial • 13 Patients • NCT02725580
69%
Upper respiratory tract infection
38%
Vomiting
31%
Haematuria
31%
Constipation
31%
Viral infection
31%
Seizure
31%
Insomnia
23%
Rhinorrhoea
23%
Myoclonic epilepsy
23%
Respiratory disorder
23%
Diarrhoea
23%
Back pain
15%
Dyskinesia
15%
Atonic seizures
15%
Myoclonus
15%
Pharyngitis streptococcal
15%
Gastroenteritis viral
15%
Pyrexia
15%
Otitis media
15%
Skin abrasion
15%
Urinary tract infection
15%
Procedural pain
15%
Fall
15%
Abnormal behaviour
15%
Nasal congestion
15%
Generalised tonic-clonic seizure
15%
Cough
15%
Rash
15%
Nasopharyngitis
15%
Dehydration
15%
Tremor
8%
Tonsillar hypertrophy
8%
Hypernatraemia
8%
Abdominal discomfort
8%
Respiratory tract infection viral
8%
Skin laceration
8%
Weight decreased
8%
Decreased appetite
8%
Hallucination
8%
Nephrolithiasis
8%
Pallor
8%
Synovitis
8%
Staring
8%
Alanine aminotransferase increased
8%
Umbilical erythema
8%
Sinusitis
8%
Restlessness
8%
Oxygen saturation decreased
8%
Hypothyroidism
8%
Ear infection
8%
Petit mal epilepsy
8%
Irritability
8%
Dermatitis contact
8%
Failure to thrive
8%
Clavicle fracture
8%
Iron deficiency anaemia
8%
Cerumen impaction
8%
Dysphagia
8%
Impaired gastric emptying
8%
Precocious puberty
8%
Impetigo
8%
Aspartate aminotransferase increased
8%
Blood creatine phosphokinase increased
8%
Flatulence
8%
Gastroenteritis
8%
Pyuria
8%
Vaginal infection
8%
Partial seizures
8%
Ecchymosis
8%
Hyperhidrosis
8%
Bronchitis
8%
Candida infection
8%
Tonsillitis
8%
Humerus fracture
8%
Dystonia
8%
Glycosuria
8%
Sterile pyuria
8%
Dermatitis
8%
Sleep disorder
8%
Proteinuria
8%
Oropharyngeal pain
8%
Pruritus
8%
Hypersensitivity
8%
Abdominal pain
8%
Leukocytosis
8%
Abdominal pain upper
8%
Muscle spasms
8%
Musculoskeletal stiffness
8%
Epstein-Barr virus infection
8%
Upper limb fracture
8%
Hypophagia
8%
Drooling
8%
Headache
8%
Astigmatism
8%
Gaze palsy
8%
Amylase increased
8%
Fatigue
8%
COVID-19
8%
Gastrointestinal viral infection
8%
Hand-foot-and-mouth disease
8%
Molluscum contagiosum
8%
Viral rash
100%
80%
60%
40%
20%
0%
Study treatment Arm
AT-GTX-501

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Subjects who received AT-GTX-501 gene transferExperimental Treatment1 Intervention
Subjects with CLN6 Batten disease who previously received AT-GTX-501 in the preceding study (Study AT-GTX-501-01).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AT-GTX-501
2016
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor
54 Previous Clinical Trials
2,717 Total Patients Enrolled
Clinical ResearchStudy DirectorAmicus Therapeutics
9 Previous Clinical Trials
606 Total Patients Enrolled
~2 spots leftby Nov 2025
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