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Enzyme Replacement Therapy
Fabrazyme for Fabry Disease
Phase 4
Recruiting
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
Mothers must be enrolled in the Fabry Registry and receiving Fabrazyme while lactating
Infants must be born to a mother who is receiving Fabrazyme during lactation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up months 1, 2, 3, 6, 12, 18 and month 24
Awards & highlights
Study Summary
This trial will last for 2 years, and both mother and infant must be present for full participation.
Who is the study for?
This trial is for breastfeeding mothers with Fabry Disease who are on Fabrazyme treatment and enrolled in the Fabry Registry. Their infants, fed with breast milk, can also participate if consent is given. Mothers or infants cannot join if the mother has taken any investigational drug within the last 30 days.Check my eligibility
What is being tested?
The study examines how treatment with agalsidase beta (Fabrazyme) affects lactating mothers and their infants over a period of up to two years. It tracks both mother's adherence to treatment and infant development.See study design
What are the potential side effects?
While specific side effects aren't listed here, treatments like agalsidase beta may cause allergic reactions, fever, chills, nausea, or discomfort at injection sites among other potential side effects.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am a mother with Fabry disease, enrolled in the Fabry Registry and taking Fabrazyme while breastfeeding.
Select...
My mother was on Fabrazyme while breastfeeding me.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ months 1, 2, 3, 6, 12, 18 and month 24
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~months 1, 2, 3, 6, 12, 18 and month 24
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Determination of pharmacokinetic parameters of αGAL in breast milk: AUC0-2h
Determination of pharmacokinetic parameters of αGAL in breast milk: Cmax
Determination of pharmacokinetic parameters of αGAL in breast milk: lactation clearance
+7 moreSide effects data
From 2005 Phase 4 trial • 67 Patients • NCT0008149756%
Nasopharyngitis
51%
Chills
51%
Cough
49%
Paraesthesia
44%
Pyrexia
41%
Back pain
41%
Oedema peripheral
41%
Upper respiratory tract infection
41%
Dizziness
41%
Headache
36%
Fatigue
36%
Pain in extremity
33%
Pharyngolaryngeal pain
33%
Nausea
33%
Vomiting
31%
Diarrhoea
31%
Abdominal pain
31%
Hypoaesthesia
28%
Pain
28%
Nasal congestion
26%
Adverse event
23%
Arthralgia
23%
Blood pressure increased
23%
Influenza
21%
Muscle spasms
21%
Procedural pain
21%
Myalgia
21%
Lower respiratory tract infection
18%
Excoriation
18%
Sinus congestion
18%
Pruritus
15%
Ear pain
15%
Bronchitis
15%
Rhinorrhoea
15%
Rash
15%
Toothache
15%
Renal impairment
15%
Blood creatinine increased
13%
Stomach discomfort
13%
Viral upper respiratory tract infection
13%
Dyspepsia
13%
Fungal infection
13%
Urinary tract infection
13%
Contusion
13%
Anaemia
13%
Abdominal pain lower
13%
Chest pain
13%
Feeling cold
13%
Sinusitis
13%
Fall
13%
Respiratory tract congestion
13%
Hypertension
13%
Tachycardia
13%
Tinnitus
10%
Anxiety
10%
Insomnia
10%
Tremor
10%
Hypoacusis
10%
Tooth abscess
10%
Depression
10%
Viral infection
10%
Bundle branch block right
10%
Palpitations
10%
Vertigo
10%
Abdominal pain upper
10%
Asthenia
10%
Post procedural nausea
10%
Blood bicarbonate decreased
10%
Blood calcium decreased
10%
Hypokalaemia
10%
Dyspnoea
8%
Constipation
8%
Epistaxis
8%
Hearing impaired
8%
Flatulence
8%
Chest discomfort
8%
Dry skin
8%
Dermatitis contact
8%
Urticaria
8%
Electrocardiogram ST segment abnormal
8%
Cholelithiasis
8%
Supraventricular extrasystoles
8%
Ventricular hypertrophy
8%
Influenza like illness
8%
Body temperature increased
8%
Cardiac murmur
8%
Haemoglobin decreased
8%
Heart rate increased
8%
Gout
8%
Lethargy
8%
Dysuria
8%
Wheezing
8%
Hypotension
8%
Skin disorder
8%
Pitting oedema
8%
Neck pain
8%
Rhinitis allergic
5%
Bradycardia
5%
Gastroenteritis viral
5%
Gastroenteritis
5%
Flank pain
5%
Depressed mood
5%
Dental caries
5%
Pain in jaw
5%
Skin laceration
5%
Thermal burn
5%
Chest wall pain
5%
Sciatica
5%
Rash erythematous
5%
Blister
5%
Psoriasis
5%
Arthropod bite
5%
Seasonal allergy
5%
Sunburn
5%
Red blood cell count decreased
5%
Anorexia
5%
Throat tightness
5%
Eyelid oedema
5%
Retinopathy
5%
Lymphadenopathy
5%
Sinus bradycardia
5%
Pallor
5%
Ventricular wall thickening
5%
Binocular eye movement disorder
5%
Gastritis
5%
Gastrooesophageal reflux disease
5%
Gingival pain
5%
Gingival infection
5%
Localised infection
5%
Pharyngitis streptococcal
5%
Postoperative infection
5%
Post procedural complication
5%
Procedural complication
5%
Blood chloride increased
5%
Blood glucose increased
5%
International normalised ratio increased
5%
Decreased appetite
5%
Skin ulcer
5%
Muscle twitching
5%
Muscular weakness
5%
Burning sensation
5%
Carpal tunnel syndrome
5%
Coordination abnormal
5%
Panic attack
5%
Renal failure chronic
5%
Productive cough
5%
Angiokeratoma
5%
Decubitus ulcer
5%
Erythema
5%
Pruritus allergic
5%
Hot flush
5%
Lymphoedema
5%
Peripheral coldness
5%
Heart rate irregular
5%
Syncope
5%
Micturition disorder
5%
Asthma
5%
Pruritus generalised
5%
Blood triglycerides increased
5%
Blood urea increased
5%
Flushing
5%
Cellulitis
3%
Tendonitis
3%
Meniere's disease
3%
Atrioventricular block first degree
3%
Eye discharge
3%
Blepharitis
3%
Paranasal sinus hypersecretion
3%
Wound secretion
3%
Traumatic haematoma
3%
Abdominal discomfort
3%
Haemorrhoids
3%
Catheter site related reaction
3%
Operative haemorrhage
3%
Bundle branch block left
3%
Faecal incontinence
3%
Phlebitis
3%
Ventricular extrasystoles
3%
Peritonitis bacterial
3%
Ear canal erythema
3%
Ear congestion
3%
Abdominal rigidity
3%
Hypoaesthesia oral
3%
Deafness
3%
Somnolence
3%
Dysphagia
3%
Chalazion
3%
Hand-arm vibration syndrome
3%
Anhidrosis
3%
Confusional state
3%
Acute myocardial infarction
3%
Otorrhoea
3%
Cerumen impaction
3%
Blepharospasm
3%
Peptic ulcer
3%
Hypersensitivity
3%
Gastric disorder
3%
Axillary pain
3%
Bronchitis acute
3%
Cataract
3%
Osteopenia
3%
Mood altered
3%
Faeces pale
3%
Haematochezia
3%
Tooth infection
3%
Groin pain
3%
Vaccination complication
3%
Tooth fracture
3%
Candidiasis
3%
Open wound
3%
Breast discomfort
3%
Nipple pain
3%
Bone pain
3%
Upper respiratory tract congestion
3%
Throat irritation
3%
Dermatitis bullous
3%
Cold sweat
3%
Arterial stenosis
3%
Feeling hot
3%
Conjunctival hyperaemia
3%
Defaecation urgency
3%
Upper respiratory tract infection bacterial
3%
Dilatation atrial
3%
Stomatitis
3%
Feeling hot and cold
3%
Back injury
3%
Electrocardiogram ST-T segment abnormal
3%
Electrocardiogram T wave abnormal
3%
Electrocardiogram T wave inversion
3%
Electrocardiogram abnormal
3%
Glucose urine present
3%
Prothrombin time ratio increased
3%
Cardiomegaly
3%
Colonic polyp
3%
Suprapubic pain
3%
Corneal abrasion
3%
Red blood cells urine positive
3%
Arrhythmia
3%
Dry eye
3%
Retching
3%
Weight decreased
3%
Conjunctival oedema
3%
Conjunctivitis
3%
Atrioventricular block second degree
3%
Cardiac failure congestive
3%
Diplopia
3%
Eye pain
3%
Ocular hyperaemia
3%
Ocular hypertension
3%
Myocardial infarction
3%
Myocardial ischaemia
3%
Pneumonia
3%
Sepsis
3%
Streptococcal infection
3%
Femoral neck fracture
3%
Implantable defibrillator malfunction
3%
Medical device complication
3%
Procedural hypertension
3%
Liver function test abnormal
3%
Dehydration
3%
Osteoarthritis
3%
Uterine prolapse
3%
Pneumothorax
3%
Sleep apnoea syndrome
3%
Nephrogenic anaemia
3%
Scleral haemorrhage
3%
Mitral valve incompetence
3%
Mitral valve prolapse
3%
Tachyarrhythmia
3%
Wolff-Parkinson-White syndrome
3%
Abdominal distension
3%
Hyperparathyroidism
3%
Dry mouth
3%
Gastrointestinal disorder
3%
Lip dry
3%
Mouth haemorrhage
3%
Oesophagitis
3%
Uvulitis
3%
Catheter site haematoma
3%
Gait disturbance
3%
Injection site thrombosis
3%
Malaise
3%
Thirst
3%
Conjunctivitis infective
3%
Kidney infection
3%
Nail tinea
3%
Oral candidiasis
3%
Pharyngitis
3%
Prostate infection
3%
Rectal abscess
3%
Respiratory tract infection
3%
Rash pruritic
3%
Seborrhoeic dermatitis
3%
Skin discolouration
3%
Electric shock
3%
Injury
3%
Joint injury
3%
Joint sprain
3%
Limb injury
3%
Muscle strain
3%
Post procedural dizziness
3%
Blood bilirubin increased
3%
Blood cholesterol increased
3%
Blood phosphorus decreased
3%
Blood potassium decreased
3%
Blood potassium increased
3%
Blood pressure decreased
3%
Blood uric acid increased
3%
Cardiac stress test abnormal
3%
Electrocardiogram P wave abnormal
3%
Electrocardiogram PR shortened
3%
Haematocrit decreased
3%
Heart rate decreased
3%
International normalised ratio fluctuation
3%
Platelet count increased
3%
Protein urine present
3%
Right ventricular systolic pressure increased
3%
Acidosis
3%
Hyperkalaemia
3%
Hyperlipidaemia
3%
Hypocalcaemia
3%
Hypomagnesaemia
3%
Oral intake reduced
3%
Skin irritation
3%
Skin lesion
3%
Skin nodule
3%
Intervertebral disc degeneration
3%
Intervertebral disc disorder
3%
Joint hyperextension
3%
Joint swelling
3%
Limb discomfort
3%
Muscle tightness
3%
Osteoporosis
3%
Encephalomalacia
3%
Hyperaesthesia
3%
Memory impairment
3%
Azotaemia
3%
Micturition urgency
3%
Nocturia
3%
Urethral stricture
3%
Urinary incontinence
3%
Breast discharge
3%
Dysmenorrhoea
3%
Penile discharge
3%
Prostatitis
3%
Scrotal angiokeratoma
3%
Allergic cough
3%
Atelectasis
3%
Chronic obstructive pulmonary disease
3%
Dyspnoea exacerbated
3%
Pulmonary hypertension
3%
Rhonchi
3%
Acne
3%
Dermal cyst
3%
Ecchymosis
3%
Hyperhidrosis
3%
Rash maculo-papular
3%
Haematoma
3%
Labile hypertension
3%
Orthostatic hypotension
3%
Raynaud's phenomenon
3%
Steal syndrome
3%
Vascular pseudoaneurysm
3%
Vasculitis
3%
Venous insufficiency
3%
Atrial fibrillation
3%
Ventricular dysfunction
3%
Hyperreflexia
3%
Heart valve insufficiency
3%
Chapped lips
3%
Peritonitis
3%
Renal failure
3%
Urethral meatus stenosis
3%
Dysphonia
3%
Pleural rub
3%
Ventricular tachycardia
3%
Renal failure acute
3%
Hip fracture
3%
Dilatation ventricular
3%
Anal fistula
3%
Cyst rupture
3%
Ear infection
3%
Soft tissue injury
3%
Blood alkaline phosphatase increased
3%
Postoperative wound complication
3%
Intracranial aneurysm
3%
Nerve compression
3%
Buttock pain
3%
Loss of consciousness
3%
Nervousness
3%
Visual acuity reduced
3%
Vision blurred
3%
Blood parathyroid hormone increased
3%
Metabolic acidosis
3%
Rash papular
3%
Dysgeusia
3%
Agitation
3%
Proteinuria
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo/Fabrazyme
Fabrazyme/Fabrazyme
Total
Trial Design
1Treatment groups
Experimental Treatment
Group I: Agalsidase betaExperimental Treatment1 Intervention
Commercially available Fabrazyme treatment at prescribed dose and regimen as determined by their treating physician
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
agalsidase beta
2004
Completed Phase 4
~150
Find a Location
Who is running the clinical trial?
Genzyme, a Sanofi CompanyLead Sponsor
524 Previous Clinical Trials
85,623 Total Patients Enrolled
25 Trials studying Fabry Disease
14,768 Patients Enrolled for Fabry Disease
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am a mother with Fabry disease, enrolled in the Fabry Registry and taking Fabrazyme while breastfeeding.My child's participation in this study has been approved by me or their legal guardian.My mother was on Fabrazyme while breastfeeding me.I have signed the consent form to participate in this study.
Research Study Groups:
This trial has the following groups:- Group 1: Agalsidase beta
Awards:
This trial has 3 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- Drug Has Already Been Approved - The FDA has already approved this drug, and is just seeking more data.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Has the Federal Drug Administration ratified agalsidase beta for usage?
"Clinical data supports the safety of agalsidase beta, so it was awarded a score of 3. This drug has been approved by authorities and is now in its fourth phase of testing."
Answered by AI
How many participants are eligible to join this trial?
"Affirmative. Clinicaltrials.gov reflects that this clinical trial, initially launched on August 30th 2006, is actively recruiting for 20 participants from two separate medical sites. It was most recently updated on May 4th 2022."
Answered by AI
How has agalsidase beta been studied in other research studies?
"Back in 2006, agalsidase beta was first studied at 03Waldek. Since then, 13 trials have been conducted with 2 of these currently recruiting participants from the Fairfax area."
Answered by AI
Is this research endeavor presently receiving volunteers?
"According to the latest updates on clinicaltrials.gov, this experiment is still recruiting patients for participation. The trial was initially listed in August 2006 and most recently modified in May of 2022."
Answered by AI
Is this an original clinical trial or has it been conducted previously?
"Research on agalsidase beta dates back to 2006 when the first clinical trial was sponsored by Genzyme, a subsidiary of Sanofi. After this initial test involving 20 participants at that time, it received its Phase 4 drug approval in subsequent years. Currently there are 2 active trials with sites located across 3 urban hubs and spanning 4 nations."
Answered by AI
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