Fabry DiseasePhase 3NCT02921620
Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
A Phase 3 study testing PRX-102 in people with Fabry Disease, run by Protalix.
Trial summary
The study will be a randomized, double blind, placebo-controlled study of the safety and efficacy of PRX-102 in ERT naïve male patients randomized 1:1. Patient age will be 14 to 45 years. Read more about this study
- Who is it for?
- Ages 14 to 45 years, men only
- Conditions the study is for
- Fabry Disease
- What is being tested?
- PRX-102
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jul 2017to Jul 2018
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 45 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Males, age 14-45 years, naïve to enzyme replacement therapy (ERT) or off ERT or off chaperone treatment for at least 6 months and negative for anti-PRX-102 antibodies
- A documented diagnosis of Fabry disease: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN)
- eGFR by CKD-EPI > 30 ml/min/1.73 m2
- Moderate to severe gastrointestinal symptoms as defined by:
- Average score of > 175 from at least two Irritable Bowel Symptom Severity Score (IBSSS) Part 1 assessments before randomization.
- Average stool consistency of ≥ 5.5 on the Bristol Stool Form Scale (BSFS) by patient diary during 2 weeks prior to randomization out of the 4 week of screening period and
- ≥ 3 stools a day with a consistency of ≥ 5 on the BSFS during the week before randomization.
- Completed electronic BSFS diary on at least 6 of the 7 days during the week prior to randomization AND at least 11 of the 14 days during the 2 weeks prior to randomization.
Who cannot join
- Patients will be evaluated to rule out other gastrointestinal comorbidity than Fabry disease as responsible for the gastrointestinal symptoms by:
- Medical History for non Fabry gastrointestinal comorbidity ii. Occult blood in stool iii. Stool culture for bacteria and parasites iv. Calprotectin in stool v. Sigmoidoscopy
- Use of any kind of laxatives
- Initiation of anti-diarrheal medications during the screening period
- History of renal dialysis or transplantation
- Use of, or change in dose of, angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) for less than 4 weeks prior to screening
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- Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
- Congestive heart failure NYHA Class IV
- Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
- Known history of hypersensitivity to Gadolinium contrast agent
- Known allergies to ERT
- Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient's compliance with the requirements of the study
About this study
Official title: A Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease Patients
The study will be a randomized, double blind, placebo-controlled study of the safety and efficacy of PRX-102 in ERT naïve male patients randomized 1:1. Patient age will be 14 to 45 years. Patients must have diarrhea defined as ≥ 3 stools a day with an average consistency of ≥ 5.5 on the Bristol Stool Form Scale (BSFS) by patient electronic diary and moderate to severe gastrointestinal symptoms as defined by the Irritable Bowel Symptom Severity Score (IBSSS) Part 1 average > 175 derived from at least two IBSSS assessments during screening period. Patients will receive intravenous infusions of PRX-102 1 mg/kg or placebo every two weeks for 6 months.
- Conditions
- Fabry Disease
- Being tested
- PRX-102, Placebo
- Keywords
- Gastrointestinal symptoms, Diarrhea, Abdominal pain
- Medical subject headings
- Fabry Disease, Diarrhea, Abdominal Pain
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PRX-102Biological
Also called pegunigalsidase alfa, recombinant human alpha galactosidase-A
Intravenous
PlaceboOther
Intravenous
| Study group | Role | What they receive |
|---|---|---|
| PRX-102 | Experimental | PRX-102 infusions every 2 weeks Receives: Biological: PRX-102 |
| Placebo | Placebo comparator | Placebo infusions every 2 weeks Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
IBSSS Part 1
Irritable Bowel Syndrome Severity Score
Measured: Every 2 weeks for 6 months
Secondary outcomes (6)
Stool frequency
from BSFS diary
Measured: After every bowel movement for 6 months
Body Weight
Measured: Every 2 weeks for 6 months
Plasma Lyso-Gb3
Measured: Every 4 weeks for 6 months
Plasma Gb3
Measured: Every 4 weeks for 6 months
Urine Lyso-GB3
Measured: Every 6 weeks for 6 months
Frequency of pain medication use
Measured: Every 2 weeks for 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2016 (estimated)
- Study startJul 2017 (estimated)
- Primary completionJul 2018 (estimated)
- Study completionJul 2018 (estimated)
Why it stopped: Protocol not feasible
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protalix
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Raul Chertkoff, MD, Study director, Protalix Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02921620
- Sponsor's protocol ID
- PB-102-F10
- Status last verified
- Jan 2018
- First submitted
- 26 Sep 2016
- Last updated
- 8 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease Patients
- ClinicalTrials.gov ID
- NCT02921620
- Acronym / sponsor ID
- PB-102-F10
- Conditions
- Fabry Disease
- Keywords
- Gastrointestinal symptoms, Diarrhea, Abdominal pain
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Protocol not feasible
- Enrolment
- 0 participants (actual)
- Lead sponsor
- ProtalixIndustry (pharma, biotech, devices)
- Study start
- Jul 2017 (estimated)
- Primary completion
- Jul 2018 (estimated)
- Study completion
- Jul 2018 (estimated)
- First posted
- 3 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study will be a randomized, double blind, placebo-controlled study of the safety and efficacy of PRX-102 in ERT naïve male patients randomized 1:1. Patient age will be 14 to 45 years. Patients must have diarrhea defined as ≥ 3 stools a day with an average consistency of ≥ 5.5 on the Bristol Stool Form Scale (BSFS) by patient electronic diary and moderate to severe gastrointestinal symptoms as defined by the Irritable Bowel Symptom Severity Score (IBSSS) Part 1 average > 175 derived from at least two IBSSS assessments during screening period. Patients will receive intravenous infusions of PRX-102 1 mg/kg or placebo every two weeks for 6 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PRX-102 | Experimental | Biological: PRX-102PRX-102 infusions every 2 weeks |
| Placebo | Placebo comparator | Other: PlaceboPlacebo infusions every 2 weeks |
Who may qualify
- Ages
- 14 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Males, age 14-45 years, naïve to enzyme replacement therapy (ERT) or off ERT or off chaperone treatment for at least 6 months and negative for anti-PRX-102 antibodies
- A documented diagnosis of Fabry disease: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN)
- eGFR by CKD-EPI > 30 ml/min/1.73 m2
- Moderate to severe gastrointestinal symptoms as defined by:
- Completed electronic BSFS diary on at least 6 of the 7 days during the week prior to randomization AND at least 11 of the 14 days during the 2 weeks prior to randomization.
Key exclusion criteria (12 listed)
- Patients will be evaluated to rule out other gastrointestinal comorbidity than Fabry disease as responsible for the gastrointestinal symptoms by:
- Use of any kind of laxatives
- Initiation of anti-diarrheal medications during the screening period
- History of renal dialysis or transplantation
- Use of, or change in dose of, angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) for less than 4 weeks prior to screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PRX-102Also: pegunigalsidase alfa, recombinant human alpha galactosidase-AIntravenous | Biological | PRX-102 |
| PlaceboIntravenous | Other | Placebo |
Primary outcome
IBSSS Part 1
Time frame: Every 2 weeks for 6 months
Irritable Bowel Syndrome Severity Score
6 secondary outcomes
Stool frequency
Time frame: After every bowel movement for 6 months
Body Weight
Time frame: Every 2 weeks for 6 months
Plasma Lyso-Gb3
Time frame: Every 4 weeks for 6 months
Plasma Gb3
Time frame: Every 4 weeks for 6 months
Urine Lyso-GB3
Time frame: Every 6 weeks for 6 months
Frequency of pain medication use
Time frame: Every 2 weeks for 6 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2018.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Raul Chertkoff, MD study director
Protalix Ltd.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing PRX-102 in people with Fabry Disease, run by Protalix.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 8 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
IBSSS Part 1 (time frame: Every 2 weeks for 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2018.