Type 1 Gaucher DiseasePhase 4NCT03021941
Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
A Phase 4 study testing Elelyso 60 units/kg in people with Type 1 Gaucher Disease, run by Pfizer at 1 site in United States.
Trial summary
In August of 2014, the FDA approved ELELYSO for long-term enzyme replacement therapy (ERT) for pediatric subjects with a confirmed diagnosis of Type 1 Gaucher disease. The recommended dosage for treatment-naïve adult and pediatric subjects 4 years of age and older is 60 units per kg of body weight administered every… Read more about this study
- Who is it for?
- Ages Up to 12 years
- Conditions the study is for
- Type 1 Gaucher Disease
- What is being tested?
- Elelyso 60 units/kg
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Jul 2019to 30 Jun 2020
- Phase
- Phase 4
- 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 Up to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female pediatric subjects with a diagnosis of Type 1 Gaucher disease (evidence of leukocyte acid-glucosidase activity 30% of the mean of the reference range for healthy persons), and meeting one of three weight categories: less than15 kg; 15 to less than 20 kg or 20-25 kg.
- ERT treatment-naïve, or able to perform the PK assessment within the initial 6 months of initial Elelyso treatment (provided the subject was ERT naïve prior to the start of Elelyso).
- Have had baseline (ie, prior to the first dose of study medication) PD measurements (spleen volume/size measured by MRI, CT or ultrasound, hemoglobin/platelet counts and growth measures including height and weight), immunogenicity sample collection and Gaucher disease diagnosis history documented in advance of treatment start.
- Presence of splenomegaly at baseline defined as spleen volume/size measurement of 5 MN.
- Subjects prescribed the nominal dose of 60 units/kg every two weeks and can tolerate an infusion rate of 1 mL/min.
- Evidence of a personally signed and dated informed consent document from parent/legal guardian (or adult caregiver) capable of providing informed consent indicating that the subject's parent(s)/legal guardian has been informed of all pertinent aspects of the study before any screening procedures are performed. When age appropriate, written assent must also be obtained.
- Have parent/legal guardian (or adult caregiver) capable and willing to comply with scheduled visits, treatment plan, laboratory tests and other study procedures.
Who cannot join
- Evidence or history of clinically significant issue or the presence of a medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator, would interfere with the subject's participation in the study, cause harm to the subject or decrease compliance with the study requirements.
- Treatment with an investigational drug within 30 days (or as determined by the local requirement) or 5 half-lives preceding the PK sample collection visit, whichever is longer.
- A diagnosis of Type 2 or 3 Gaucher disease, or the presence of neurological signs and symptoms characteristic of Type 2 or 3 Gaucher disease.
- Any change during the registry study to the subjects dose of taliglucerase alfa infusion (ie, change from 60 units/kg every two weeks to a different dose of taliglucerase alfa) or a change in infusion duration or rate or a change of ERT medication (ie, switch from taliglucerase alfa to a different ERT).
- In the judgment of the Investigator, the subject's vital signs (eg, blood pressure, pulse) prior to infusion on the day of the PK visit indicate that participation in the study would not be in the study candidate's best interest.
- A hemoglobin level of <10 g/dL within 30 days of the PK sample visit or on the day of the PK sample visit.
- History of sensitivity to heparin or heparin-induced thrombocytopenia. (Note: applies only if heparin lock or flush is to be used on the day of the PK sample visit).
- Parents or legal guardians who are investigational site staff members directly involved in the conduct of the study and their family members, children of site staff members otherwise supervised by the Investigator, or subjects who are children of Pfizer employees directly involved in the conduct of the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A MULTICENTER, OPEN LABEL, PHARMACOKINETICS, PHARMACODYNAMICS AND SAFETY STUDY OF ELELYSO(TM) (TALIGLUCERASE ALFA) IN PEDIATRIC SUBJECTS WITH TYPE 1 GAUCHER DISEASE
In August of 2014, the FDA approved ELELYSO for long-term enzyme replacement therapy (ERT) for pediatric subjects with a confirmed diagnosis of Type 1 Gaucher disease. The recommended dosage for treatment-naïve adult and pediatric subjects 4 years of age and older is 60 units per kg of body weight administered every other week as a 60 to 120 minute intravenous infusion. As a postmarketing commitment, the Sponsor agreed to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of Elelyso (taliglucerase alfa) in pediatric subjects with Type 1 Gaucher Disease. in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body weight of 20-25 kg with Type 1 Gaucher disease dosed at 60 units/kg every other week.
When applicable, PD measurements for children enrolled in the PK study may be obtained through the taliglucerase alfa registry (PMR 1895-5) and will include organ volumes (spleen and liver), hematological values (hemoglobin and platelets) as well as growth (height and weight) data. Safety data, including any serious hypersensitivity reactions, such as anaphylaxis, as well as changes in antibody status (ie, detection and titers of binding and neutralizing antibodies, and detection of IgE antibodies), will also be collected through the taliglucerase alfa registry.
Read the detailed description
This study (B3031003) is an open-label study in pediatric subjects with Type 1 Gaucher Disease to characterize PK, PD and safety following an infusion of taliglucerase alfa in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body weight of 20-25 kg. The PK sample collection will take approximately 4 hours to complete and will be performed one month or up to 6 months after the subject's first taliglucerase infusion in the registry study. The subject will be contacted the day after the PK samples are collected to assess any continuing or new adverse events and to review the subject's concomitant medications. Body weight at the time of the PK blood draw will determine the weight category for each subject. Subjects enrolled into the study will be assigned the same unique subject number assigned to them in the registry study (B3031002). This will allow linkage to relevant data from the registry study for analysis in this study.
For the purposes of this study, baseline evaluations will be obtained from the registry study and must be performed prior to the subject's first dose of taliglucerase alfa. The Month 6 and Month 12 evaluations will be performed 6 months and 12 months after the start of taliglucerase alfa treatment, respectively.
Subjects will be eligible for the PK study (B3031003) only if the PD assessments (spleen volume/size, hemoglobin/platelet counts and height/weight measurements) immunogenicity data and Gaucher disease diagnostic history are available from the registry study baseline visit and prior to the start of taliglucerase alfa treatment. If liver volume/size is available, it will also be analyzed but is not necessary for eligibility for the PK study.
Pediatric subjects prescribed 60 units/kg of taliglucerase alfa every other week by their physician will be recruited from the registry study. Subjects can be screened for the PK study at the same time as they are enrolled into the registry study (ERT treatment-naïve subjects) or up to 6 months after they are enrolled into the registry study (previously ERT naïve subjects) if PD assessments (spleen volume/size, hemoglobin/platelet counts and height/weight measurements), immunogenicity data and Gaucher disease diagnostic history (residual enzyme activity and genotype data) were performed at baseline of the registry study and prior to the start of taliglucerase alfa treatment.
Baseline data from the registry study for PD and immunogenicity testing are defined as PD measurements (spleen volume/size, liver volume/size (if available), hemoglobin and platelet counts and growth measures), Gaucher disease diagnostic history and immunogenicity samples that were collected at entry into the registry study and prior to the start of taliglucerase alfa treatment.
For the secondary PD endpoints, spleen volume/size and liver volume/size (if available) will be measured using MRI, CT or ultrasound, whichever is the standard of care according to the investigator. The method used to measure spleen and liver volume/size at Baseline will be the same one used at Month 12. Change from Baseline and percent change from Baseline at Month 12 of registry study will be calculated for spleen volume, liver volume (if available), hemoglobin and platelet counts, and growth measures (height, weight and Z-scores).
For each subject enrolled in this study, safety data while on taliglucerase alfa, starting from the baseline visit (entry into the registry study) and continuing until 28 days after the Month 12 PD data collection visit, will be obtained for reporting.
Safety assessments for the study will include collection of all adverse event and serious adverse event data, including serious hypersensitivity reactions, procedures for immunogenicity testing (ie, detection and titers of binding and neutralizing antibodies and detection of IgE antibodies) as well as vital signs taken as standard of care during infusions. A pre-dose blood sample for testing of antidrug antibodies (ADA) on the day of PK Sample Visit will be collected in an effort to assess the impact of immunogenicity on PK.
- Conditions
- Type 1 Gaucher Disease
- Being tested
- Elelyso 60 units/kg
- Keywords
- Gaucher Disease, Enzyme Replacement Therapy
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Elelyso 60 units/kgDrug
All patients receive Elelyso 60 units/kg.
| Study group | Role | What they receive |
|---|---|---|
| Elelyso 60 units/kg | Other | All patients receive 60 units/kg of Elelyso. Receives: Drug: Elelyso 60 units/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetics - AUCinf
AUCinf of taliglucerase alfa assessed within 6 months from the subject's first dose of taliglucerase alfa.
Measured: Up to 6 Months
Pharmacokinetics - AUClast
AUClast of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Measured: Up to 6 Months
Pharmockinetics - Cmax
Cmax of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Measured: Up to 6 Months
Pharmacokinetics - t½
t½ of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Measured: Up to 6 Months
Pharmacokinetics - CL
CL of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Measured: Up to 6 Months
Pharmacokinetics - Vss
Vss of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Measured: Up to 6 Months
Secondary outcomes (7)
Pharmacodynamics - Spleen volume/size
Spleen volume/size measured at Baseline and at Month 12.
Measured: 12 Months
Pharmacodynamics - Liver volume/size
Liver volume/size measured at Baseline and at Month 12.
Measured: 12 Months
Pharmacodynamics - Growth measurements
Growth measurements taken at Baseline and at Month 12.
Measured: 12 Months
Pharmacodynamics - Hemoglobin and Platelet Counts
Hemoglobin and platelet counts measured at Baseline and at Month 12.
Measured: 12 Months
Safety - Antibody Assessment
Antibody assessment measured at Month 6 and Month 12.
Measured: 12 Months
Safety - Adverse Events related to IV Infusion
Adverse events related to intravenous infusions of taliglucerase alfa.
Measured: 12 Months
Safety - Vital Signs
Vital signs prospectively collected during the taligluceras alfa infusion associated with PK measurements.
Measured: 1 Day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jan 2017 (estimated)
- Study start31 Jul 2019 (estimated)
- Primary completion30 Jun 2020 (estimated)
- Study completion30 Jun 2020 (estimated)
Why it stopped: In a communication dated June 12, 2019 the FDA has released Pfizer from the Postmarketing Commitment (PMC 2767-2) that was associated with study B3031003.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type 1 Gaucher DiseaseStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Other
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03021941
- Sponsor's protocol ID
- B3031003
- Status last verified
- Aug 2019
- First submitted
- 23 Sep 2016
- Last updated
- 24 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A MULTICENTER, OPEN LABEL, PHARMACOKINETICS, PHARMACODYNAMICS AND SAFETY STUDY OF ELELYSO(TM) (TALIGLUCERASE ALFA) IN PEDIATRIC SUBJECTS WITH TYPE 1 GAUCHER DISEASE
- ClinicalTrials.gov ID
- NCT03021941
- Acronym / sponsor ID
- B3031003
- Conditions
- Type 1 Gaucher Disease
- Keywords
- Gaucher Disease, Enzyme Replacement Therapy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: In a communication dated June 12, 2019 the FDA has released Pfizer from the Postmarketing Commitment (PMC 2767-2) that was associated with study B3031003.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 31 Jul 2019 (estimated)
- Primary completion
- 30 Jun 2020 (estimated)
- Study completion
- 30 Jun 2020 (estimated)
- First posted
- 16 Jan 2017 (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.
In August of 2014, the FDA approved ELELYSO for long-term enzyme replacement therapy (ERT) for pediatric subjects with a confirmed diagnosis of Type 1 Gaucher disease. The recommended dosage for treatment-naïve adult and pediatric subjects 4 years of age and older is 60 units per kg of body weight administered every other week as a 60 to 120 minute intravenous infusion. As a postmarketing commitment, the Sponsor agreed to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of Elelyso (taliglucerase alfa) in pediatric subjects with Type 1 Gaucher Disease. in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body weight of 20-25 kg with Type 1 Gaucher disease dosed at 60 units/kg every other week.
When applicable, PD measurements for children enrolled in the PK study may be obtained through the taliglucerase alfa registry (PMR 1895-5) and will include organ volumes (spleen and liver), hematological values (hemoglobin and platelets) as well as growth (height and weight) data. Safety data, including any serious hypersensitivity reactions, such as anaphylaxis, as well as changes in antibody status (ie, detection and titers of binding and neutralizing antibodies, and detection of IgE antibodies), will also be collected through the taliglucerase alfa registry.
Detailed description
This study (B3031003) is an open-label study in pediatric subjects with Type 1 Gaucher Disease to characterize PK, PD and safety following an infusion of taliglucerase alfa in at least 5 subjects with body weight less than 15 kg; at least 5 subjects with body weight 15 to less than 20 kg; and at least 5 subjects with body weight of 20-25 kg. The PK sample collection will take approximately 4 hours to complete and will be performed one month or up to 6 months after the subject's first taliglucerase infusion in the registry study. The subject will be contacted the day after the PK samples are collected to assess any continuing or new adverse events and to review the subject's concomitant medications. Body weight at the time of the PK blood draw will determine the weight category for each subject. Subjects enrolled into the study will be assigned the same unique subject number assigned to them in the registry study (B3031002). This will allow linkage to relevant data from the registry study for analysis in this study.
For the purposes of this study, baseline evaluations will be obtained from the registry study and must be performed prior to the subject's first dose of taliglucerase alfa. The Month 6 and Month 12 evaluations will be performed 6 months and 12 months after the start of taliglucerase alfa treatment, respectively.
Subjects will be eligible for the PK study (B3031003) only if the PD assessments (spleen volume/size, hemoglobin/platelet counts and height/weight measurements) immunogenicity data and Gaucher disease diagnostic history are available from the registry study baseline visit and prior to the start of taliglucerase alfa treatment. If liver volume/size is available, it will also be analyzed but is not necessary for eligibility for the PK study.
Pediatric subjects prescribed 60 units/kg of taliglucerase alfa every other week by their physician will be recruited from the registry study. Subjects can be screened for the PK study at the same time as they are enrolled into the registry study (ERT treatment-naïve subjects) or up to 6 months after they are enrolled into the registry study (previously ERT naïve subjects) if PD assessments (spleen volume/size, hemoglobin/platelet counts and height/weight measurements), immunogenicity data and Gaucher disease diagnostic history (residual enzyme activity and genotype data) were performed at baseline of the registry study and prior to the start of taliglucerase alfa treatment.
Baseline data from the registry study for PD and immunogenicity testing are defined as PD measurements (spleen volume/size, liver volume/size (if available), hemoglobin and platelet counts and growth measures), Gaucher disease diagnostic history and immunogenicity samples that were collected at entry into the registry study and prior to the start of taliglucerase alfa treatment.
For the secondary PD endpoints, spleen volume/size and liver volume/size (if available) will be measured using MRI, CT or ultrasound, whichever is the standard of care according to the investigator. The method used to measure spleen and liver volume/size at Baseline will be the same one used at Month 12. Change from Baseline and percent change from Baseline at Month 12 of registry study will be calculated for spleen volume, liver volume (if available), hemoglobin and platelet counts, and growth measures (height, weight and Z-scores).
For each subject enrolled in this study, safety data while on taliglucerase alfa, starting from the baseline visit (entry into the registry study) and continuing until 28 days after the Month 12 PD data collection visit, will be obtained for reporting.
Safety assessments for the study will include collection of all adverse event and serious adverse event data, including serious hypersensitivity reactions, procedures for immunogenicity testing (ie, detection and titers of binding and neutralizing antibodies and detection of IgE antibodies) as well as vital signs taken as standard of care during infusions. A pre-dose blood sample for testing of antidrug antibodies (ADA) on the day of PK Sample Visit will be collected in an effort to assess the impact of immunogenicity on PK.
Study design
- Primary purpose
- Other
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Elelyso 60 units/kg | Other | Drug: Elelyso 60 units/kgAll patients receive 60 units/kg of Elelyso. |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Male or female pediatric subjects with a diagnosis of Type 1 Gaucher disease (evidence of leukocyte acid-glucosidase activity 30% of the mean of the reference range for healthy persons), and meeting one of three weight categories: less than15 kg; 15 to less than 20 kg or 20-25 kg.
- ERT treatment-naïve, or able to perform the PK assessment within the initial 6 months of initial Elelyso treatment (provided the subject was ERT naïve prior to the start of Elelyso).
- Have had baseline (ie, prior to the first dose of study medication) PD measurements (spleen volume/size measured by MRI, CT or ultrasound, hemoglobin/platelet counts and growth measures including height and weight), immunogenicity sample collection and Gaucher disease diagnosis history documented in advance of treatment start.
- Presence of splenomegaly at baseline defined as spleen volume/size measurement of 5 MN.
- Subjects prescribed the nominal dose of 60 units/kg every two weeks and can tolerate an infusion rate of 1 mL/min.
Key exclusion criteria (8 listed)
- Evidence or history of clinically significant issue or the presence of a medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator, would interfere with the subject's participation in the study, cause harm to the subject or decrease compliance with the study requirements.
- Treatment with an investigational drug within 30 days (or as determined by the local requirement) or 5 half-lives preceding the PK sample collection visit, whichever is longer.
- A diagnosis of Type 2 or 3 Gaucher disease, or the presence of neurological signs and symptoms characteristic of Type 2 or 3 Gaucher disease.
- Any change during the registry study to the subjects dose of taliglucerase alfa infusion (ie, change from 60 units/kg every two weeks to a different dose of taliglucerase alfa) or a change in infusion duration or rate or a change of ERT medication (ie, switch from taliglucerase alfa to a different ERT).
- In the judgment of the Investigator, the subject's vital signs (eg, blood pressure, pulse) prior to infusion on the day of the PK visit indicate that participation in the study would not be in the study candidate's best interest.
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 |
|---|---|---|
| Elelyso 60 units/kgAll patients receive Elelyso 60 units/kg. | Drug | Elelyso 60 units/kg |
Primary outcome
Pharmacokinetics - AUCinf
Time frame: Up to 6 Months
AUCinf of taliglucerase alfa assessed within 6 months from the subject's first dose of taliglucerase alfa.
Pharmacokinetics - AUClast
Time frame: Up to 6 Months
AUClast of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Pharmockinetics - Cmax
Time frame: Up to 6 Months
Cmax of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Pharmacokinetics - t½
Time frame: Up to 6 Months
t½ of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Pharmacokinetics - CL
Time frame: Up to 6 Months
CL of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
Pharmacokinetics - Vss
Time frame: Up to 6 Months
Vss of taliglucerase alfa assessed within 6 months after the subject's first dose of taliglucerase alfa.
7 secondary outcomes
Pharmacodynamics - Spleen volume/size
Time frame: 12 Months
Pharmacodynamics - Liver volume/size
Time frame: 12 Months
Pharmacodynamics - Growth measurements
Time frame: 12 Months
Pharmacodynamics - Hemoglobin and Platelet Counts
Time frame: 12 Months
Safety - Antibody Assessment
Time frame: 12 Months
Safety - Adverse Events related to IV Infusion
Time frame: 12 Months
Safety - Vital Signs
Time frame: 1 Day
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 24 Sep 2019.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
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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.
Pfizer CT.gov Call Center study director
Pfizer
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Elelyso 60 units/kg in people with Type 1 Gaucher Disease, run by Pfizer at 1 site in United States.
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 7 inclusion and 8 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 24 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Pharmacokinetics - AUCinf (time frame: Up to 6 Months). The study lists 6 primary outcomes in all. 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 24 Sep 2019; AzTrials last refreshed its copy on 8 Nov 2026.