Mucopolysaccharidosis IPhase 4NCT00418821

A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

A Phase 4 study testing Laronidase in people with Mucopolysaccharidosis I and Hurler's Syndrome, run by Genzyme, a Sanofi Company at 1 site in Italy.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

The purpose of this study is to determine if laronidase is present in the breast milk of post-partum women receiving Aldurazyme® (laronidase) and the effects of Aldurazyme (laronidase) on the growth, development, and immunologic response of their breastfed infants. Read more about this study

Who is it for?: Any age, women only
Conditions the study is for: Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome
What is being tested?: Laronidase
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 2 participants
Where: 1 siteItaly
Study dates: 22 Oct 2010to 21 Dec 2022
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 Any age
Women only
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

The patient must have a documented laronidase deficiency with a fibroblast, plasma, serum, leukocyte, or dried blood spot laronidase enzyme activity assay.
Be pregnant, planning to breastfeed post-partum, and receiving Aldurazyme (laronidase) therapy while breastfeeding.
Provide signed, written informed consent prior to any protocol-related procedures. Consent of a legally authorized guardian(s) is (are) required for mothers younger than 18 years of age. If a mother is under 18 years old and can understand the consent, written informed consent is required from both the mother and the authorized guardian(s).
Provide signed, written informed consent for their infants to participate as study patients. If a mother is younger than 18 years of age, consent for mother and infant will be obtained from the legal guardian.

Who cannot join

Have a medical condition, serious intercurrent illness, or other extenuating circumstance that may interfere with study compliance, including all prescribed evaluations and follow-up activities.
Have received an investigational drug within 30 days prior to study enrollment.

Trial locations and contacts

1 site in 1 country.

Italy1 site

Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo
Rome

Other trials in Italy

About this study

Official title: A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme® (Laronidase) Treatment on Lactation in Women With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

Read the detailed description

Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient.

Conditions: Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie
Being tested: Laronidase
Medical subject headings: Mucopolysaccharidosis I

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

LaronidaseBiological
Also called Aldurazyme, Recombinant human alpha L iduronidase
dose of 0.58mg/kg body weight intravenously (IV) every week

Study groups in NCT00418821
Study group	Role	What they receive
Laronidase	Experimental	Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase and their infants who were breastfeed while the mothers were receiving laronidase. Receives: Biological: Laronidase

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Lactating Women With Serum IgG Antibodies to Laronidase
Measured: Up to 18 months
Amount of IgG Antibody Titers to Laronidase in Lactating Women
Measured: Baseline and Week 12
Number of Women Who Breastfed
Measured: Up to 18 months
Number of Women Who Were Successful at Breastfeeding
Measured: Up to 18 months
Number of Women Whose Breast Milk Contains Laronidase
Measured: Up to 18 months
Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease
Measured: Up to 18 months
Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels
Measured: Up to 18 months
Amount of uGAG in the Urine of Women
Urine samples were collected at specified intervals to measure uGAG in the urine of women. Reference range of uGAG between 2.64 - 37.65 was considered as normal. mg/g creatinine = milligram per gram of creatinine.
Measured: Baseline and Week 12
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
Measured: Baseline
Physical Examination Findings of the Mother
Physical examination was performed at specified intervals. Physical examination included the following physical observations: general appearance, skin, head, ears, eyes, nose, and throat, lymph nodes, abdomen, extremities/joints, neurological, mental status, and the following, if appropriate, breasts, external genitalia, pelvic, and rectal.
Measured: Up to 18 months
Temperature of the Mother
Measured: Baseline and Week 12
Heart Rate of the Mother
Measured: Baseline and Week 12
Respiratory Rate of the Mother
Measured: Baseline and Week 12
Blood Pressure of the Mother
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Measured: Baseline and Week 12
Weight of the Mother
Measured: Baseline and Week 12
Height of the Mother
Measured: Baseline
Number of Infants With Abnormal uGAG Levels
Measured: Up to 72 weeks
Amount of uGAG in the Urine of Infants
Urine samples were collected at specified intervals to measure uGAG in the urine of infant. Reference range of uGAG between 30 - 300 was considered as normal.
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72
Number of Participants With Medical History of the Infant:Pre-Existing Conditions
Measured: Baseline
Number of Infants With Abnormal Physical Finding
Physical examination included the following physical observations: general appearance, skin, lymph nodes, heart, lungs, abdomen, extremities/joints, neurological, mental status, breasts, external genitalia, pelvic, rectal, and Heent.
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Heart Rate of the Infant
Heart rate was measured at specified timepoints.
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Respiratory Rate of the Infant
Respiratory rate was measured at specified timepoints.
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Blood Pressure of the Infant
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Weight of the Infant
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Height of the Infant
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Temperature of the Infant
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Head Circumference of the Infant
Measured: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
Infant development was assessed with the Denver II Developmental Screening Test. It consisted of 5 areas/subscore i.e., test behavior, personal-social, fine motor, language, and gross motor. The number of participants with normal overall assessment measured using Denver II Developmental Screening Scores have been reported by visit.
Measured: Week 12, Week 24, Week 36, Week 48, and Week 72
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
Measured: Baseline, Week 12, Week 24, Week 36, and Week 72
Time to Development of IgM and IgG Antibodies to Laronidase
Measured: up to Week 72
Amount of IgG and IgM Antibody Titers to Laronidase
Measured: Baseline, Week 12, Week 24, Week 36, and Week 72

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Oct 201021 Dec 2022

First posted5 Jan 2007 (estimated)
Study start22 Oct 2010
Primary completion21 Dec 2022
Study completion21 Dec 2022

Why it stopped: Study was conducted to fulfill a post marketing commitment (PMC). FDA acknowledged closure of PMC.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Mucopolysaccharidosis IStudies 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 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 2 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00418821
Sponsor's protocol ID: OBS12874
Other IDs: 2007-007003-33, ALID01803
Status last verified: Feb 2024
First submitted: 3 Jan 2007
Last updated: 20 Feb 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Publications

Castorina M, Antuzzi D, Richards SM, Cox GF, Xue Y. Successful pregnancy and breastfeeding in a woman with mucopolysaccharidosis type I while receiving laronidase enzyme replacement. therapy. Clin Exp Obstet Gynecol. 2015;42(1):108-13.
PubMed 25864295

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
RecruitingObservational study
Cardiac Structure and Function in MPS
MPS I, MPS IV A
Active, not recruitingPhase 1/2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
RecruitingPhase 2/3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIA, Sanfilippo Syndrome

All Mucopolysaccharidosis I trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter, Multinational, Open-Label Study of the Effects of Aldurazyme® (Laronidase) Treatment on Lactation in Women With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
ClinicalTrials.gov ID: NCT00418821
Acronym / sponsor ID: OBS12874
Conditions: Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie
Phase: Phase 4
Study type: Interventional
Recruitment status: TerminatedReason stopped: Study was conducted to fulfill a post marketing commitment (PMC). FDA acknowledged closure of PMC.
Enrolment: 2 participants (actual)
Lead sponsor: Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
Collaborators: BioMarin/Genzyme LLC
Study start: 22 Oct 2010 (actual)
Primary completion: 21 Dec 2022 (actual)
Study completion: 21 Dec 2022 (actual)
First posted: 5 Jan 2007 (estimated)
Results: Posted 20 Feb 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Recruitment is not limited to the facility listed; facilities not yet active may be added upon identification of a patient.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Laronidase	Experimental	Biological: LaronidaseMothers treated with laronidase who intended to breastfeed their infants while receiving laronidase and their infants who were breastfeed while the mothers were receiving laronidase.

Who may qualify

Sex: Female only
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (4 listed)

The patient must have a documented laronidase deficiency with a fibroblast, plasma, serum, leukocyte, or dried blood spot laronidase enzyme activity assay.
Be pregnant, planning to breastfeed post-partum, and receiving Aldurazyme (laronidase) therapy while breastfeeding.
Provide signed, written informed consent prior to any protocol-related procedures. Consent of a legally authorized guardian(s) is (are) required for mothers younger than 18 years of age. If a mother is under 18 years old and can understand the consent, written informed consent is required from both the mother and the authorized guardian(s).
Provide signed, written informed consent for their infants to participate as study patients. If a mother is younger than 18 years of age, consent for mother and infant will be obtained from the legal guardian.

Key exclusion criteria (2 listed)

Have a medical condition, serious intercurrent illness, or other extenuating circumstance that may interfere with study compliance, including all prescribed evaluations and follow-up activities.
Have received an investigational drug within 30 days prior to study enrollment.

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
LaronidaseAlso: Aldurazyme, Recombinant human alpha L iduronidasedose of 0.58mg/kg body weight intravenously (IV) every week	Biological	Laronidase

Primary outcome

Number of Lactating Women With Serum IgG Antibodies to Laronidase
Time frame: Up to 18 months
Amount of IgG Antibody Titers to Laronidase in Lactating Women
Time frame: Baseline and Week 12
Number of Women Who Breastfed
Time frame: Up to 18 months
Number of Women Who Were Successful at Breastfeeding
Time frame: Up to 18 months
Number of Women Whose Breast Milk Contains Laronidase
Time frame: Up to 18 months
Amount of Laronidase in the Breast Milk of Lactating Mothers With Mucopolysaccharidosis I (MPS I) Disease
Time frame: Up to 18 months
Number of Women With Abnormal Urine Glycosaminoglycans (uGAG) Levels
Time frame: Up to 18 months
Amount of uGAG in the Urine of Women
Time frame: Baseline and Week 12
Urine samples were collected at specified intervals to measure uGAG in the urine of women. Reference range of uGAG between 2.64 - 37.65 was considered as normal. mg/g creatinine = milligram per gram of creatinine.
Number of Participants With Medical History of the Mother: Pre-Existing Conditions
Time frame: Baseline
Physical Examination Findings of the Mother
Time frame: Up to 18 months
Physical examination was performed at specified intervals. Physical examination included the following physical observations: general appearance, skin, head, ears, eyes, nose, and throat, lymph nodes, abdomen, extremities/joints, neurological, mental status, and the following, if appropriate, breasts, external genitalia, pelvic, and rectal.
Temperature of the Mother
Time frame: Baseline and Week 12
Heart Rate of the Mother
Time frame: Baseline and Week 12
Respiratory Rate of the Mother
Time frame: Baseline and Week 12
Blood Pressure of the Mother
Time frame: Baseline and Week 12
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Weight of the Mother
Time frame: Baseline and Week 12
Height of the Mother
Time frame: Baseline
Number of Infants With Abnormal uGAG Levels
Time frame: Up to 72 weeks
Amount of uGAG in the Urine of Infants
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, Week 60, and Week 72
Urine samples were collected at specified intervals to measure uGAG in the urine of infant. Reference range of uGAG between 30 - 300 was considered as normal.
Number of Participants With Medical History of the Infant:Pre-Existing Conditions
Time frame: Baseline
Number of Infants With Abnormal Physical Finding
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Physical examination included the following physical observations: general appearance, skin, lymph nodes, heart, lungs, abdomen, extremities/joints, neurological, mental status, breasts, external genitalia, pelvic, rectal, and Heent.
Heart Rate of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Heart rate was measured at specified timepoints.
Respiratory Rate of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Respiratory rate was measured at specified timepoints.
Blood Pressure of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Systolic blood pressure (SBP) and diastolic blood pressure (DBP) were measured at specified timepoints.
Weight of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Height of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Temperature of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Head Circumference of the Infant
Time frame: Baseline, Week 12, Week 24, Week 36, Week 48, and Week 72
Number of Participants With Normal Overall Assessment Measured Using Denver II Developmental Screening Scores
Time frame: Week 12, Week 24, Week 36, Week 48, and Week 72
Infant development was assessed with the Denver II Developmental Screening Test. It consisted of 5 areas/subscore i.e., test behavior, personal-social, fine motor, language, and gross motor. The number of participants with normal overall assessment measured using Denver II Developmental Screening Scores have been reported by visit.
Number of Infants With IgM and IgG Antibodies to Laronidase Present at Any Time Point
Time frame: Baseline, Week 12, Week 24, Week 36, and Week 72
Time to Development of IgM and IgG Antibodies to Laronidase
Time frame: up to Week 72
Amount of IgG and IgM Antibody Titers to Laronidase
Time frame: Baseline, Week 12, Week 24, Week 36, and Week 72

Results and safety

Results posted to the registry on 20 Feb 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Laronidase	2	1

Primary outcome: Number of Lactating Women With Serum IgG Antibodies to Laronidase

Time frame: Up to 18 months. Count of participants. Unit: Participants

Measure	Laronidase n = 1
Value	1

Population analysed: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.

Primary outcome: Amount of IgG Antibody Titers to Laronidase in Lactating Women

Time frame: Baseline and Week 12. Mean (Standard Deviation). Unit: antibody titer

Measure	Laronidase n = 1
Baseline	25600 (NA)
Week 12	51200 (NA)

Population analysed: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase

Primary outcome: Number of Women Who Breastfed

Time frame: Up to 18 months. Count of participants. Unit: Participants

Measure	Laronidase n = 1
Value	1

Population analysed: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.

Primary outcome: Number of Women Who Were Successful at Breastfeeding

Time frame: Up to 18 months. Count of participants. Unit: Participants

Measure	Laronidase n = 1
Value	1

Population analysed: Mothers treated with laronidase who intended to breastfeed their infants while receiving laronidase.

Adverse events

Time frame: Up to approximately 18 months for mother and 72 weeks for the infant.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Mothers Treated With Laronidase	0 of 1 (0%)	1 of 1 (100%)	0 of 1
Infants Who Were Breastfeed	0 of 1 (0%)	1 of 1 (100%)	0 of 1

Limitations and caveats (from the sponsor): Study was conducted to fulfill a post marketing commitment (PMC). Upon acknowledgement of closure of PMC by FDA, the study was terminated by the Sponsor.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 25864295Castorina M, Antuzzi D, Richards SM, Cox GF, Xue Y. Successful pregnancy and breastfeeding in a woman with mucopolysaccharidosis type I while receiving laronidase enzyme replacement. therapy. Clin Exp Obstet Gynecol. 2015;42(1):108-13.
Successful pregnancy and breastfeeding in a woman with mucopolysaccharidosis type I while receiving laronidase enzyme replacement therapyLink provided by the sponsor.
Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Related trials for Mucopolysaccharidosis I

Active, not recruitingObservational study
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
RecruitingObservational study
Cardiac Structure and Function in MPS
MPS I, MPS IV A
Active, not recruitingPhase 1/2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
RecruitingPhase 2/3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIA, Sanfilippo Syndrome

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.

Clinical Sciences & Operations study director

Sanofi

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 Laronidase in people with Mucopolysaccharidosis I and Hurler's Syndrome, run by Genzyme, a Sanofi Company at 1 site in Italy.

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 4 inclusion and 2 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: stopped early, as of the sponsor's last update on 20 Feb 2024. 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 Italy. 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?

Yes. Results are posted in the registry (first posted 20 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Lactating Women With Serum IgG Antibodies to Laronidase (time frame: Up to 18 months). The study lists 31 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00418821, which also lists 1 linked publication.

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 20 Feb 2024.