Postural Orthostatic Tachycardia SyndromePhase 1/2NCT01978535
Iron Sucrose in Adolescents With Iron Deficiency and Postural Orthostatic Tachycardia Syndrome (POTS)
A Phase 1/2 study testing Iron infusion and Normal saline infusion in people with Postural Orthostatic Tachycardia Syndrome, run by Mayo Clinic at 1 site in United States.
Trial summary
This study is designed to investigate whether the treatment of non-anemic iron deficiency with intervenous iron sucrose will result in decreased symptom reporting and improved cardiovascular indices in adolescents (age 12-21) with Postural Orthostatic Tachycardia Syndrome (POTS). Read more about this study
- Who is it for?
- Ages 12 to 21 years
- Conditions the study is for
- Postural Orthostatic Tachycardia Syndrome
- What is being tested?
- Iron infusion and Normal saline infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Dec 2014to 8 Sep 2017
- Phase
- Phase 1/2
- 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 12 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- aged 12-21 years
- chronic (>3 months)symptoms of orthostatic intolerance (including but not limited to lightheadedness, syncope, headache, fatigue, weakness, sweating, nausea and palpitations)
- symptomatic orthostatic heart rate increase of greater than or equal to 40 beats per minute during a 10 minute 70 degree head up tilt study
- presence of non-anemic iron deficiency, defined as serum ferritin level less than or equal to 20 ug/L with normal hemoglobin
Who cannot join
- orthostatic hypotension within 3 minutes of 70 degree head up tilt
- pregnant or lactating females
- presence of other organ failure or systemic illness that can affect autonomic function
- concurrent medication therapy with anticholinergic, alpha-adrenergic antagonists, beta-adrenergic antagonists unless medication is held for five half-lives prior to study
- laboratory evidence of anemia or iron overload
- personal history of hematochromatosis or first degree relative with hematochromatosis
- known sensitivity to Venofer (TM) or other intravenous iron preparations
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
Official title: A Randomized, Controlled, Double Blinded Clinical Trial of Intravenous Iron Sucrose in Adolescents With Non-anemic Iron Deficiency and Postural Orthostatic Tachycardia Syndrome (POTS)
This study is designed to investigate whether the treatment of non-anemic iron deficiency with intervenous iron sucrose will result in decreased symptom reporting and improved cardiovascular indices in adolescents (age 12-21) with Postural Orthostatic Tachycardia Syndrome (POTS).
Read the detailed description
Postural orthostatic tachycardia syndrome (POTS) is characterized by an exaggerated heart rate response to the standing position in a patient with symptoms of orthostatic intolerance. It is likely a heterogeneous disorder representing a common manifestation of a number of possible underlying derangements such as impaired sympathetically mediated vasoconstriction, excessive sympathetic drive, volume dysregulation and deconditioning. While the actual prevalence of POTS is unknown, it has been estimated to affect at least 500,000 individuals in the United States. It predominately affects young individuals, and particularly women.
The prevalence of iron deficiency in adolescents with POTS has been reported to be greater than that expected in the general adolescent population. The use of intravenous iron sucrose has been shown to be a safe and effective therapy for correcting iron deficiency in children and adolescents. Anecdotal experience at our clinic has demonstrated an immediate reduction in self-reported orthostatic symptoms in several patients diagnosed with POTS following the administration of intravenous iron sucrose for iron deficiency.
This study is a randomized, double blind, placebo-controlled study to assess the efficacy of a single iron sucrose infusion in adolescents with non-anemic iron deficiency and POTS. Subjects will be screened at outpatient clinic visit appointments. Interested qualified subjects will be consented and offered participation in this trial. Once consent and assent (for patients less than 18 years of age) has been obtained, subjects will be randomized to either treatment or placebo group. Subjects will participate in two on site study visits. During the first visit subjects will complete baseline laboratory studies, tilt table test and study questionnaires. Subjects will then receive the intervention study drug or placebo. The second study visit will occur 7 days + 2 days from the first study visit. During the second study visit subjects will repeat laboratory studies, tilt table test and study questionnaires. Follow up questionnaires will be sent to all subjects six months following the initial study visit.
- Being tested
- Iron infusion, Normal saline infusion
- Keywords
- POTS
- Medical subject headings
- Postural Orthostatic Tachycardia Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Iron infusionDrug
Also called Iron sucrose, Venofer (TM)
5 mg/kg of intravenous iron sucrose supplied as Venofer (TM) with a maximum dose of 200mg. Iron sucrose will be diluted to 1 mg of elemental iron in 1 mL of NaCl 0.9% with a maximum volume of 210 mL.
Normal saline infusionDrug
Also called Normal saline, NaCl 0.9%
Normal saline (NaCl 0.9%) 5 mL/kg up to a maximum volume 210 mL
| Study group | Role | What they receive |
|---|---|---|
| Iron infusion | Experimental | Iron Sucrose (Venofer (R)) Receives: Drug: Iron infusion |
| Normal saline infusion | Placebo comparator | Equal volume to intervention of normal saline Receives: Drug: Normal saline infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cardiovascular Indices-interval Change in Heart Rate During 10-minute Head up Tilt Table Test
This study will assess whether a single infusion of iron sucrose will improve cardiovascular indices, specifically a reduction in the interval of measured heart rate change, during a ten minute head up tilt, in adolescent subjects with POTS and non-anemic iron deficiency
Measured: 7 (+/- 2) days following intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2013 (estimated)
- Study start17 Dec 2014
- Primary completion8 Sep 2017
- Study completion8 Sep 2017
Why it stopped: Difficulty in recruiting subjects.
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.
- Other trials for Postural Orthostatic Tachycardia SyndromeStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amie E. Jones, M.D., Mayo Clinic
- Investigators
- Amie Jones, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01978535
- Sponsor's protocol ID
- 12-009963
- Status last verified
- Oct 2017
- First submitted
- 7 Jun 2013
- Last updated
- 17 Nov 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Controlled, Double Blinded Clinical Trial of Intravenous Iron Sucrose in Adolescents With Non-anemic Iron Deficiency and Postural Orthostatic Tachycardia Syndrome (POTS)
- ClinicalTrials.gov ID
- NCT01978535
- Acronym / sponsor ID
- 12-009963
- Keywords
- POTS
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Difficulty in recruiting subjects.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 17 Dec 2014 (actual)
- Primary completion
- 8 Sep 2017 (actual)
- Study completion
- 8 Sep 2017 (actual)
- First posted
- 7 Nov 2013 (estimated)
- Results
- Posted 17 Nov 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to investigate whether the treatment of non-anemic iron deficiency with intervenous iron sucrose will result in decreased symptom reporting and improved cardiovascular indices in adolescents (age 12-21) with Postural Orthostatic Tachycardia Syndrome (POTS).
Detailed description
Postural orthostatic tachycardia syndrome (POTS) is characterized by an exaggerated heart rate response to the standing position in a patient with symptoms of orthostatic intolerance. It is likely a heterogeneous disorder representing a common manifestation of a number of possible underlying derangements such as impaired sympathetically mediated vasoconstriction, excessive sympathetic drive, volume dysregulation and deconditioning. While the actual prevalence of POTS is unknown, it has been estimated to affect at least 500,000 individuals in the United States. It predominately affects young individuals, and particularly women.
The prevalence of iron deficiency in adolescents with POTS has been reported to be greater than that expected in the general adolescent population. The use of intravenous iron sucrose has been shown to be a safe and effective therapy for correcting iron deficiency in children and adolescents. Anecdotal experience at our clinic has demonstrated an immediate reduction in self-reported orthostatic symptoms in several patients diagnosed with POTS following the administration of intravenous iron sucrose for iron deficiency.
This study is a randomized, double blind, placebo-controlled study to assess the efficacy of a single iron sucrose infusion in adolescents with non-anemic iron deficiency and POTS. Subjects will be screened at outpatient clinic visit appointments. Interested qualified subjects will be consented and offered participation in this trial. Once consent and assent (for patients less than 18 years of age) has been obtained, subjects will be randomized to either treatment or placebo group. Subjects will participate in two on site study visits. During the first visit subjects will complete baseline laboratory studies, tilt table test and study questionnaires. Subjects will then receive the intervention study drug or placebo. The second study visit will occur 7 days + 2 days from the first study visit. During the second study visit subjects will repeat laboratory studies, tilt table test and study questionnaires. Follow up questionnaires will be sent to all subjects six months following the initial study visit.
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 |
|---|---|---|
| Iron infusion | Experimental | Drug: Iron infusionIron Sucrose (Venofer (R)) |
| Normal saline infusion | Placebo comparator | Drug: Normal saline infusionEqual volume to intervention of normal saline |
Who may qualify
- Ages
- 12 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- aged 12-21 years
- chronic (>3 months)symptoms of orthostatic intolerance (including but not limited to lightheadedness, syncope, headache, fatigue, weakness, sweating, nausea and palpitations)
- symptomatic orthostatic heart rate increase of greater than or equal to 40 beats per minute during a 10 minute 70 degree head up tilt study
- presence of non-anemic iron deficiency, defined as serum ferritin level less than or equal to 20 ug/L with normal hemoglobin
Key exclusion criteria (7 listed)
- orthostatic hypotension within 3 minutes of 70 degree head up tilt
- pregnant or lactating females
- presence of other organ failure or systemic illness that can affect autonomic function
- concurrent medication therapy with anticholinergic, alpha-adrenergic antagonists, beta-adrenergic antagonists unless medication is held for five half-lives prior to study
- laboratory evidence of anemia or iron overload
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 |
|---|---|---|
| Iron infusionAlso: Iron sucrose, Venofer (TM)5 mg/kg of intravenous iron sucrose supplied as Venofer (TM) with a maximum dose of 200mg. Iron sucrose will be diluted to 1 mg of elemental iron in 1 mL of NaCl 0.9% with a maximum volume of 210 mL. | Drug | Iron infusion |
| Normal saline infusionAlso: Normal saline, NaCl 0.9%Normal saline (NaCl 0.9%) 5 mL/kg up to a maximum volume 210 mL | Drug | Normal saline infusion |
Primary outcome
Cardiovascular Indices-interval Change in Heart Rate During 10-minute Head up Tilt Table Test
Time frame: 7 (+/- 2) days following intervention
This study will assess whether a single infusion of iron sucrose will improve cardiovascular indices, specifically a reduction in the interval of measured heart rate change, during a ten minute head up tilt, in adolescent subjects with POTS and non-anemic iron deficiency
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Amie Jones, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Iron infusion and Normal saline infusion in people with Postural Orthostatic Tachycardia Syndrome, run by Mayo Clinic 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 4 inclusion and 7 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 17 Nov 2017. 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. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 17 Nov 2017). 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?
Cardiovascular Indices-interval Change in Heart Rate During 10-minute Head up Tilt Table Test (time frame: 7 (+/- 2) days following intervention). 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 17 Nov 2017.