Pseudohypoparathyroidism Type 1aPhase 2NCT02463409
Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy)
A Phase 2 study testing Theophylline in people with Pseudohypoparathyroidism Type 1a and Albright Hereditary Osteodystrophy, run by Vanderbilt University Medical Center at 1 site in United States.
Trial summary
This study will test an investigational drug, theophylline, in children with pseudohypoparathyroidism type 1a (PHP1a). This study involves a 3 day visit to the Vanderbilt Clinical Research Center. Read more about this study
- Who is it for?
- Ages 10 to 21 years
- Conditions the study is for
- Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
- What is being tested?
- Theophylline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2015to Oct 2016
- Phase
- Phase 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 10 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 10 to 21 years old
- English proficiency
- Clinical and genetic diagnosis of PHP1a
Who cannot join
- Use of a PDE inhibitor in the past 30 days
- History of a seizure disorder unrelated to hypocalcemia
- History of a cardiac arrhythmia (not including bradycardia)
- History of hepatic insufficiency
- AST or ALT >2x upper limit of normal
- Total bilirubin >1.5 x upper limit of normal (unless patient has a diagnosis of Gilbert's syndrome and no other causes leading to hyperbilirubinemia are identified)
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- Congestive heart failure
- Cigarette use in the past 30 days
- Alcohol use within the past 24 hours
- Current pregnancy
- Untreated hypothyroidism (defined as free T4 level < 0.6 ng/dL or TSH >10 mcU/mL)
- Active peptic ulcer disease
- Fever >101 degrees in the past 24 hours
- Current use of medications known to effect theophylline levels (listed below)
- Severe sleep apnea requiring BiPAP
- Drugs with clinically significant drug interactions with theophylline:
- Allopurinol
- Cimetidine
- Ciprofloxacin
- Clarithromycin
- Enoxacin
- Ephedrine
- Erythromycin
- Estrogen
- Flurazepam
- Fluvoxamine
- Lithium
- Lorazepam
- Methotrexate
- Midazolam
- Pentoxifylline
- Propranolol
- Rifampin
- Sulfinpyrazone
- Tacrine
- Thiabendazole
- Ticlopidine
- Troleandomycin
- Verapamil
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effects of Theophylline on cAMP Signaling in Children With Pseudohypoparathyroidism Type 1a
This study will test an investigational drug, theophylline, in children with pseudohypoparathyroidism type 1a (PHP1a). This study involves a 3 day visit to the Vanderbilt Clinical Research Center.
Read the detailed description
Study measures will be done at baseline and during a 24h infusion of IV theophylline. Theophylline levels will be drawn to ensure therapeutic dosing and to monitor for toxicity. Measures include laboratory values, response to PTH infusion and resting energy expenditure.
- Being tested
- Theophylline
- Medical subject headings
- Pseudohypoparathyroidism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TheophyllineDrug
Also called Theo
24 hour infusion of IV theophylline
| Study group | Role | What they receive |
|---|---|---|
| Theophylline | Experimental | Patients will receive a 24 hour continuous infusion of intravenous theophylline. Receives: Drug: Theophylline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Urine cAMP
Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline
Measured: 1 day
Secondary outcomes (2)
Change in Resting Energy Expenditure (REE)
Change in REE before and during treatment with theophylline
Measured: 1 day
Change in Apnea Hypopnea Index (AHI)
Change in AHI before and during treatment with theophylline
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 posted4 Jun 2015 (estimated)
- Study startJun 2015
- Primary completionOct 2016
- Study completionOct 2016
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 (2)Papers the sponsor has linked to this study.
- Other trials for Pseudohypoparathyroidism Type 1aStudies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Massachusetts General Hospital
- Responsible party
- Principal investigator, Ashley Shoemaker, Vanderbilt University Medical Center
- Investigators
- Ashley H Shoemaker, MD, MSCI, Principal investigator, Vanderbilt University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02463409
- Sponsor's protocol ID
- IRB 150497
- Status last verified
- May 2017
- First submitted
- 20 May 2015
- Last updated
- 26 Jun 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Investigators may contact Dr. Shoemaker for data after our primary analysis is completed and published (if applicable).
Publications
Landreth H, Malow BA, Shoemaker AH. Increased Prevalence of Sleep Apnea in Children with Pseudohypoparathyroidism Type 1a. Horm Res Paediatr. 2015;84(1):1-5. doi: 10.1159/000381452. Epub 2015 Apr 23.
PubMed 25925491Shoemaker AH, Lomenick JP, Saville BR, Wang W, Buchowski MS, Cone RD. Energy expenditure in obese children with pseudohypoparathyroidism type 1a. Int J Obes (Lond). 2013 Aug;37(8):1147-53. doi: 10.1038/ijo.2012.200. Epub 2012 Dec 11.
PubMed 23229731
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Theophylline on cAMP Signaling in Children With Pseudohypoparathyroidism Type 1a
- ClinicalTrials.gov ID
- NCT02463409
- Acronym / sponsor ID
- IRB 150497
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Collaborators
- Massachusetts General Hospital
- Study start
- Jun 2015
- Primary completion
- Oct 2016 (actual)
- Study completion
- Oct 2016 (actual)
- First posted
- 4 Jun 2015 (estimated)
- Results
- Posted 30 May 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 will test an investigational drug, theophylline, in children with pseudohypoparathyroidism type 1a (PHP1a). This study involves a 3 day visit to the Vanderbilt Clinical Research Center.
Detailed description
Study measures will be done at baseline and during a 24h infusion of IV theophylline. Theophylline levels will be drawn to ensure therapeutic dosing and to monitor for toxicity. Measures include laboratory values, response to PTH infusion and resting energy expenditure.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Theophylline | Experimental | Drug: TheophyllinePatients will receive a 24 hour continuous infusion of intravenous theophylline. |
Who may qualify
- Ages
- 10 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Age 10 to 21 years old
- English proficiency
- Clinical and genetic diagnosis of PHP1a
Key exclusion criteria (39 listed)
- Use of a PDE inhibitor in the past 30 days
- History of a seizure disorder unrelated to hypocalcemia
- History of a cardiac arrhythmia (not including bradycardia)
- History of hepatic insufficiency
- AST or ALT >2x upper limit of normal
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 |
|---|---|---|
| TheophyllineAlso: Theo24 hour infusion of IV theophylline | Drug | Theophylline |
Primary outcome
Change in Urine cAMP
Time frame: 1 day
Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline
2 secondary outcomes
Change in Resting Energy Expenditure (REE)
Time frame: 1 day
Change in Apnea Hypopnea Index (AHI)
Time frame: 1 day
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 25925491Landreth H, Malow BA, Shoemaker AH. Increased Prevalence of Sleep Apnea in Children with Pseudohypoparathyroidism Type 1a. Horm Res Paediatr. 2015;84(1):1-5. doi: 10.1159/000381452. Epub 2015 Apr 23.
- Background: PubMed 23229731Shoemaker AH, Lomenick JP, Saville BR, Wang W, Buchowski MS, Cone RD. Energy expenditure in obese children with pseudohypoparathyroidism type 1a. Int J Obes (Lond). 2013 Aug;37(8):1147-53. doi: 10.1038/ijo.2012.200. Epub 2012 Dec 11.
- Vanderbilt PHP Research PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Investigators may contact Dr. Shoemaker for data after our primary analysis is completed and published (if applicable).
Related trials for Pseudohypoparathyroidism Type 1a
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Setmelanotide to Treat Obesity in a Patient With Pseudohypoparathyroidism Type 1a (PHP1a)
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Albright Hereditary Osteodystrophy: Natural History, Growth, and Cognitive/Behavioral Assessments
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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.
Ashley H Shoemaker, MD, MSCI principal investigator
Vanderbilt University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Theophylline in people with Pseudohypoparathyroidism Type 1a and Albright Hereditary Osteodystrophy, run by Vanderbilt University Medical Center 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 3 inclusion and 39 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: finished, as of the sponsor's last update on 26 Jun 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. 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 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 30 May 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?
Change in Urine cAMP (time frame: 1 day). 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 NCT02463409, which also lists 2 linked publications.
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 26 Jun 2017.