PHP IaPhase 4NCT03718403
Effect of Theophylline in Pseudohypoparathyroidism
A Phase 4 study testing Theophylline in people with PHP Ia and PHP IB, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism. Read more about this study
- Who is it for?
- Ages 5 to 21 years
- What is being tested?
- Theophylline
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Nov 2019to 10 Apr 2032
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 5 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects with known diagnosis of PHP1A and PHP1B
- Age >5 years
Who cannot join
- • Significant kidney disease, i.e. any CKD stage
- Hypercalciuria
- Kidney stones
- Severe Heart disease
- Uncontrolled seizure disorder
- Peptic ulcer
Show all 13Show fewer
- Liver dysfunction
- Bleeding disorder
- Allergy to theophylline preparation
- Muscle disorder
- Infection
- Any neurologic disease
- Chronic drugs interacting with theophylline
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
Amita Sharma, 617-726-2908, asharma5@mgh.harvard.edu
About this study
The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.
Read the detailed description
It is an open label intervention study for the off-label use of theophylline in psuedohypoparathyroidism (PHP).Theophylline is a is non-specific phosphodiesterase inhibitor which increases the levels of secondary messenger,cAMP. PHP patients have end organ resistance to hormones involving cAMP signaling. Thus, theophylline has a potential to abrogate hormone resistance. Also, it may have a role in bone maturation, neurocognition and metabolism.
- Being tested
- Theophylline
- Keywords
- PHP
- Medical subject headings
- Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TheophyllineDrug
Theophylline will be given to maintain a peak level between 10-15 mcg/mL
| Study group | Role | What they receive |
|---|---|---|
| Single arm open labeled intervention study | Experimental | Subjects with PHP will be given theophylline to decrease the end organ resistance by increasing levels of cAMP, a second messenger. Theophylline will be dosed twice a day for a period of 52 weeks. Receives: Drug: Theophylline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of Theophylline on Bone Age in PHP
Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2018
- Study start11 Nov 2019
- Primary completion10 Apr 2030 (estimated)
- Study completion10 Apr 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PHP IaStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label intervention study, off label use of theophylline
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Amita Sharma, Massachusetts General Hospital
- Investigators
- AMITA SHARMA, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03718403
- Sponsor's protocol ID
- Theophylline in PHP
- Status last verified
- Jul 2022
- First submitted
- 8 Sep 2017
- Last updated
- 22 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Theophylline in Pseudohypoparathyroidism
- ClinicalTrials.gov ID
- NCT03718403
- Acronym / sponsor ID
- Theophylline in PHP
- Keywords
- PHP
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- 11 Nov 2019 (actual)
- Primary completion
- 10 Apr 2030 (estimated)
- Study completion
- 10 Apr 2032 (estimated)
- First posted
- 24 Oct 2018 (actual)
- 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 evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.
Detailed description
It is an open label intervention study for the off-label use of theophylline in psuedohypoparathyroidism (PHP).Theophylline is a is non-specific phosphodiesterase inhibitor which increases the levels of secondary messenger,cAMP. PHP patients have end organ resistance to hormones involving cAMP signaling. Thus, theophylline has a potential to abrogate hormone resistance. Also, it may have a role in bone maturation, neurocognition and metabolism.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label intervention study, off label use of theophylline
| Arm | Type | Receives |
|---|---|---|
| Single arm open labeled intervention study | Experimental | Drug: TheophyllineSubjects with PHP will be given theophylline to decrease the end organ resistance by increasing levels of cAMP, a second messenger. Theophylline will be dosed twice a day for a period of 52 weeks. |
Who may qualify
- Ages
- 5 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Subjects with known diagnosis of PHP1A and PHP1B
- Age >5 years
Key exclusion criteria (13 listed)
- • Significant kidney disease, i.e. any CKD stage
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 |
|---|---|---|
| TheophyllineTheophylline will be given to maintain a peak level between 10-15 mcg/mL | Drug | Single arm open labeled intervention study |
Primary outcome
Effect of Theophylline on Bone Age in PHP
Time frame: 52 weeks
Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2022.
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.
AMITA SHARMA, MD principal investigator
Massachusetts General Hospital
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 Theophylline in people with PHP Ia and PHP IB, run by Massachusetts General Hospital 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 2 inclusion and 13 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: looking for participants now, as of the sponsor's last update on 22 Jul 2022. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact AMITA SHARMA, MD (6177262908, asharma5@mgh.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effect of Theophylline on Bone Age in PHP (time frame: 52 weeks). 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 22 Jul 2022; AzTrials last refreshed its copy on 6 Nov 2026.