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Could a common asthma drug help treat a rare genetic disorder?

NCT ID NCT04240821

Disease control Sponsor: Jaclyn Tamaroff Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 03, 2026 · Updated 2 times

Summary

This study tests whether theophylline, a drug used for asthma, can help children and young adults with pseudohypoparathyroidism—a rare genetic condition causing early obesity, hormone problems, and short stature. Researchers will check for weight loss, better blood sugar control, and fewer hormone issues. About 34 people who completed a prior trial will take part.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 34 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2020

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 99 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 1\. Successful completion of the randomized clinical trial "Phase 2 Study of Theophylline Treatment of Pseudohypoparathyroidism IND 133103 (11/1/2016)". Exclusion Criteria: 1. History of a seizure disorder unrelated to hypocalcemia 2. History of a cardiac arrhythmia (not including bradycardia) 3. Hepatic insufficiency including cirrhosis and acute hepatitis (AST or ALT \>3x upper limit of normal) 4. Congestive heart failure 5. Current cigarette use or alcohol abuse 6. Pregnancy or intention to become pregnant during the next year 7. Active peptic ulcer disease 8. Current use of medications known to effect theophylline levels 9. History of hypersensitivity to theophylline or other medication components 10. Unable to comply with study procedures in the opinion of the investigator

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Conditions

The condition(s) this trial relates to.

pseudohypoparathyroidism pseudohypoparathyroidism type 1A

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Albright hereditary osteodystrophy Pseudohypoparathyroidism Pseudohypoparathyroidism type 1A

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37212, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.