Phase 2 N=15 Basic Science

PET Imaging of Phosphodiesterase-4 (PDE4) in Brain and Peripheral Organs of McCune-Albright Syndrome

Nervous System Disease

Bottom Line

View on ClinicalTrials.gov: NCT02743377 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Oct 2020

Primary outcome: Primary: Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) - Bladder — 575.1; 650.8 SUV x min

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Brain PET Imaging with 11C Rolipram (Other); Whole Body PET Baseline with 11C Rolipram (Drug); Whole Body PET Blocked with Roflumilast (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: National Institute of Mental Health (NIMH)
Primary completion: Oct 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) - Bladder	575.1; 650.8	—
PRIMARY Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) -Gallbladder	581.2; 417.9	—
PRIMARY Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) - Heart	27.4; 25.4	—
PRIMARY Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) - Kidneys	63.3; 80.4	—
PRIMARY Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) - Liver	47.6; 52.4	—
PRIMARY Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) - Lungs	10.2; 9.6	—
PRIMARY Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) -Spleen	20.1; 18.7	—
PRIMARY Standard UptakeValue (SUV) Area Under the Curve (AUC) (30-120min) - Stomach	39.4; 34.1	—
PRIMARY Whole Brain Total Distribution Volume (VT)	0.67; 0.52	—
PRIMARY MAS Affected Bone SUV AUC(60-120min) at Baseline	11.2	—
PRIMARY MAS Affected Bone SUV AUC(60-120min) - Blocked	11.1	—
SECONDARY MAS Affected Bone SUV AUC(60-120min) - for Baseline and Blocked Subjects With MAS	13.5	—
SECONDARY Normal Bone SUV AUC (60-120min) - for Healthy Controls	4.2	—

Summary

Background: McCune-Albright Syndrome (MAS) is a disorder that affects the bones, skin, and some hormone-producing tissues. It is associated with a mutation in a gene. This gene affects enzymes in the brain and body. Researchers want to learn more about one of these enzymes, Phosphodiesterase 4 (PDE4), in people with MAS. Objective: To see if people with MAS have higher levels of PDE4 than people without MAS. Eligibility: People ages 18 and older who have MAS and participated in protocol 98-D-0145, Screening and Natural History of Patients with Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome. Healthy adult volunteers are also needed. Design: This study requires 1 to 4 outpatient visits to the NIH Clinical Center. Some visits may take place on the same day. Participants with MAS will be screened with medical history and physical exam. They will have blood and urine tests. Participants will have a magnetic resonance imaging scan. Participants will have a full body positron emission tomography (PET) scan. A small amount of a radioactive chemical, [11C](R)-rolipram, will be given through an intravenous tube. Participants will have a brain PET scan with [11C](R)-rolipram. For this, a thin plastic tube will also be put into an artery at their wrist or elbow crease area. For the scans, participants will lie on a bed that slides in and out of a scanner. They may wear a plastic mask to hold their head in place. They will have blood drawn. Participants with MAS will be interviewed about their thinking and mood. They may complete questionnaires about how they feel or think.

Eligibility Criteria

INCLUSION CRITERIA:

Subjects with MAS:

At least 18 years of age
Able to provide self-consent
Diagnosed with MAS under 98-D-0145.
Have craniofacial fibrous dysplasia

Healthy Subjects:

At least 18 years of age.
Healthy based on medical history and physical examination.

EXCLUSION CRITERIA

Subjects with MAS:

Serious medical conditions, which may interfere with study procedures. Such conditions include but not limited to significant bone abnormalities in wrist areas of both arms, which makes it difficult to place a radial arterial line, clinically marked dysfunction of liver or kidney, which may delay clearance of [(11)C](R)-rolipram.
Clinically significant laboratory abnormalities not linked to endocrine abnormalities but that may interfere with the PET measurement or affect safety of the participant during this study.
Positive HIV test.
Head trauma resulting in a period of unconsciousness lasting longer than one hour.
Metallic foreign bodies that would be affected by the magnetic resonance imaging (MRI) magnet, or fear of enclosed spaces likely to make the subject unable to undergo an MRI scan.
Recent research-related exposure to radiation (i.e., PET from other research) that, when combined with this study, would be above the allowable limits.
Inability to lie flat on camera bed for about two and a half hours.
Pregnancy or breastfeeding.
NIMH employees/staff and their immediate family members will be excluded from the study per NIMH policy.

Healthy Subjects:

Serious medical conditions, which may interfere with study procedures. Such conditions include but not limited to clinically marked dysfunction of liver or kidney, which may delay clearance of [(11)C](R)-rolipram.
Clinically significant laboratory abnormalities that may interfere with the PET measurement or affect safety of the participant during this study.
Personal history of any DSM Axis I disorder.
Positive HIV test.
Head trauma resulting in a period of unconsciousness lasting longer than one hour.
Metallic foreign bodies that would be affected by the MRI magnet, or fear of enclosed spaces likely to make the subject unable to undergo an MRI scan.
Recent research-related exposure to radiation (i.e., PET from other research) that, when combined with this study, would be above the allowable limits.
Inability to lie flat on camera bed for about two and a half hours.
Pregnancy or breastfeeding.
Current substance use disorder based on DSM.
NIMH employees/staff and their immediate family members will be excluded from the study per NIMH policy.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02743377). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.