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Monoclonal Antibodies

Burosumab for Fibrous Dysplasia

Phase 2

Waitlist Available

Led By Alison M Boyce, M.D.

Research Sponsored by National Institute of Dental and Craniofacial Research (NIDCR)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed diagnosis of fibrous dysplasia

- Male sterilization (at least 6 months prior to screening). For female participants on the study the vasectomized male partner should be the sole partner for that participant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 48 weeks (adults), 50 weeks (children)

Awards & highlights

Study Summary

This trial is testing a study drug (burosumab) to see if it helps people with FD who have low blood phosphate levels. People with FD are prone to deformities, fractures, and other problems. The study will last 48 weeks, and participants will visit the NIH 3 times. They will self-inject the drug at home 1-2 times/month.

Who is the study for?

This trial is for people of any age over 1 year with fibrous dysplasia (FD) and low blood phosphate levels. Participants must be able to self-inject the study drug, burosumab, agree to use effective contraception if of reproductive potential, and have a minimum body weight of 7.5 kg. Those who are pregnant or lactating, have severe kidney issues, or have used burosumab or other investigational drugs recently cannot join.Check my eligibility

What is being tested?

The trial tests burosumab in individuals with FD who suffer from weak bones due to low phosphate levels. Over 48 weeks, participants will visit NIH three times for extensive testing including physical exams and bone scans while self-administering injections at home regularly under telehealth guidance.See study design

What are the potential side effects?

Potential side effects of burosumab may include reactions at the injection site such as redness or pain, headache, tiredness, dizziness and possibly allergic reactions in those sensitive to the drug's components.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with fibrous dysplasia.

Select...

I am a male sterilized at least 6 months ago, or my only partner is a vasectomized male.

Select...

I use a highly effective hormonal birth control method.

Select...

I weigh at least 7.5 kilograms.

Select...

My blood phosphate levels are very low and my FGF23 hormone level is high.

Select...

I am at least 1 year old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 48 weeks (adults), 50 weeks (children)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~48 weeks (adults), 50 weeks (children)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 weeks (adults), 50 weeks (children) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

The proportion of subjects achieving serum phosphate levels within the target range (Z-score -1 to +2) at Week 48

Secondary outcome measures

Adverse events and clinical safety laboratory tests for up to 4 weeks after the final burosumab dose

Change and percent change from baseline to post-baseline visits in serum phosphate, serum 1,25(OH)2D, ratio of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR).

Change and percent change in serum bone turnover markers, including procollagen 1 N-terminal propeptide (P1NP), beta crosslaps C-telopeptides (CTX), osteocalcin, and bone-specific alkaline phosphatase from baseline to 48 weeks.

+6 more

Side effects data

From 2021 Phase 2 trial • 17 Patients • NCT02304367

64%

Pain In Extremity

64%

Arthralgia

50%

Upper Respiratory Tract Infection

50%

Cough

43%

Diarrhoea

36%

Nausea

36%

Vomiting

36%

Nasopharyngitis

36%

Back Pain

36%

Muscle Spasms

29%

Hypertension

29%

Insomnia

29%

Constipation

29%

Neoplasm Progression

29%

Urinary Tract Infection

29%

Musculoskeletal Pain

29%

Pain

29%

Fall

29%

Myalgia

29%

Amylase Increased

21%

Abdominal Pain

21%

Influenza

21%

Procedural Pain

21%

Contusion

21%

Ligament Sprain

21%

Pulmonary Mass

21%

Hypomagnesaemia

21%

Oropharyngeal Pain

21%

Nasal Congestion

21%

Rash

21%

Musculoskeletal Chest Pain

21%

Restless Legs Syndrome

21%

Oedema Peripheral

21%

Vitamin D Deficiency

21%

Joint Range Of Motion Decreased

21%

Fatigue

21%

Respiratory Tract Congestion

21%

Bronchitis

14%

Injection Site Reaction

14%

Abdominal Pain Lower

14%

Lipase Increased

14%

Cardiac Arrest

14%

Creatinine Renal Clearance Decreased

14%

Hyperphosphataemia

14%

Foot Fracture

14%

Upper Respiratory Tract Congestion

14%

Asthenia

14%

Acute Respiratory Failure

14%

Hypokalaemia

14%

Hypotension

14%

Hyperglycaemia

14%

Hypocalcaemia

14%

Dyspnoea

14%

Anxiety

14%

Pyrexia

14%

Anaemia

14%

Tinnitus

14%

Toothache

14%

Decreased Appetite

14%

Bone Lesion

14%

Bone Pain

14%

Nephrolithiasis

14%

Tooth Fracture

14%

Depression

14%

Chills

14%

Exostosis

14%

Neck Pain

14%

Dizziness

14%

Headache

14%

Acute Kidney Injury

14%

Abdominal Discomfort

14%

Abdominal Distension

14%

Rib Fracture

14%

Joint Swelling

Blood Creatine Increased

Thyroid Neoplasm

Testicular Mass

Skin Lesion

Computerised Tomogram Abdomen Abnormal

Hyponatraemia

Intervertebral Disc Annular Tear

Cholangitis

Coagulopathy

Metabolic Acidosis

Multi-Organ Failure

Pickwickian Syndrome

Blood Glucose Increased

Nodule

Cystitis

Metastases To Lung

Hepatic Lesion

Flank Pain

Hydronephrosis

Skin Irritation

Liver Function Test Abnormal

Rash Papular

Melanocytic Naevus

Application Site Rash

Injection Site Bruising

Gout

Electrocardiogram Qt Prolonged

Electrocardiogram Abnormal

Hypoproteinaemia

Hip Fracture

Tooth Injury

Clonus

Viith Nerve Paralysis

H1n1 Influenza

Oxygen Saturation Decreased

Hypercalcaemia

Injection Site Swelling

Medical Device Site Reaction

Tumour Compression

Diverticulitis

Rheumatoid Arthritis

Pulseless Electrical Activity

Sialoadenitis

Blood Creatinine Increased

Waist Circumference Increased

Hypoalbuminaemia

Sensory Disturbance

Panic Attack

Micturition Urgency

Precancerous Skin Lesion

Deep Vein Thrombosis

Abdominal Pain Upper

Squamous Cell Carcinoma Of Skin

Metastases To Pancreas

Prostatomegaly

Blood Cholesterol Increased

Haemothorax

Glycosylated Haemoglobin Increased

Haematuria

Pulmonary Artery Dilatation

Hyperuricaemia

Decubitus Ulcer

Hypophosphataemia

Joint Stiffness

Agitation

Keloid Scar

Peripheral Venous Disease

Nephrocalcinosis

Vasodilatation

Skin Mass

Basal Cell Carcinoma

Actinic Keratosis

Cerebral Cyst

Sleep Talking

Skin Odour Abnormal

Urinary Incontinence

Pulmonary Embolism

Renal Impairment

Concussion

Muscle Mass

Vaginal Haemorrhage

Echocardiogram Abnormal

Eyelid Ptosis

Ascites

Bile Duct Stone

Hepatic Cyst

Poor Quality Sleep

Ovarian Cancer

Leukocytosis

Bradycardia

Tachycardia

Abdominal Mass

Dysphagia

Flatulence

Gastrooesophageal Reflux Disease

Hypoaesthesia Oral

Large Intestine Polyp

Localised Intraabdominal Fluid Collection

Poor Dental Condition

Retching

Stomatitis

Calcinosis

Chest Pain

Generalised Oedema

Seasonal Allergy

Oesophageal Candidiasis

Oral Candidiasis

Pneumonia

Sinusitis

Tooth Abscess

Viral Upper Respiratory Tract Infection

Ligament Rupture

Blood Calcium Increased

Blood Parathyroid Hormone Increased

Blood Potassium Decreased

Blood Pressure Systolic Increased

Blood Sodium Decreased

Blood Uric Acid Increased

White Blood Cell Count Decreased

Aneurysmal Bone Cyst

Bursitis

Osteosclerosis

Neuroma

Pollakiuria

Proteinuria

Renal Cyst

Dental Caries

Road Traffic Accident

Calculus Bladder

Pancreatic Carcinoma Metastatic

Pleural Effusion

Pancreatitis

Dyspepsia

Gait Disturbance

Peripheral Swelling

Gastrointestinal Infection

Gastrointestinal Viral Infection

Laceration

Post Procedural Haematoma

Scar

Skin Abrasion

Spinal Compression Fracture

Stress Fracture

Tibia Fracture

Ultrasound Kidney Abnormal

Hypercholesterolaemia

Soft Tissue Mass

Tendon Pain

Schwannoma

Squamous Cell Carcinoma

Dysgeusia

Hypoaesthesia

Sciatica

Vulva Cyst

Respiratory Acidosis

Pruritus

Septic Shock

Abdominal Hernia

Injection Site Pain

Conjunctivitis

Corona Virus Infection

Femur Fracture

Fibula Fracture

Radiation Mucositis

Musculoskeletal Stiffness

Migraine

Neuralgia

Paraesthesia

Chylothorax

Prostatic Specific Antigen Increased

100%

80%

60%

40%

20%

Study treatment Arm

KRN23 TIO

KRN23 XLH and ENS

Trial Design

1Treatment groups

Experimental Treatment

Group I: TreatmentExperimental Treatment1 Intervention

Patients receiving treatment

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Burosumab

2021

Completed Phase 3

~120

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Institute of Dental and Craniofacial Research (NIDCR)Lead Sponsor

300 Previous Clinical Trials

848,329 Total Patients Enrolled

1 Trials studying Fibrous Dysplasia

15 Patients Enrolled for Fibrous Dysplasia

Alison M Boyce, M.D.Principal InvestigatorNational Institute of Dental and Craniofacial Research (NIDCR)

3 Previous Clinical Trials

524 Total Patients Enrolled

1 Trials studying Fibrous Dysplasia

15 Patients Enrolled for Fibrous Dysplasia

Media Library

Burosumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT05509595 — Phase 2

Fibrous Dysplasia Research Study Groups: Treatment

Fibrous Dysplasia Clinical Trial 2023: Burosumab Highlights & Side Effects. Trial Name: NCT05509595 — Phase 2

Burosumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05509595 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is there still time to enrol in the experiment?

"The data available on clinicaltrials.gov reveals that this medical research project is not actively recruiting at the moment, despite being initially posted in December 5th 2022 and last revised November 29th 2022. However, there are thirteen other trials open to enrolling participants currently."

Answered by AI

What preventative measures have been taken to ensure Burosumab is secure for patient use?

"Research conducted by the Power team concluded that Burosumab is a reasonably safe medication, scoring it a 2 on their safety scale. This determination was made as this drug is currently undergoing Phase 2 trials; so far evidence has been gathered to suggest its safety but not yet its efficacy."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia

2022. "Burosumab for Fibroblast Growth Factor-23 Mediated Hypophosphatemia in Fibrous Dysplasia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05509595.

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