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Gilbert's syndrome

Gilbert's syndrome, often shortened to the acronym GS, is a genetic disorder of bilirubin metabolism, found in about 5% of the population. The main symptom is elevated bilirubin (hyperbilirubinamia) leading to otherwise harmless mild jaundice. Alternative, less common names for this disorder are as follows: more...

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Medicines

Familial Benign Unconjugated Hyperbilirubinaemia
Constitutional Liver Dysfunction
Familial Non-Hemolytic-Non-Obstructive Jaundice
Icterus Intermittens Juvenilis
Low-Grade Chronic Hyperbilirubinemia
Unconjugated Benign Bilirubinemia

Signs and symptoms

The Gilbert's syndrome produces an elevated level of unconjugated bilirubin in the bloodstream but normally has no other effect. Rarely, mild jaundice may appear.

More controversially, some patients report fatigue and "brain fog" during episodes of high bilirubin levels. There is some evidence that Gilbert's syndrome also reduces the liver's ability to detoxify certain chemicals; it may be wise to avoid drugs that tax liver function, such as paracetamol.

Diagnosis

While this syndrome is considered harmless, it is clinically important because it may be confused with much more dangerous liver conditions. However, these will show other indicators of liver dysfunction. Haemolysis can be excluded by a full blood count and lactate dehydrogenase levels. Liver biopsy is rarely necessary. The onset of GS is often in childhood or early adulthood.

Normal levels of Total Bilirubin (conjugated and unconjugated) are under 20 mmol/dl.Patients with GS show only elevated unconjugated bilirubin, while conjugated is in normal ranges and forms less that 20% of the total. Levels of bilirubin in GS patients should be between 20 mmol/dl and 80 mmol/dl. It is proven that GS patients have a 30% slower Gluconuitril transferase rate than normal.

The level of Total Bilirubin is often increased if the blood sample is taken whilst fasted.

Gilbert's syndrome causes a 31% slower than normal rate of glucuronidation in the Phase II detoxification pathway of the liver. The phase II detoxification pathway of the liver deals with conjugation rather than the oxidation, reduction and hydrolysis of the phase I pathway.

More severe types of gluconitril transferase disorders like GS are Criggler-Najjar Syndrome Type I and Criggler-Najjar Syndrome Type II. Patients with type I disorder show no bilirubin detoxification and suffer from brain damage due to exessive bilirubin levels(both conjugated and unconjugated bilirubin are elevated). Infants with this disorder live not more than one year. There are cases of Criggler-Najjar Type I patients living twenty or thirty years.

It is arguable that GS is benign, due to many reported symptoms by GS patients.

Controversial Dietary Recommendations

No treatment is necessary for most individuals and Gilbert's syndrome is clinically defined as being without symptoms except for periodic mild jaundice.

Sponsored by: BioCryst Pharmaceuticals

RATIONALE: BCX-1777 may stop the growth of cancer cells by blocking the enzymes necessary for cancer cell growth.

PURPOSE: Phase I/II trial to study the effectiveness BCX-1777 in treating patients who have refractory cutaneous T-cell lymphoma.

Study Type: Interventional

Study Design: Treatment

OBJECTIVES:

* Determine the maximum tolerated dose of BCX-1777 in patients with refractory cutaneous T-cell lymphoma.

* Determine the efficacy of this drug in these patients.

* Determine the toxicity profile of this drug in these patients.

* Correlate plasma concentration of deoxyguanosine with clinical response and toxicity in patients treated with this drug.

* Determine the provisional optimal biological dose of this drug in these patients.

OUTLINE: This is an open-label, nonrandomized, dose-escalation, multicenter study.

* Patients receive BCX-1777 IV over 30 minutes every 12 hours on days 1-5 (a total of 9 doses). Treatment repeats every 21 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Cohorts of 3-6 patients receive escalating doses of BCX-1777 until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose at which no more than 2 of 6 patients experience dose-limiting toxicity.

* Phase II: Patients receive treatment as in phase I at the MTD of BCX-1777. Patients (including those who respond to treatment) are followed at 14 and 30 days, monthly for 6 months, every 2 months for 6 months, and then every 6 months thereafter.

Ages eligible for study: 18 years and above, both genders

Inclusion Criteria:

* Histologically confirmed cutaneous T-cell lymphoma (CTCL)

* Any stage except IA patch only

* Previously treated according to 1 of the following:

* Stage IA plaque, IB, or IIA:

* At least 4 prior conventional and/or experimental regimens (topical or systemic, including psoralen-ultraviolet light [PUVA] and systemic corticosteroids)

* Stage IIB, III, or IV:

* At least 1 prior systemic regimen (systemic corticosteroids and PUVA do not count as systemic regimens for this purpose)

NOTE: Repeated use of the same regimen is considered one regimen

* Measurable disease

Patient Characteristics:

Performance status

* ECOG 0-3

Life expectancy

* At least 3 months

Hematopoietic

* Granulocyte count at least 2,000/[mm.sup.3]

* Platelet count at least 75,000/[mm.sup.3]

* Hemoglobin at least 10.0 g/dL

Hepatic

* Bilirubin no greater than 1.5 times upper limit of normal (ULN) (unless due to Gilbert's syndrome)

* ALT no greater than 2 times ULN

* Alkaline phosphatase no greater than 2 times ULN

* No hepatitis B or C

Renal

* Creatinine clearance at least 45 mL/min

Other

* Not pregnant or nursing

* Negative pregnancy test

* Fertile patients must use effective contraception

* HIV negative

* Human T-cell leukemia virus type 1 (HTLV-1) negative

* No other malignancy within the past 5 years except basal cell or squamous cell skin cancer or carcinoma in situ of the cervix

* No other illness that would limit study participation

* No active serious infection not controlled by antibiotics

PRIOR CONCURRENT THERAPY:

Biologic therapy

* No concurrent anticancer antibody therapy

* No concurrent anticancer immunotherapy

* No concurrent anticancer gene therapy

* No concurrent anticancer vaccine therapy

* No concurrent anticancer angiogenesis inhibitors

* No concurrent sargramostim (GM-CSF)

* No concurrent filgrastim (G-CSF) during course 1 of therapy

Chemotherapy

* More than 21 days since prior chemotherapy unless fully recovered

* No concurrent anticancer chemotherapy

Endocrine therapy

* More than 2 weeks since prior topical corticosteroids

* No concurrent anticancer hormonal therapy

Radiotherapy

* More than 2 weeks since prior radiotherapy

* No concurrent radiotherapy

Other

* More than 2 weeks since prior antineoplastic therapy

* More than 21 days since prior investigational agents unless fully recovered

* No concurrent citrate-blood products within 30 minutes before or after study treatment

* No concurrent anticancer matrix metalloprotease inhibitors

* No other concurrent anti-CTCL therapy

* No concurrent use of tanning beds

* No other concurrent investigational agents

Location and Contact Information

Jim Alexander, MD, MPH, Study Chair, Pharma Research Corporation Tuft-New England Medical Center, Boston, Massachusetts, 02111, United State

Massachusetts

A total of 3-64 patients (3-24 for phase I and 40 for phase II) will be accrued for this study.

Study ID Numbers CDR0000301763; BIOCRYST-1777BC-103

NLM Identifier NCT00061880

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