AzTrials
AzTrials

LeukemiaPhase 1NCT00006239

Phenylbutyrate and Tretinoin in Treating Patients With Hematologic Cancer

A Phase 1 study testing sodium phenylbutyrate and tretinoin in people with Leukemia and Myelodysplastic Syndromes, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Tretinoin may help hematologic cancer cells develop into normal white blood cells. Read more about this study

Who is it for?
Ages 18 to 120 years
What is being tested?
sodium phenylbutyrate and tretinoin
How many people
Not given
Where
1 siteUnited States
Study dates
Dec 2000to Mar 2005
Phase
Phase 1
Are there results?
None posted in the registry

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 18 to 120 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

DISEASE CHARACTERISTICS:

  • Histologically or cytologically confirmed myelodysplastic syndrome (MDS)
  • Refractory anemia
  • Primary refractory leukopenia or thrombocytopenia with morphologic features of MDS
  • Refractory anemia with excess blasts (RAEB)
  • Refractory anemia with ringed sideroblasts
  • RAEB in transformation
  • Must have excess blasts or be hematopoietically compromised, defined as one of the following:
  • RBC transfusion dependent
  • Granulocyte count less than 1,000/mm^3
  • Platelet count less than 50,000/mm^3 OR
  • Diagnosis of chronic myelomonocytic leukemia
  • Hematopoietically compromised (as defined above) OR
  • Excess blasts OR
  • Evaluable disease related symptomatology (organomegaly or leukemia cutis) OR
  • Diagnosis of acute myeloid leukemia
  • WBC less than 20,000/mm^3 and stable for at least 2 weeks
  • Unlikely to require cytotoxic therapy during study
  • No CNS or pulmonary leukostasis or CNS leukemia

PATIENT CHARACTERISTICS:

Age:

  • 18 and over

Performance status:

  • Zubrod 0-2

Life expectancy:

  • Not specified

Hematopoietic:

  • See Disease Characteristics
  • Hemoglobin at least 8 g/dL (transfusion allowed)
  • No disseminated intravascular coagulation

Hepatic:

  • Bilirubin less than 2.0 mg/dL (unless due to hemolysis or Gilbert's syndrome)

Renal:

  • Creatinine less than 2.0 mg/dL

Other:

  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception for 2 weeks prior, during, and for 3 months after study
  • No active infection

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • See Disease Characteristics
  • At least 3 weeks since prior biologic therapy, including hematopoietic growth factors, and recovered

Chemotherapy:

  • See Disease Characteristics
  • At least 3 weeks (1 month for MDS patients) since prior chemotherapy and recovered

Endocrine therapy:

  • Not specified

Radiotherapy:

  • At least 3 weeks since prior radiotherapy and recovered

Surgery:

  • Not specified

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    Baltimore, Maryland, 21231-2410

Other trials in United States

About this study

Official title: A Phase I, Dose-Finding Trial of Sodium Phenylbutrate (NSC 657802) in Combination With All Trans-retinoic Acid (ATRA, NSC 122758) in Patients With Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Tretinoin may help hematologic cancer cells develop into normal white blood cells.

PURPOSE: Phase I trial to study the effectiveness of combining phenylbutyrate and tretinoin in treating patients who have hematologic cancer.

Read the detailed description

OBJECTIVES:

  • Determine the safety and toxicity of phenylbutyrate and tretinoin in patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, or acute myeloid leukemia.
  • Determine the pharmacokinetic interaction of this regimen in these patients.
  • Determine any potential therapeutic activity of this regimen in these patients.

OUTLINE: This is a dose escalation study of tretinoin.

Patients receive phenylbutyrate IV continuously on days 1-7 of weeks 1, 5, 7, 9, 11, 13, 15, 17, and 19. Patients also receive oral tretinoin three times daily on days 1-7 of weeks 3, 5, 7, 9, 11, 13, 15, 17, and 19. Treatment continues in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of tretinoin until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 6 patients experience dose limiting toxicities.

An additional cohort of 6 patients is accrued at the MTD. These patients receive phenylbutyrate IV continuously on days 1-3 of weeks 1 and 3-18. These patients also receive oral tretinoin three times daily on days 1-3 of weeks 2-18. Treatment continues in the absence of disease progression or unacceptable toxicity.

Patients are followed every 3 months.

PROJECTED ACCRUAL: A total of 3-24 patients will be accrued for this study within 18 months.

Being tested
sodium phenylbutyrate, tretinoin
Keywords
recurrent adult acute myeloid leukemia, untreated adult acute myeloid leukemia, refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, chronic myelomonocytic leukemia, previously treated myelodysplastic syndromes, atypical chronic myeloid leukemia, myelodysplastic/myeloproliferative disease, unclassifiable, adult acute myeloid leukemia with t(8;21)(q22;q22), adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with inv(16)(p13;q22), adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with t(15;17)(q22;q12)
Medical subject headings
Leukemia, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Leukemia, Myeloid, Acute, Anemia, Refractory, Anemia, Refractory, with Excess of Blasts, Leukemia, Myelomonocytic, Chronic, Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • sodium phenylbutyrateDrug

  • tretinoinDrug

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Jan 2003 (estimated)
  2. Study startDec 2000
  3. Primary completionFeb 2005
  4. Study completionMar 2005

Resources

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 1
Primary purpose
Treatment
IDs, dates and publications
ClinicalTrials.gov ID
NCT00006239
Sponsor's protocol ID
J9879 CDR0000068164
Other IDs
R01CA067803, P30CA006973, JHOC-J9879, JHOC-99072306, NCI-T98-0068
Status last verified
Sep 2017
First submitted
11 Sep 2000
Last updated
29 Sep 2017
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing sodium phenylbutyrate and tretinoin in people with Leukemia and Myelodysplastic Syndromes, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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?

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: finished, as of the sponsor's last update on 29 Sep 2017. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00006239.

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 29 Sep 2017; AzTrials last refreshed its copy on 8 Nov 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in