Trial · NCT01001598
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
NCT01001598, Phase 1, Phase 2, Terminated, Boston Children's Hospital; results first posted 2019-02-19
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita. Trials, record NCT01001598. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01001598
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
- NCT number
- NCT01001598
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 5 participants, actual
- Lead sponsor
- Boston Children's Hospital
- Conditions
- Fanconi Anemia, Dyskeratosis Congenita
- Interventions
- danazol
- First posted
- 2009-10-26
- Last updated
- 2019-02-19
- Results first posted
- 2019-02-19
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Toxicity Associated With Danazol Therapy: Virilization, and/or New or Progressive Evidence of Either Hepatic or Renal Toxicity at a Grade II Level Using National Cancer Institute Common Toxicity Criteria (NCI-CTC). | 48 weeks (24 weeks treatment and 24 weeks extension phase) | All toxicities were collected and adjudicated to definitely-related, possibly-related, or unrelated to the treatment. |
| Secondary | The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | 12, 18 and 24 weeks | The optimal dose could not be calculated because the number of participants needed to do this were not enrolled. Hematologic response rate (HR) was calculated for each participant at Week 12, 18, and 24. HR was defined by hemoglobin (Hg), platelets or neutrophil response. Please find the evaluation criteria used below: Hemoglobin response: Hgb≥8 g/dL if baseline Hgb≤7 g/dL, or Hgb rise ≥1 g/dL from baseline if baseline Hgb\>7 g/dL. No RBC transfusion during the 8 weeks prior to response evaluation. Platelet response: Platelet count ≥30,000/ μL if baseline platelet count ≤20,000/ μL, or platelet count rise \>10,000/ μL from baseline if baseline platelet count \>20,000/ μL. No platelet transfusion during the 4 weeks prior to response evaluation. ANC response: ANC count ≥1,000/ μL if baseline ANC count ≤500/ μL, or ANC count rise \>500/ μL from baseline if baseline ANC count \>500/ μL. |
| Secondary | The Gene Expression Profile of Progenitor Cells in Response to Danazol, Both to Predict Responsiveness and to Screen for Small Molecules That Show a Profile Similar to That of Responsive Patients | Baseline and 24 weeks | The gene expression profiles were planned to be run on bone marrow samples collected from patients at baseline and 24 weeks but bone marrow was never collected at 24 weeks. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Toxicity Associated With Danazol Therapy: Virilization, and/or New or Progressive Evidence of Either Hepatic or Renal Toxicity at a Grade II Level Using National Cancer Institute Common Toxicity Criteria (NCI-CTC). | 48 weeks (24 weeks treatment and 24 weeks extension phase) | 4 patients with Fanconi anemia and 1 with dyskeratosis congenita | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | 12, 18 and 24 weeks | 2 participants withdrew before HR could be assessed. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | The Gene Expression Profile of Progenitor Cells in Response to Danazol, Both to Predict Responsiveness and to Screen for Small Molecules That Show a Profile Similar to That of Responsive Patients | Baseline and 24 weeks | Baseline samples were too noisy to determine gene expression. |
Source ClinicalTrials.gov
Results by group11
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
Number of Participants With Toxicity Associated With Danazol Therapy: Virilization, and/or New or Progressive Evidence of Either Hepatic or Renal Toxicity at a Grade II Level Using National Cancer Institute Common Toxicity Criteria (NCI-CTC). | Danazol | Virilization - definitely related | 2 | Participants | |
Number of Participants With Toxicity Associated With Danazol Therapy: Virilization, and/or New or Progressive Evidence of Either Hepatic or Renal Toxicity at a Grade II Level Using National Cancer Institute Common Toxicity Criteria (NCI-CTC). | Danazol | Hepatic - possibly related | 1 | Participants | |
| The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | Danazol | HR by hemoglobin at 12 weeks | 3 | Participants | |
| The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | Danazol | HR by hemoglobin at 18 weeks | 3 | Participants | |
| The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | Danazol | HR by hemoglobin at 24 weeks | 3 | Participants | |
| The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | Danazol | HR by platelets at 12 weeks | 0 | Participants | |
| The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | Danazol | HR by platelets at 18 weeks | 0 | Participants | |
| The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | Danazol | HR by platelets at 24 weeks | 2 | Participants | |
| The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | Danazol | HR by neutrophils at 12 weeks | 0 | Participants | |
| The Optimal Dose and Number of Participants With Hematologic Response Rate in Fanconi Anemia (FA) and Dyskeratosis Congenita (DC) Patients Receiving Danazol Therapy | Danazol | HR by neutrophils at 18 weeks | 0 | Participants |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Danazol | 5 | |
| Overall Study | COMPLETED | Danazol | 3 | |
| Overall Study | NOT COMPLETED | Danazol | 2 |
Source ClinicalTrials.gov
Adverse events5
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Mood changes | Nervous system disorders | Single Arm Phase I/II Study | 1 | 5 | 1 |
| other | Constipation | Gastrointestinal disorders | Single Arm Phase I/II Study | 1 | 5 | 1 |
| other | Hepatomegaly | Hepatobiliary disorders | Single Arm Phase I/II Study | 1 | 5 | 1 |
| other | Virilization | Endocrine disorders | Single Arm Phase I/II Study | 2 | 5 | 2 |
| serious | Rash | Skin and subcutaneous tissue disorders | Single Arm Phase I/II Study | 1 | 5 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Single Arm Phase I/II Study | ||
| Total, other adverse events | Single Arm Phase I/II Study | 2 | 5 |
| Total, serious adverse events | Single Arm Phase I/II Study | 1 | 5 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01001598
GET https://conductscience.org/api/trials/v1/NCT01001598/registered