Trial · NCT03910244

Pomalidomide for the Treatment of Bleeding in HHT

NCT03910244, Phase 2, Completed, The Cleveland Clinic; results first posted 2024-10-23

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Pomalidomide for the Treatment of Bleeding in HHT. Trials, record NCT03910244. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03910244

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Pomalidomide for the Treatment of Bleeding in HHT
NCT number: NCT03910244
Status: Completed
Phases: Phase 2
Enrollment: 145 participants, actual
Lead sponsor: The Cleveland Clinic
Conditions: Telangiectasia, Hereditary Hemorrhagic
Interventions: Pomalidomide Oral Product, Placebo oral capsule
First posted: 2019-04-10
Last updated: 2024-10-23
Results first posted: 2024-10-23

Source ClinicalTrials.gov

Registered outcomes26

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline Epistaxis Severity Score	4, 8, 12, 16, 20, and 24 Weeks and 4 weeks post treatment	The primary outcome measure is the change from baseline in Epistaxis Severity Score (ESS) after 6 months of treatment administration to compare the outcomes of Pomalidomide versus Placebo. The ESS ranges from 0-10 with higher scores indicating worse condition in the prior 4 weeks. The minimal important difference is 0.71
Secondary	Average Total Daily Duration of Nosebleeds - Change From Baseline	After 12 and 24 weeks of treatment, and 4 weeks post-treatment	The daily epistaxis duration is calculated as the total duration of all reported nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary. The outcome is the change between the baseline diary on the specified timepoint
Secondary	Weighted Average Total Daily Duration of Nosebleeds - Change From Baseline	After 12 and 24 weeks of treatment, and 4 weeks post-treatment	The daily epistaxis duration is calculated as the total duration of all reported nose bleeding events in a day, averaged across all days reported in a 4-week smartphone diary, weighted by the intensity, with 90%% winsorization. The outcome is the change between the baseline diary on the specified timepoint
Secondary	Total Iron Infused	Baseline through 24 Weeks	Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the 24-week treatment period. Patients with no infusions have a value of zero.
Secondary	Total Iron Infused	Baseline through 12 Weeks	Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the first 12 weeks of the treatment period. Patients with no infusions have a value of zero.
Secondary	Total Iron Infused	12 through 24 Weeks	Total iron infused (mg) is calculated as the total in 4 weeks, averaged across all visits reported through the second 12 weeks of the treatment period. Patients with no infusions have a value of zero.
Secondary	Patients With Any Packed Red Blood Cells Transfusion Through 24 Weeks	Baseline through 24 Weeks	Patients with any packed red blood cells transfusion through the 24-week treatment period
Secondary	Patients With Any Packed Red Blood Cells Transfusion Through 12 Weeks	Baseline through 12 Weeks	Patients with any packed red blood cells transfusion through the first 12 weeks of the treatment period
Secondary	Patients With Any Packed Red Blood Cells Transfusion 12-24 Weeks	12 through 24 Weeks	Patients with any packed red blood cells transfusion through the second 12 weeks of the treatment period
Secondary	Neuro-QoL - Satisfaction With Social Roles and Activities - T Score	Baseline, after 12 and 24 weeks of treatment, and 4 weeks post-treatment	The outcome measure is the Neuro-QoL Satisfaction with Social Roles and Activities T-Score to compare the outcomes of Pomalidomide versus Placebo. The Neuro-QoL Satisfaction with Social Roles and Activities Short Form (V1.1) T-score has a value of 50 representing the average general US population, with a standard deviation of 10, and with higher scores indicating more satisfaction. The minimal detectable change is 3.7 T score points

1 to 10 of 26Source ClinicalTrials.gov

See all 26 registered outcomes

Outcomes26

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change From Baseline Epistaxis Severity Score	4, 8, 12, 16, 20, and 24 Weeks and 4 weeks post treatment	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	units on a scale	MEAN
SECONDARY	Average Total Daily Duration of Nosebleeds - Change From Baseline	After 12 and 24 weeks of treatment, and 4 weeks post-treatment	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	minutes	MEAN
SECONDARY	Weighted Average Total Daily Duration of Nosebleeds - Change From Baseline	After 12 and 24 weeks of treatment, and 4 weeks post-treatment	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	minutes	MEAN
OTHER_PRE_SPECIFIED	Average Total Daily Duration of Low Intensity Nosebleeds - Change From Baseline	After 12 and 24 weeks of treatment, and 4 weeks post-treatment	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	minutes	MEAN
OTHER_PRE_SPECIFIED	Average Total Daily Duration of Medium Intensity Nosebleeds - Change From Baseline	After 12 and 24 weeks of treatment, and 4 weeks post-treatment	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	minutes	MEAN
OTHER_PRE_SPECIFIED	Average Total Daily Duration of High Intensity Nosebleeds - Change From Baseline	After 12 and 24 weeks of treatment, and 4 weeks post-treatment	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	minutes	MEAN
SECONDARY	Total Iron Infused	Baseline through 24 Weeks	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	mg/4 weeks	MEDIAN
SECONDARY	Total Iron Infused	Baseline through 12 Weeks	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	mg/4 weeks	MEDIAN
SECONDARY	Total Iron Infused	12 through 24 Weeks	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	mg/4 weeks	MEDIAN
SECONDARY	Patients With Any Packed Red Blood Cells Transfusion Through 24 Weeks	Baseline through 24 Weeks	An intent-to-treat (ITT) analysis which included all randomized participants who provided outcome data.	Participants	COUNT_OF_PARTICIPANTS

1 to 10 of 26Source ClinicalTrials.gov

See all 26 outcomes

Results by group198

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Change From Baseline Epistaxis Severity Score	Placebo	4 Weeks	-1	95% Confidence Interval -1.40 to -0.60	units on a scale
Change From Baseline Epistaxis Severity Score	Pomalidomide	4 Weeks	-1.1	95% Confidence Interval -1.50 to -0.70	units on a scale
Change From Baseline Epistaxis Severity Score	Placebo	8 Weeks	-1.3	95% Confidence Interval -1.80 to -0.70	units on a scale
Change From Baseline Epistaxis Severity Score	Pomalidomide	8 Weeks	-1.2	95% Confidence Interval -1.60 to -0.80	units on a scale
Change From Baseline Epistaxis Severity Score	Placebo	12 Weeks	-1.3	95% Confidence Interval -1.90 to -0.80	units on a scale
Change From Baseline Epistaxis Severity Score	Pomalidomide	12 Weeks	-1.6	95% Confidence Interval -2.00 to -1.20	units on a scale
Change From Baseline Epistaxis Severity Score	Placebo	16 Weeks	-1.2	95% Confidence Interval -1.90 to -0.60	units on a scale
Change From Baseline Epistaxis Severity Score	Pomalidomide	16 Weeks	-2	95% Confidence Interval -2.40 to -1.60	units on a scale
Change From Baseline Epistaxis Severity Score	Placebo	20 Weeks	-1.3	95% Confidence Interval -1.90 to -0.60	units on a scale
Change From Baseline Epistaxis Severity Score	Pomalidomide	20 Weeks	-2	95% Confidence Interval -2.40 to -1.50	units on a scale

1 to 10 of 198Source ClinicalTrials.gov

See all 198 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Pomalidomide	95
Overall Study	STARTED	Placebo	49
Overall Study	COMPLETED	Pomalidomide	63
Overall Study	COMPLETED	Placebo	41
Overall Study	NOT COMPLETED	Pomalidomide	32
Overall Study	NOT COMPLETED	Placebo	8

Source ClinicalTrials.gov

Adverse events612

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	Anaemia	Blood and lymphatic system disorders	Placebo	2	49	2
other	Anaemia	Blood and lymphatic system disorders	Pomalidomide	1	95	1
other	Increased tendency to bruise	Blood and lymphatic system disorders	Placebo	1	49	1
other	Increased tendency to bruise	Blood and lymphatic system disorders	Pomalidomide	0	95	0
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Placebo	1	49	1
other	Iron deficiency anaemia	Blood and lymphatic system disorders	Pomalidomide	5	95	5
other	Leukocytosis	Blood and lymphatic system disorders	Placebo	1	49	1
other	Leukocytosis	Blood and lymphatic system disorders	Pomalidomide	0	95	0
other	Leukopenia	Blood and lymphatic system disorders	Placebo	1	49	1
other	Leukopenia	Blood and lymphatic system disorders	Pomalidomide	9	95	15

1 to 10 of 612Source ClinicalTrials.gov

See all 612 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo	0	49
Total, all-cause mortality	Pomalidomide	1	95
Total, other adverse events	Placebo	43	49
Total, other adverse events	Pomalidomide	90	95
Total, serious adverse events	Placebo	8	49
Total, serious adverse events	Pomalidomide	22	95

Source ClinicalTrials.gov

Papers5

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
20087969	BACKGROUND	PubMed
25674101	BACKGROUND	PubMed
28314138	BACKGROUND	PubMed
39292928	DERIVED	PubMed
41719457	DERIVED	PubMed

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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Pomalidomide vs Placebo: deaths from any cause	Trial comparisons
Pomalidomide vs Placebo: serious adverse events	Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT03910244

GET https://conductscience.org/api/trials/v1/NCT03910244/registered?page=2

API reference For AI assistants